In the News

The Latest News (Posted September 1, 2010)...

Stem Cell Ruling Will be Appealed

The Obama administration said in August that it would appeal a court ruling challenging the legality of President Obama’s rules governing human embryonic stem cell research, as the head of the National Institutes of Health said the decision would most likely force the cancellation of dozens of experiments in diseases ranging from diabetes to Parkinson’s. Chief Judge Royce C. Lamberth’s ruling revived a moral and political debate over human embryonic stem cell research. Officials said experiments already under way could continue, but if the ruling is upheld, the government will be forced to suspend $54 million in financing for 22 scientific projects by the end of September. An additional 60 projects are threatened. Beyond human embryonic stem cell studies, research on vaccines, viruses, and lung disease could also be affected, experts said, because cells commonly used in such research were derived from either aborted fetuses or destroyed embryos. Source: New York Times 8/25/10

What to Tell the Patients After a Trial Goes Awry

Dr. Joel Ross, the founder and chief executive of the Memory Enhancement Centers of New Jersey, makes his living enrolling subjects in drug company clinical trials that are testing drugs for Alzheimer’s disease, among others. So when Eli Lilly announced in August that its promising Alzheimer’s drug was making patients worse and that it was halting two large clinical trials, it seemed likely that Dr. Ross would hear from family members of his patients. Are other experimental Alzheimer’s drugs safe? they might ask. Should they get their family members out of those Alzheimer’s studies? Not a single family member called. And Dr. Ross is not sure why. “Possibly, the overworked caregiver has precious little time to listen to news or read a paper,” Dr. Ross said. Whatever the reason, the silence of Alzheimer’s family members gives rise to an ethical question. Do families now need extra protection or warnings about the Lilly experience when they sign up Alzheimer’s patients for studies? Source: New York Times 8/23/10

Drugs Block Ebola, Marburg Viruses in Tests

Synthetic nucleotides injected into monkeys can block the replication of Ebola and Marburg viruses, suggesting it eventually may be possible to protect humans against these deadly bioterrorism agents, researchers said in August. The monkeys get very sick, but most of them survive. The agents, called morpholino oligomers, are the first drugs approved by the Food and Drug Administration (FDA) to go into clinical trials against the viruses—although those trials will, at least initially, be conducted in primates, not humans. The experimental drugs represent the first ray of hope against two viruses that are extremely difficult to deal with in natural outbreaks and that keep bioterrorism experts awake at night worrying about their potential use by unprincipled attackers. Because clinical trials cannot be conducted ethically in humans, the FDA permits such drugs to be tested in primates. If they prove successful, the drugs will then be tried when outbreaks of infection occur in Africa. Source: Los Angeles Times 8/23/10

Lilly Stops Alzheimer’s Drug Trials

Eli Lilly halted two late-stage clinical trials of an experimental Alzheimer’s treatment in August, representing a setback to one leading theory on treating the degenerative disease and a new blow to Lilly’s business prospects. The company said patients who had taken the drug, intended to reduce plaque in the brain, actually showed worse cognitive functioning and less ability to perform daily living tasks than patients who had taken a placebo. “A completely unexpected result,” Dr. Eric R. Siemers, medical director for the Alzheimer’s team at Lilly, said in an interview. The patients also had a higher risk of skin cancer. The trials involving the drug, semagacestat, began in 2008. Lilly officials said that they would still follow the more than 2,600 Alzheimer’s patients in 31 countries who were enrolled in the trials for six more months. The company is conducting a separate test of a biologic therapy aimed at Alzheimer’s. While hundreds of drugs are under study, Lilly’s halted effort was one of only five to reach late-stage clinical trials. Source: New York Times 8/17/10

Earlier News Items...

FDA May Revoke Breast Cancer Drug

The Food and Drug Administration (FDA) is expected to revoke its approval of the nation's best-selling cancer drug this week. Avastin is mainly used by patients with very advanced breast cancer. The agency is saying it doesn't help most breast cancer patients. There are some patients--maybe their genetics of their cancer is different--who might get some benefit from this drug, but many won't. Bob Goldberg at the Center for Medicine in the Public Interest says that if the FDA revokes, it is basically sending a message: not "this never works," but more like "this probably doesn't work for you, so you and your doctor should think very hard about whether it does." Avastin still is approved for lots of other cancers--colon cancer, lung cancer, brain cancer. So if the FDA revokes it for cancer, women can still get it if their doctor prescribes it off-label. It will just be a lot harder to get an insurance company to pay for it and Roche could lose half-a-billion in sales. The a message to the drug industry as a whole is that the bar is being raised--that not all drugs work on all people, and it's going to be increasingly up to drug companies not just to say "hey, this drug works," but who does it work for. Source: Marketplace 8/16/10 

Sanofi Seeks Efficiencies with New Model

The placement of a joint headquarters of Sanofi-Aventis SA’s new cancer research unit in Cambridge, Mass., announced last month, marks a turning point for the world’s third-largest pharmaceutical company. The company is dividing its vast resources into decentralized disease-based units, each with its own departments for research and development, regulatory affairs, marketing, and sales — a plan designed to identify promising drugs more quickly and weed out failures before spending billions on unsuccessful clinical trials. The cancer unit’s scientists will work closely with partners in the Boston area’s biotechnology companies, universities, and hospitals, Sanofi executives said. The company’s expanded collaborations with universities and others in the industry will allow it to bring more resources to preclinical development and early-stage clinical trials, with the aim of raising the level of scientific scrutiny sooner in the drug-development process. Source: Boston Globe 8/16/10 

Gene Testing Could Have Saved Weight-Loss Drug

Genetic testing might have helped identify people who would become depressed or suicidal while taking Sanofi-Aventis' weight loss drug Acomplia, which might have helped keep the drug on the market, U.S. researchers said. They said partial results from a study of the drug in which five people committed suicide confirmed that it increased the risk of psychiatric side effects. The study was halted in 2008 and the company pulled the drug from the market in Europe, but the researchers think genetic testing might have been able to identify people who were at risk of the side effects, and rescue the once-promising treatment, said Dr. Eric Topol of Scripps Translational Science Institute in La Jolla, Calif., whose study appears in the journal Lancet. Acomplia, known generically as rimonabant, blocks the same reward receptors in the brain that become active during marijuana use, and for some people, it caused serious bouts of anxiety and depression that led to suicide. "Finding the gene for severe adverse drug reactions is a lot easier than we ever thought it would be," Topol said in a telephone interview. Topol thinks if they had thought to collect genetic information on the study's more than 18,000 participants, they might have spared the drug. Source: Reuters/Yahoo! News 8/12/10 For more information, click here.

New Gel Could Speed Wound Healing

For three years, Connie McPherson had debilitating leg ulcers that were so painful she sometimes couldn't sleep. Despite repeated surgery, antibiotics, steroids, and other treatments, nothing helped. Then last year, she took part in a trial for a new gel aimed at chronic wounds. "It was the answer to my prayers," said McPherson, 58, a real estate agent in Tulsa, Okla. Within weeks, McPherson said the ulcer treated was completely healed. "I tried everything possible and this is the only thing that worked." The gel used to treat McPherson was developed by a team led by David Becker, a professor of cell and developmental biology at University College London. The gel, named Nexagon, works by interrupting how cells communicate and prevents the production of a protein that blocks healing. That allows cells to move faster to the wound to begin healing it. Though it has only been tested on about 100 people so far, experts say if it proves successful, the gel could have a major impact on treating chronic wounds, like leg or diabetes ulcers, and even common scrapes or injuries from accidents. The gel has also been used on a handful of people who have suffered serious chemical burns to their eyes, and is approved for this use in the United States. Source: Associated Press/Yahoo! News 8/12/10 

Biotechs Have Big Appeal for Drug Makers

Genzyme Corp., which is locked in merger talks with French drug maker Sanofi-Aventis SA, is not the first major Massachusetts biotechnology company to be pursued by a major pharmaceutical company. And it likely will not be the last. Analysts and industry executives note that drug makers and other life sciences companies have engineered a flurry of billion-dollar merger deals over the past few years as they try to expand their product lines, acquire cutting-edge technologies, and move into new markets — a trend that does not show any signs of letting up. Last year alone, Thomson Reuters, a business information firm, tracked more than 1,400 life sciences mergers worldwide. They totaled about $206 billion, including 46 deals worth more than $5 billion in Massachusetts. Many of the biggest potential deals, such as Sanofi’s pursuit of Genzyme, involve giant pharmaceutical companies trying to buy mid-to-large biotech firms to lift their product portfolio as patents on blockbuster drugs expire. Source: Boston Globe 8/11/10 

FDA Moves Toward Tighter Medical Device Oversight

Makers of X-ray machines, drug pumps, and other medical devices would have to submit more safety information to win federal approval under a proposal designed to tighten regulation of thousands of products reviewed each year. The Food and Drug Administration (FDA) released recommendations on August 2 designed to improve oversight of the U.S. device industry, including the government's ability to revoke approval for products that prove unsafe or ineffective. The agency's report comes nearly a year after its medical devices division endured a storm of criticism from public health advocates and lawmakers. The recommendations come from two internal FDA panels that were tapped to draft changes in the wake of the outside criticism. The FDA is not bound by the reports, and will accept public comments for 90 days. Source: Associated Press/Yahoo! News 8/3/10

Pentagon Questions Drug Study On Troops

The Department of Defense is investigating whether 80 wounded American service members in Iraq were improperly used as subjects in a test of a possible treatment for brain injuries, according to the Pentagon’s Office of Inspector General. The study, sponsored by the United States Naval Medical Center in San Diego, was designed to test whether a drug made to treat Tylenol overdoses, among other uses, could also reduce the harmful effects of traumatic brain injury, such as balance loss and brain function problems, in service members who had been hit by explosions. The investigation, triggered by an allegation made last year to a Department of Defense hotline, is reviewing the study for possible research misconduct on human subjects. The Pentagon has not said whether anyone was hurt as a result of the administration of the drug. Source: Boston Globe 8/3/10

Editorial: Publish Data On Drug Trials—Even When Not Flattering

Lurking beneath some of the biggest prescription drug scandals of our time is the specter of unflattering data that was known to pharmaceutical companies and could have revealed problems sooner had it been made public. Now, an international federation of drug manufacturers, whose members include the industry’s main players, has pledged to submit most clinical trial results for publication in peer-reviewed journals, whether the outcome is positive or negative. This step should inject transparency into the now-murky process of data disclosure. But it will work only if there is a concerted effort by companies, journals, and regulators to ensure that all trials are published, and in a timely manner. Disclosure of unflattering data doesn’t just provide a fuller picture of how useful and safe a drug might be in treating a given medical condition; it also provides some guidance to researchers studying similar drugs and similar illnesses. Source: Boston Globe 7/26/10

Editorial: When a Drug Fails

The flameout of an enormously expensive drug to treat advanced breast cancer will pose a critical test for the Food and Drug Administration (FDA). Will the agency have the courage to reverse course when a medical treatment that it approved based on preliminary evidence flops badly in follow-up studies? When Avastin was granted “accelerated approval” by the FDA, the primary evidence was a single clinical trial that found that the drug, when used with another drug, slowed progression of the disease, but did not significantly extend patients’ lives. Now two follow-up trials by the manufacturer have failed to confirm even those meager gains. By a 12-to-1 vote in July, an FDA advisory committee quite sensibly urged the agency to revoke Avastin’s approval for breast cancer. That would not affect its other approvals, gained through the standard regulatory process, for treating colon, lung, kidney, and brain cancers. The FDA has rarely removed drugs that were given accelerated approval and sometimes has failed even to compel completion of follow-up studies. But there are signs it may get tougher. Source: New York Times 7/25/10 

FDA Issues Hold On Much-Debated Avandia Study

Federal health officials are barring new patients from enrolling in a safety study of GlaxoSmithKline's controversial diabetes pill Avandia, a week after a panel of experts ruled that the drug increases heart risks. The Food and Drug Administration (FDA) said it issued a "partial clinical hold" on the study to update researchers on the latest concerns about Avandia, which has been under scrutiny since 2007. GlaxoSmithKline said in a statement it would halt recruitment for the so-called TIDE trial and update the study's chief investigators on last week's meeting. Patients already in the study will be permitted to continue participating. The TIDE study is designed to give a definitive assessment of whether Avandia's heart risks are greater than its chief competitor Actos. The TIDE study is supposed to enroll 16,000 patients, though safety concerns surrounding Avandia have slowed recruitment. Researchers reported last week that just 1,100 patients have volunteered for the study. Source: Associated Press/Yahoo! News 7/21/10 

FDA Panel's Vote on Avandia Reveals Mixed Opinions on Diabetes Drug's Safety

Federal advisers have delivered a mixed verdict on the diabetes drug Avandia, with a significant number of experts voting to recommend that it be pulled from the market because of safety concerns, but a majority urging to keep it available, perhaps with tough new restrictions and new warnings. The highly anticipated recommendations by the FDA advisory panel mark a crucial development in the long debate over Avandia, which was once the world's most popular diabetes drug, but has become the focus of intense debate because of concerns that it increases the risk for heart attacks and strokes. FDA Commissioner Margaret A. Hamburg will make the final decision about Avandia's future. Hamburg has pledged to make drug safety a priority, and many observers are watching what the agency does about Avandia as a potentially key moment for the FDA under the Obama administration. Agency scientists are divided about the drug's safety. Source: Washington Post 7/15/10 

Panel Hears Final Pleas on Diabetes Drug

A 67-year-old diabetes patient, Chuck Keyserling, said he had taken Avandia for a decade with good results and argued that diabetics need the drug as an option to prevent serious complications, including heart disease, the leading killer of people with diabetes. "Please think about those of us who have flourished on Avandia" and cannot take rival drug Actos, he told the advisers gathered in a hotel ballroom packed with spectators, journalists, and several television cameras. "For those people, a negative decision on Avandia may be a death sentence." On the other side, the head of a research and advocacy group said enough evidence had stacked up against Avandia. "The data is so consistent that it's time to act," said Diana Zuckerman, president of the National Research Center for Women & Families. The agency will make the final call in the coming months, but usually follows the advice of its panels. The committee also heard a plea from a Canadian researcher to continue a study aimed at providing a clearer answer on Avandia's heart risks. The trial is comparing the drug to Takeda Pharmaceutical Co's Actos, which some FDA staff see as clearly safer. Source: Reuters/Yahoo! News 7/14/10

GlaxoSmithKline Defends Avandia to U.S. Advisory Panel

GlaxoSmithKline Plc insisted its diabetes pill Avandia was safe as U.S. advisers began a two-day meeting to consider if the medicine is too dangerous to stay on the market. The British-based firm, which could face a new wave of lawsuits if the drug comes off the market, said diabetes was a serious disease that needed multiple treatment options to control blood sugar and prevent devastating complications including amputations and blindness.The pill was once the company's second-biggest drug, but its sales have plunged since safety fears erupted three years ago. Revenues totaled 771 million pounds ($1.16 billion) in 2009, equivalent to 2.7 percent of GlaxoSmithKline's group sales. Added pressure is coming from the European Medicines Agency, which has launched a new review into Avandia's risks and benefits. Source: Reuters/Yahoo! News 7/13/10 

Diabetes Drug Maker Hid Test Data on Risks, Files Indicate

In the fall of 1999, the drug giant SmithKline Beecham secretly began a study to find out if its diabetes medicine, Avandia, was safer for the heart than a competing pill, Actos, made by Takeda. Avandia's success was crucial to SmithKline, whose labs were otherwise all but barren of new products. But the study's results, completed that same year, were disastrous. Not only was Avandia no better than Actos, but the study also provided clear signs that it was riskier to the heart. But instead of publishing the results, the company spent the next 11 years trying to cover them up, according to documents recently obtained by The New York Times. The company did not post the results on its website or submit them to federal drug regulators, as is required in most cases by law. Besides the trial comparing Avandia with Actos, the company also conducted trials comparing Avandia with glyburide, a cheaper and older diabetes medicine. Source: New York Times 7/12/10 

Caustic Government Report Deals Blow to Diabetes Drug

A federal drug official dealt a severe blow to the popular diabetes drug Avandia, issuing a scathing review of a major clinical trial that its manufacturer has been using to argue that the drug was safe. The reviewer, Dr. Thomas Marciniak of the Food and Drug Administration, found a dozen instances in which patients taking Avandia appeared to suffer serious heart problems that were not counted in the study's tally of adverse events. Such repeated mistakes "should not be found even as single occurrences" and "suggest serious flaws with trial conduct," Dr. Marciniak wrote. The review of the RECORD study found one case in which a seizure patient was hospitalized for bleeding in the brain, but all mention of the episode was deleted from records. Another patient was hospitalized for 67 days after a severe stroke, but the study record showed no sign of a cardiovascular problem. Still another died after being hospitalized for a serious heart problem, but the death was listed as arising from an unknown cause and not heart-related. Correctly interpreted, Dr. Marciniak concluded, the study actually supports critics' contentions that Avandia may cause heart attacks and strokes. Source: New York Times 7/9/10 

FDA Review Spotlights Heart Risk of Diabetes Pill

In 2007, an analysis of dozens of studies first linked the drug to heart attacks. The FDA responded by adding a warning label to the drug later that year. Despite the reams of information posted online, the FDA's main problem remains one of too little data. Avandia, like many other drugs of the 1990s, was approved based on relatively small studies in several thousand patients. While those studies were sufficient to show the drug helped control blood sugar levels--the key measure for diabetes drugs--they were not large enough to detect all of the drug's potential side effects. Since 2009, the FDA has required longer, larger studies of diabetes drugs that include more high-risk patients. Source: Associated Press/Yahoo! News 7/10/10 

Editorial: More Questions About Avandia

The safety of the diabetes drug Avandia looked increasingly suspect late last month after two major studies found that it raises the risk of cardiovascular ailments. Then a third study seemed to exonerate Avandia, sowing more confusion. Expert advisers to the FDA will soon deliver a more definitive judgment, but at this point patients should probably be asking their doctors about alternatives. The FDA has compelled GlaxoSmithKline to sponsor a large randomized clinical trial, the gold standard, that will test the cardiovascular effects of both Avandia and Actos. It won't be completed until 2015, and some critics believe it should be abandoned as too risky for the Avandia-takers. The long struggle over Avandia has also raised serious questions over the degree of certainty that should be required before the FDA removes a drug from the market as too risky. And it has raised the issue of which officials should have the power to make that decision--those who approved the drug as safe and effective in the first place and might be reluctant to reverse themselves, or safety specialists focused solely on risks that emerge after the drug is in use. We side with the safety experts. Source: New York Times 7/4/10 

Experimental Diet Pill Shows Promise, Little Risk

An experimental diet pill helped about half the people who tried it lose some weight and keep it off a year later, without the heart problems that some earlier drugs caused, a study found. Arena Pharmaceuticals' lorcaserin is one of three drugs that are boosting hope for a new generation of more effective weight-loss medicines. In the study, lorcaserin caused more people to lose at least 5 percent of their body weight over one year, more than twice the rate achieved by those on dummy pills. The tablet would be the first truly novel weight-loss pill in a dozen years if it wins approval. The drug targets the same appetite pathway fen-phen did, but in a more selective, and perhaps safer, manner. Source: Associated Press/Yahoo! News 7/15/10 

Anti-TB Compounds Could Fight Neglected Diseases

Compounds being developed against tuberculosis (TB) also show promise against deadly tropical diseases threatening millions of people, two not-for-profits groups said in July, announcing a deal to speed up drug development. The Global Alliance for TB Drug Development has granted the Drugs for Neglected Diseases Initiative (DNDi) rights to develop a class of potential anti-TB compounds offering hope of treating Chagas disease, African sleeping sickness, and leishmaniasis. It is the "first-ever royalty-free license agreement between two not-for-profit drug developers," according to a statement from the New York-based TB Alliance and the Geneva-based DNDi about the deal backed by the Bill & Melinda Gates Foundation. One of the furthest advanced and most promising TB drug candidates, PA-824, is in Phase II testing, the drug developers said. Source: Reuters/Yahoo! News 7/7/10 

India Expands Role as Drug Producer

India's drug industry--on track to grow about 13 percent this year, to just over $24 billion--was once notorious for making cheap knockoffs of Western medicines and selling them in developing countries. But India, seasoned in the basics of medicine making, is now starting to take on a more mainstream role in the global drug industry, as a result of recent strengthening of patent law here and cost pressures on name-brand drug makers in the West. And while the Indian industry has had quality-control problems, it nonetheless benefits from growing wariness about the reliability of ingredients from that other historically low-cost drug provider--China. The United States is India's top export customer for drugs. The next opportunities for India could come at the more sophisticated end of the drug making spectrum, including research and development for the world's drug giants and even development of proprietary medicines. Source: New York Times 7/6/10 

When is a Drug Too Risky to Stay on the Market?

The arthritis pill Vioxx was withdrawn but menopause hormones were not, even though both were tied to heart risks. A multiple sclerosis medicine was pulled and later allowed back on. So, when is a drug too risky to stay on the market? Drug safety questions arose again in late June, as calls mount for the diabetes pill Avandia to be withdrawn. Surprisingly, the Food and Drug Administration (FDA) has no firm rules for deciding such cases—just a murky guideline of "when the risks exceed the benefits." The agency does need better criteria for weighing drug safety, an FDA official said. It has asked a group of outside scientists, the Institute of Medicine, to give advice. A report is expected before the July 13-14 hearing on Avandia, a controversial pill whose maker, GlaxoSmithKline PLC, insists is safe. One of the issues the Institute of Medicine had been asked to advise the FDA on is how to weigh comparison studies, especially if they are not gold-standard trials where similar groups of people are given one or the other pill and followed over time to see how each group fares. Source: Associated Press/Yahoo! News 6/30/10 

New Studies Add to Avandia Concerns

Two scientific studies published in late June reinforced serious health warnings about a heavily marketed diabetes drug, Avandia, made by GlaxoSmithKline PLC. The studies increase pressure on the Food and Drug Administration (FDA) as the agency prepares for a meeting July 13 to consider halting a controversial clinical trial and withdrawing the drug from the market. Avandia and a competitor drug, Actos, manufactured by Takeda Pharmaceutical Co. of Japan, are prescribed to treat type 2 diabetes. Actos has not been associated with the same negative health effects as Avandia, however, leading to questions about why the FDA should allow Avandia to remain on the market when there is an apparently safer drug that treats the same disease. One of the two new studies—a review by government researchers of data for 227,000 Medicare patients taking either Avandia or Actos—found that Avandia increased the risk of heart attack, stroke, heart failure, or death by 17 percent. The second study was performed by Cleveland Clinic cardiologist Dr. Steven Nissen, who was the first to publish a broad analysis of clinical trials in 2007 that said Avandia raised the risk of heart attack. Nissen’s new work analyzed 56 separate clinical trials involving 35,000 patients and confirm his analysis of three years ago. The study found that Avandia elevated the risk of heart attack by 28 to 39 percent. In 2008, the FDA required GlaxoSmithKline to conduct a clinical trial directly comparing the safety of Avandia and Actos, but it has been slow to recruit patients and critics say it is unethical. Source: Boston Globe 6/29/10 

FDA Panel Explores Boost for Rare Drugs

U.S. regulators are exploring how to make it easier and cheaper for drug companies to develop treatments for rare diseases--an underserved slice of the market that typically offers slim profits. The U.S. Food and Drug Administration (FDA) already offers companies grants and guarantees seven years of market exclusivity for drugs that treat rare diseases. The agency is looking to find other ways to encourage drug makers through its new rare disease review group, which held its first public hearing in late June. The new rare disease review group is part of a broadened effort to encourage companies to spend more money on the more than 6,000 rare diseases identified, including relatively well-known conditions like cystic fibrosis and Huntington's disease. Companies and advocates say that drugs for rare diseases should not have to go through as many clinical trials as other drugs. These drugs often have fewer potential test subjects, making it more expensive to coordinate and harder to meet the same scientific standards. Source: Reuters 6/28/10 

The Myth of the Perfect Drug: Focusing Too Much on Side Effects Might Forfeit Important New Medications

Writing in the Boston Globe, Christoph Westphal, a biotech entrepreneur and a partner at Longwood Founders Fund, in June said that, when it comes to prescription drugs, patients expect benefits but appear intolerant of risks. What would happen in a world that accepts no risks in its pharmaceuticals? We would have very empty medicine cabinets. In fact, it is possible that aspirin would not be approved today by the Food and Drug Administration, so dramatic is the shift in society’s risk-benefit views regarding pharmaceutical products. That shift spelled the doom a few years ago of another drug with significant benefit, but some risk, namely Vioxx. As society has shifted to a view that medications should have virtually no risks, the inevitable effect has been to reduce the flow of important new drugs. It is certainly fair to rigorously test any new medication prior to approval. In addition, any new drugs should be monitored for safety and efficacy after they have reached the market. But society must be careful to weigh the benefits of effective new drugs for diseases that until now have been poorly treated, versus the added risks of the new medications. If we focus too much on the risks of drugs, and do not balance those risks against the benefits, fewer drugs will be approved and reach patients in need. That is a risk in and of itself. Source: Boston Globe 6/28/10 

Data Show Declining Productivity in Drug R&D

Drug makers still rely heavily on sales from an aging portfolio of products and the proportion of sales from newer medicines actually fell last year, after a decade of record research spending yielded few new winners. The 2010 Pharmaceutical R&D Factbook, compiled by CMR International and released in late June, painted a gloomy picture of the global pharmaceuticals sector. New drugs launched within the last five years accounted for less than 7 percent of industry sales in 2009, down from 8 percent in 2008, the publication showed, highlighting the big problems that companies are having in trying to reinvigorate their portfolios. Many companies have been taking a knife to research operations in a bid to improve returns, a trend which analysts expect to gather momentum this year and next. As a result, total research and development (R&D) expenditure dropped by 0.3 percent in 2009, after a 6.6 percent rise in 2008 and rapid growth seen in earlier years. A total of 26 new molecular entities were launched onto the global market in 2009, an increase on 2008's 20-year low of 21, but still only a little more than half the peak level in 1997. CMR also said that the number of experimental drug projects terminated at the final Phase III stage of development had doubled in the period 2007-09 compared to 2004-06. Source: Reuters/Yahoo! News 6/27/10 

FDA to Alter Rules on Devices

Fielding complaints about poor communication and delays in reviewing and approving new products, the top U.S. medical device regulator in June said his agency will overhaul its procedures to speed innovation while protecting patient safety. "We have not been clear about our expectations and we need to be," said Dr. Jeffrey E. Shuren, who took over as director of the Food and Drug Administration’s (FDA's) Center for Devices and Radiological Health in January. Shuren said regulators are committed to making "midcourse corrections," including seeking more clinical data before devices win clearance. Hosting a "town meeting" for more than 400 medical device executives and other industry professionals in Boston, Shuren heard plenty of criticism from leaders of start-up companies who said FDA delays, mixed signals, and lack of predictability were causing confusion and threatening their businesses. An initial report on revamping the FDA’s approval process for medical devices, including changes in the development of clinical trials, is expected to be released for public comment in the coming weeks. New procedures could take effect later this year in what is known in the industry as the 510(k) medical device approval process. Source: Boston Globe 6/23/10 

Concern Over Foreign Trials for Drugs Sold in U.S.

Medical ethicists have worried for years about the growing share of new drugs whose human trials took place in foreign countries where federal auditors could not make sure patients were protected, but no one knew how big the potential problem was. However, according to a report by the inspector general of the Department of Health and Human Services, 80 percent of the drugs approved for sale in 2008 had trials in foreign countries, and 78 percent of all subjects who participated in clinical trials were enrolled at foreign sites. Ten medicines approved in 2008 were tested entirely abroad with not a single test patient in the United States, the report said. In many cases, foreign trials provide invaluable information proving that drugs are effective in a variety of ethnic groups. The report found that most foreign clinical trial sites and subjects were in Western Europe, where ethical controls over research are generally as robust as those in the United States, but that Central and South America had the highest number of subjects per site and accounted for 26 percent of all subjects enrolled at foreign trial sites. Source: New York Times 6/21/10

FDA Staff Question Female Sex-Drive Pill Data

A pill designed to offer women a counterpart to Viagra failed to boost female sex drive in two studies, U.S. drug reviewers said in June. Women who took Boehringer Ingelheim's experimental drug flibanserin also reported depression, fainting, fatigue, and other problems. The drug's tolerability was "only moderate," Food and Drug Administration (FDA) staff said. The once-a-day pill, nicknamed the "pink Viagra," is the latest attempt at a female version of Pfizer Inc's successful blue pill for men. Boehringer is seeking FDA approval to sell flibanserin by prescription for treating premenopausal women with hypoactive sexual desire disorder, defined as a lack of sex drive in otherwise healthy women. The proposed brand name is Girosa. It is not yet approved anywhere in the world. In two clinical trails, the drug increased the number of sexually satisfying experiences but failed to improve sexual desire. Source: Reuters/Yahoo! News 6/16/10 

Pfizer Adds Focus on Rare Diseases

Pfizer Inc. is setting up a rare disease research and development group in Cambridge, Mass., a move that will put the world’s largest drug maker in direct competition with local biotechnology firms such as Genzyme Corp. The move follows that of another pharmaceutical giant, Britain’s GlaxoSmithKline, which in February unveiled plans for a stand-alone rare diseases unit. Pfizer’s research will draw on the expertise the company has in marketing a treatment for hemophilia, although it is too early to say how many additional researchers Pfizer would hire for the new Cambridge unit. In addition to hemophilia, the unit will initially concentrate on treatments for muscular dystrophy and serious diseases caused by genetic mutations. Source: Boston Globe 6/16/10 

Editorial: To Act Fast on Lethal Side Effects, FDA Needs Internal Changes

If the Food and Drug Administration (FDA) hopes to get new drugs to sick patients as quickly as necessary, it must closely monitor any side effects that might appear after initial testing. Right now, the people overseeing those who monitor the drugs are the same ones who oversaw the approval process. That lack of checks and balances can easily be remedied. Congress should make the watchdogs who oversee newly approved drugs report directly to the FDA commissioner. In [recent] cases, the FDA failed to heed early warnings from its own staffers responsible for monitoring problems with approved drugs. Those staffers would have a better chance of getting their concerns heard if they reported independently to the commissioner, rather than to the Center for Drug Evaluation and Research. That center handles the initial tests and would be understandably reluctant to second-guess itself. Source: Boston Globe 6/14/10 

Why Patients Aren’t Getting the Shingles Vaccine

In 2006, the Food and Drug Administration approved a new vaccine against shingles. Clinical trials on the vaccine revealed that it could, with relatively few side effects, reduce the risk of developing shingles by more than half, and the risk of post-herpetic neuralgia by over two-thirds. In 2008, a national panel of experts on immunizations at the Centers for Disease Control and Prevention went on to recommend the vaccine to all adults age 60 and older. However, since the vaccine became available, fewer than 10 percent of all eligible patients have received it. Last month in The Annals of Internal Medicine, researchers from the University of Colorado in Denver and the Centers for Disease Control and Prevention surveyed almost 600 primary care physicians and found that fewer than half strongly recommended the shingles vaccine. Doctors were not worried about safety — a report in the same issue of the journal confirmed that the vaccine has few side effects; rather, they were concerned about patient cost. Source: New York Times 6/10/10 

Drug Companies Share Data to Speed Brain Research

In an unusual step, a dozen competing drug companies have agreed to share data on thousands of Alzheimer's patients in hopes that the extra information will spark new ideas for treatments. The database covers 4,000 patients and counting. It was created in consultation with government regulators and the National Institutes of Health. In pooling resources from clinical trials, scientists can hunt trends that may suggest what to study next. The database also will be available to brain researchers not affiliated with drug companies, to compare their own findings, and eventually will address other neurodegenerative diseases such as Parkinson's. Source: Associated Press/Yahoo! News 6/11/10 

Cancer Wins May be Bigger Than They Seem

Doctors reported gains against nearly every form of cancer at a conference that ended in June. However, the top achievements reported at the American Society of Clinical Oncology added an average of just two to six months of life. One pricey drug made headlines merely for delaying the time until ovarian cancer got worse. Progress has always been slow for cancer treatment. New therapies are tested on people who are so sick and out of options that any extension of life is considered a success. A cure is not usually possible. But some of the victories reported against breast and prostate cancer, leukemia, and the deadly skin cancer called melanoma may be larger than they appear. Newer drugs seem to be making a bigger difference for small, specific groups of patients, as companies develop treatments that more precisely target genes behind subtypes of cancer. Also, quicker answers are coming from smaller, focused studies; big gains have been seen from novel combinations; and comparison tests of long-used treatments could lead to new standards of care. Source: Associated Press/Yahoo! News 6/9/10 

FDA Warns Pfizer for Not Reporting Side Effects

The U.S. Food and Drug Administration (FDA) has warned Pfizer Inc for failing to quickly report serious and unexpected potential side effects from its drugs already on the market. In a 12-page warning letter to Pfizer Chief Executive Jeffrey Kindler, the FDA cited numerous examples involving some of the company's top-known brands, including impotence drug Viagra, cholesterol pill Lipitor, and seizure medicine Lyrica. The delays in reporting side effects date back as far as 2004 and have grown in recent years, according to the FDA's letter that was released by Pfizer in June. In a statement, Pfizer said it would work with the FDA to satisfy the agency "and to assure optimal surveillance and reporting of post-marketing adverse events." Source: Reuters/Yahoo! News 6/10/10 

U.S. Medical Staff Experimented on Terror Suspects

Medical personnel apparently experimented on terror detainees during CIA-led torture after the September 11 attacks, aiming to improve interrogation techniques, a human rights group said June 7. Physicians for Human Rights said it obtained public records showing health professionals worked under the supervision of the Central Intelligence Agency during interrogations of "war on terror" detainees after the 2001 attacks. The doctors and medical staff witnessed waterboarding, forced nudity, sleep deprivation, temperature extremes, and prolonged isolation, among other techniques. The experimentation and research "appear to have been performed to provide legal cover for torture," the group said. Although the use of cruel and inhuman treatment has been documented, the report said the new evidence shows health professionals actively participated in research and experimentation on detainees in U.S. custody during questioning. Source: Agence France Presse/Yahoo! News 6/7/10

Experimental Drugs Perform Well in Major Skin and Lung Cancer Studies

Researchers have scored the first big win against melanoma, the deadliest form of skin cancer. An experimental drug significantly improved survival in a major study of people with very advanced disease. The results, reported June 5 at a cancer conference, left doctors elated. The drug from Bristol-Myers Squibb Co., ipilimumab, works by helping the immune system fight tumors. The federal Food and Drug Administration has pledged a quick review, and doctors think the drug could be available by the end of this year. Doctors also reported at the conference that an experimental drug for lung cancer patients with a certain gene showed extraordinary promise in early testing. The drug, Pfizer Inc.'s crizotinib, targets a gene that promotes tumor growth and is found in about 4 percent of lung cancers, especially among younger, nonsmokers. Source: Associated Press/Yahoo! News 6/6/10

Antidepressant Cools Hot Flashes in Study

The antidepressant citalopram may help lessen the severity of menopausal hot flashes, at least in the short term, a new clinical trial suggests. The findings, from a seven-week study of 254 women, add to evidence that certain antidepressants can offer relief from severe hot flashes. Studies have found that some antidepressants in the class known as selective serotonin reuptake inhibitors (SSRIs) may help treat hot flashes; those drugs include paroxetine (Paxil), fluoxetine (Prozac), and venlafaxine (Effexor). However, citalopram (Celexa), another SSRI, has an advantage over some of those medications—namely, paroxetine and fluoxetine—in that it can be used by women on tamoxifen. Tamoxifen is a drug prescribed to treat and lower the risk of breast cancer, and hot flashes are a common side effect. Because paroxetine and fluoxetine inhibit an enzyme needed to metabolize tamoxifen, women on the drug cannot use those antidepressants for hot flashes. Source: Reuters/Yahoo! News 6/3/10

Prostate Cancer Vaccine Shows Few Side Effects

The newly approved therapeutic prostate cancer vaccine, Provenge, is safe and has few side effects, a new study finds. In April, the U.S. Food and Drug Administration approved the vaccine for use in men with advanced prostate cancer who had failed hormone therapy. "Provenge was approved based on both safety and clinical data," said lead researcher Dr. Simon J. Hall, chair of urology at Mount Sinai Medical Center in New York City. This safety data shows that there are very limited side effects. The advantage of the vaccine for patients with metastatic hormone-resistant prostate cancer is that it has fewer side effects than chemotherapy, which is the only other treatment option for these patients, Hall explained. In addition, Provenge has improved survival over chemotherapy. The average survival time for men given Provenge is 4.5 months, although some patients saw their lives extended by two to three years. Source: HealthDay/Yahoo! News 5/31/10

Extreme Medicine Gets Pentagon Push; Human Trials Rushed

Some of the most debilitating war injuries, from lost limbs to mangled muscle tissue to permanent burn scars, could soon benefit from cutting-edge regenerative procedures. Human clinical trials of the latest in extreme regenrative medicine—including bone-fusing cement and muscle-growing cell scaffolds—are being fast-tracked, thanks to an extra $12 million in funding from the Department of Defense. Researchers at the University of Pittsburgh are working on some of the most promising Pentagon-backed medical research projects. In April, Joint Chiefs of Staff Chairman Admiral Mike Mullen visited the university’s labs to see the science firsthand. Despite the looming threat of a shrinking Pentagon budget, he told them that “10 years doesn’t satisfy any of us,” where clinical trials were concerned. Source: Wired.com 5/26/10

Negative Research Often Spun to Look Good

Scientists are no strangers to spinning their research, a new study--presumably not spun--shows. More than half of 72 reports examined by French and British researchers had dressed up their conclusions to make it seem as if new treatments were beneficial, even though they weren't, according to the statistics in the report. For instance, one study concluded a cancer detection system worked, but couldn't back it up with actual results, said Dr. Isabelle Boutron, who worked on the study at the Universite Paris Descartes in France. Although she didn't examine the motives behind the spin, Boutron found several dubious strategies to misrepresent data. Researchers would try to focus on only positive parts of their analysis, or compare patients before and after they received a treatment, without noting how patients responded to placebo. "I'm sure most of the authors weren't cheating, they were just very enthusiastic about their results," Boutron said. Source: Reuters/Yahoo! News 5/25/10

Years of FDA Supervision Ordered for Genzyme

Genzyme Corp. will remain under federal oversight for the next seven to eight years as it works to fix quality-control problems that have bedeviled its Allston Landing plant for 15 months. The timetable was spelled out in a consent decree struck between Genzyme and the Food and Drug Administration (FDA). Under its terms, Genzyme will pay a previously disclosed $175 million federal fine, the first in its 29-year history. The agreement, filed with the U.S. District Court in Boston, is subject to court approval. Genzyme’s plant in Allston produces drugs to treat rare genetic disorders such as Gaucher, Fabry, and Pompe diseases. Last summer, the company had to suspend drug production after a virus was found at the plant. The temporary shutdown delayed shipments of enzyme replacement therapies Cerezyme for Gaucher disease and Fabrazyme for Fabry disease, frustrating patients and depressing sales. Source: Boston Globe 5/25/10

British Medical Council Bars Doctor Who Linked Vaccine With Autism

A doctor whose research and public statements caused widespread alarm that a common childhood vaccine could cause autism was banned on May 24 from practicing medicine in his native Britain for ethical lapses, including conducting invasive medical procedures on children that they did not need. The General Medical Council applied its most severe sanction against the doctor, Andrew Wakefield, 53, who abandoned his medical practice in Britain in 2004 as questions intensified about his research and set up a center to study childhood developmental disorders in Texas, despite not being licensed as a physician there. Wakefield said he would appeal the decision, which will take effect, unless suspended for legal reasons, within 28 days. Most scientific papers have failed to find any links between vaccines and autism. Source: New York Times 5/25/10

FDA Needs New Tools to Check Food, Drugs

The Food and Drug Administration (FDA) is stuck using crude tools to measure the benefits of food, drugs, and supplements and needs a whole new set of standards, a panel of experts said in May. Relying on so-called biomarkers is confusing the entire process of drug development, the public and doctors alike, they said. The FDA also needs to use the same strict standards for assessing health claims of food and supplements as it does for drugs, said the panel appointed by the Institute of Medicine, which advises the federal government. The committee recommended a new framework the FDA could use for judging studies that companies provide to support health and safety claims for their products. Source: Reuters/Yahoo! News 5/12/10

Companies, Regulators Team Up for Drug Testing

Regulators, scientists at 17 companies, and academic researchers have teamed up in a new international experiment to find agreed-upon ways to test new drugs in the lab and eventually in people. The hope is to find a few simple measures that will tell whether a drug is likely to cause serious side-effects, and then share them among drug developers and regulators, members of the team reported in the journal Nature Biotechnology on May 10. The group, called the Predictive Safety Testing Consortium, chose a panel of biomarkers--proteins that indicate disease or injury--to predict kidney injury from experimental drugs. The unprecedented collaboration includes the U.S. Food and Drug Administration and the European Medicines Agency, and the hope is to streamline the approval process as well. Source: Reuters/Yahoo! News 5/10/10 

Study of "Red Wine" Drug Suspended

A clinical trial testing an experimental GlaxoSmithKline drug that mimics a health-boosting compound found in red wine has been suspended due to safety issues. A company spokeswoman said on May 4 that the trial of SRT501 in patients with multiple myeloma had been suspended after a number of them developed a complication generally associated with the disease, which is a type of blood cancer. The mid-stage Phase II study, conducted in Britain and Denmark, was assessing the safety and tolerability of SRT501 with or without the established cancer drug Velcade from Takeda Pharmaceutical. SRT501 is a proprietary formulation of resveratrol, a substance found in grapes and in red wine. The clinical trial was started in March 2009 and was due to run until December 2010. It was suspended prematurely following "unexpected safety events," according to an update to the clinicaltrials.gov database on April 22. The suspension came after the enrollment of 24 patients out of a target of 61. Source: Reuters/Yahoo! News 5/4/10 

Drug Makers Enter Crucial Phase in Search for Alzheimer's Disease Treatments

After decades of research and multiple failed attempts to find a treatment, the pharmaceutical industry is entering a crucial phase in the search for a drug that can slow, or stop, the progression of Alzheimer's disease. Drug makers see huge moneymaking potential because of the aging population. There are an unprecedented five drugs being developed to modify the insidious disease that are in the Phase III final stage of U.S.-approved clinical trials. These "interventional drugs," if approved, would be markedly different from what's available, including Pfizer Inc.'s much-promoted Aricept, which temporarily treats symptoms of Alzheimer's. Exactly when one of these experimental treatments might be submitted to the U.S. Food and Drug Administration for approval is unclear, and there is no guarantee they will reach consumers. However, researchers and some Wall Street analysts are speculating that a disease-modifying product could be on the U.S. market within five years. Source: Los Angeles Times 5/4/10 

Provenge Approved for Advanced Prostate Cancer

Provenge (sipuleucel-T), a therapy designed to boost the immune system's ability to combat prostate cancer, has been approved by the U.S. Food and Drug Administration (FDA). The therapy is produced by obtaining the person's own immune cells, exposing them to a protein found in most forms of prostate cancer, then returning them to the donor. The therapy is administered three times, each approximately two weeks apart, the agency said in a news release. Provenge has been approved for men with no symptoms or minimal symptoms of the disease, which has spread to other parts of the body and is resistant to standard hormone therapy. In a study of 512 men, those who took Provenge had average survival of 25.8 months, compared to 21.7 months among those who did not get the treatment, the FDA said. Provenge is produced by Seattle-based Dendreon Corp. Source: HealthDay/Yahoo! News 4/29/10  See also Washington Post 

Promise Seen in Drug for Retardation Syndrome

An experimental drug succeeded in a small clinical trial in bringing about what the researchers called substantial improvements in the behaviors associated with retardation and autism in people with fragile X syndrome, the most common inherited cause of these mental disabilities. The surprising results, disclosed in an interview in late April by Novartis, the Swiss pharmaceutical giant that makes the drug, grew out of three decades of painstaking genetic research, leaps in the understanding of how the brain works, the advocacy of families who refused to give up, and a chance meeting between two scientists who mistakenly showed up at the same conference. If authenticated in further, larger trials, the results could also become a landmark in the field of autism research, since scientists speculated that the drug may help some patients with autism not caused by fragile X, perhaps becoming the first medicine to address autism’s core symptoms. Source: New York Times 4/29/10 

Editorial: Faltering Cancer Trials

The nation's most important system for judging the clinical effectiveness of cancer treatments is approaching "a state of crisis." That is the disturbing verdict of experts assembled by the National Academy of Sciences to review the performance of clinical trials sponsored by the National Cancer Institute (NCI). Unless the shortcomings are remedied, some of President Obama's ambitious healthcare reforms will be jeopardized and his audacious goal of finding "a cure for cancer in our time" will have almost no chance at all. The most shocking deficiency highlighted by the report, issued by the academy's Institute of Medicine, is that about 40 percent of all advanced clinical trials sponsored by NCI are never completed. That is an incredible waste of effort and money, and a huge obstacle at a time when researchers are developing promising new therapies that must be rigorously tested. ...[A] series of reviews in recent years found that the testing operation is mired in bureaucracy and poorly coordinated. A typical trial must navigate past dozens of overlapping reviews by different boards and agencies that must approve the original concept for the trial and then the protocol that will govern how it is conducted before the investigators can start enrolling any patients. The average time between developing the concept for a study and getting it started is about 2.5 years. The longer a study takes to get started, the more likely it is to become scientifically out of date, and the less likely it is that doctors or patients will want to participate. Other factors, including failure to pay investigators and their institutions the full costs of a trial, can also impede enrollment. And if not enough patients are enrolled, the study lacks the statistical power to generate meaningful results and cannot be completed. ...Repairing the clinical trials system is critical not only for healthcare reform, but for the health of millions of Americans. Source: New York Times 4/24/10 

Genzyme Drug Shortage Leaves Users Feeling Betrayed

Because of a drug factory shutdown, Jeannine Lipez of Lock Haven, Pa., says she can no longer even walk across the street without getting spasms in her left leg and will probably need an operation to replace an artery. Carol Fink of Yountville, Calif., says lack of the medicine she needed left her constantly in pain, sapped her energy, and made her thinking fuzzy. For Dr. William Schubert, an obstetrician and gynecologist in Pocatello, Idaho, the factory shutdown may have contributed to an even worse outcome. Even as his wife and doctor raced to find doses of the drug he needed, Dr. Schubert’s heart deteriorated rapidly. He died on March 6, at the age of 63. These people, and thousands more in this country and abroad, have been hard hit by a shortage of drugs made by the biotechnology company Genzyme to treat two rare inherited diseases. The supply problems, which have dragged on since last summer, have frayed the close relationship—unusual in the pharmaceutical industry—that Genzyme had carefully built with the several thousand users of its high-price medicines over the last two decades. Source: New York Times 4/15/10

Research Offers Promise for Diabetics

Boston researchers have made a major step toward the development of an artificial pancreas that overcomes the bugaboo of most previous such attempts—dangerously low blood sugar caused by injection of too much insulin. Their experimental device secretes two hormones normally produced by the pancreas—insulin and its counterbalancing hormone, called glucagon—and has been shown to control blood sugar levels in about a dozen people for at least 24 hours, they reported in April. The team is now planning longer trials as it gears up for what it hopes will be approval by the Food and Drug Administration in as little as seven years. What the Boston team has invented is a computer algorithm that responds to changes in blood sugar and computes how much insulin or glucagon to inject. Source: Los Angeles Times 4/15/10

Study Sees a Slant in Articles on Drug

A new analysis of reviews and articles about the controversial diabetes drug Avandia has found that experts who were paid by its manufacturer have been significantly more likely than others to draw positive conclusions about the drug’s safety and efficacy. Researchers reviewed 202 articles by 180 authors who wrote about Avandia and the risk of heart attack, and had independent reviewers with no conflicts of interest grade each article as favorable, neutral, or unfavorable, based on the authors’ positions on an association between Avandia and heart attacks and on their recommendations for continuing or ending its use. The study was published online on March 18 in the journal BMJ. Often, authors with favorable opinions of the drug were paid both by Avandia’s maker, GlaxoSmithKline, and by its competitors. Of those who offered favorable views, 87 percent had potential conflicts with GlaxoSmithKline. Among authors who had unfavorable opinions, only 20 percent had received money from GlaxoSmithKline. Source: New York Times 4/12/10

Lithium Does Not Slow Lou Gehrig's Disease

Lithium doesn't help patients with ALS, or Lou Gehrig's disease, contrary to previous study results, new research says. Results from a small study published two years ago suggested the drug, often used for depression, could slow the fatal neurological disorder. Many ALS sufferers and their families rushed to try it, spearheading a patient-led effort to test lithium without doctors. In the first trial to scientifically assess whether lithium works for Lou Gehrig's disease, doctors found it had no effect, and stopped the study early because it seemed futile. The results were published online April 6 in the medical journal, Lancet Neurology. American and Canadian doctors enrolled 84 patients with Lou Gehrig's disease into their study across both countries. About half the patients got lithium and riluzole, the standard drug used for the disease. The other half got riluzole plus placebo pills. After nearly six months, researchers saw no difference: 22 of the 40 patients in the lithium group had gotten worse versus 20 of 44 patients in the placebo group. Source: Associated Press/Yahoo! News 4/6/10

In the Search for Cancer Drugs, Mice Get New Role

Dr. Pier Paolo Pandolfi has begun an experiment that could radically change the way new cancer drugs are tested: Not far from where his colleagues give trial treatments to patients at Beth Israel Deaconess Medical Center in Boston, he's giving the same regimen to mice. Normally, mice are test subjects for drugs long before they make it into the clinic. Animal models of disease are important to better understand cancer and to determine which of myriad compounds are safe and promising enough to be tried in people. But in the new work, funded with a $4.2 million economic stimulus grant from the National Cancer Institute, mice are receiving treatment in tandem with humans, in the hope that what is learned at the bedside can be integrated with results from the lab bench to speed up and streamline the development of cancer drugs. The novel strategy is an attempt to address a bottleneck that has emerged in the cancer drug pipeline because of a proliferation of drugs in development. Source: Boston Globe 4/5/10

Study Finds Possible Heart Risk with Prostate Drug

Full results of a big study testing a drug for preventing prostate cancer show a higher risk of heart failure, a surprise finding that could dampen enthusiasm for expanding its use. GlaxoSmithKline PLC has asked the federal Food and Drug Administration to approve its drug Avodart as a cancer preventive for men at higher-than-normal risk of the disease. The drug is already sold for urinary problems, and no heart failure risks have been seen with that use, doctors say. Results of a study testing it as a cancer preventive were given at a medical conference last year, but the heart risk information had not been analyzed at that time, so only the main results of the study were presented. Full results were published in April in the New England Journal of Medicine. The study involved 6,700 men with high scores from PSA blood tests but no sign of cancer on biopsy. They were given Avodart or dummy pills and new biopsies four years later. Prostate cancer was found in 25 percent of those on dummy pills and 20 percent of those on Avodart. That's the same risk reduction as other studies have found from a similar drug, finasteride, sold in generic form and as Proscar, made by Merck & Co. Inc. Studies did not find an increased risk of heart failure with Proscar. In the Avodart study, heart failure developed in 30 men on the drug versus only 16 on dummy pills. Source: Associated Press/Yahoo! News 4/1/10

Recalls Put Company in Regulators' Crosshairs

Stepped-up government scrutiny of Boston Scientific Corp. stems from heightened concern over medical safety and disappointment that the company made new missteps after resolving previous problems with the Food and Drug Administration (FDA), analysts said in late March. The medical device maker, which has been working to settle patent suits and federal investigations dating back years, recently was notified of fresh investigations begun by the Department of Justice and the Securities Exchange Commission into problems that forced it to recall implantable heart defibrillators. While the scope of the new inquiries could not be determined, analysts said federal investigators most likely are focusing on whether investors were blindsided by Boston Scientific's failure to get required FDA approval for changes in its manufacturing processes for the defibrillators. Source: Boston Globe 3/31/10

Regulators Reject Alertness Pill to Overcome Jet Lag

Air travelers for now may have to rely on coffee instead of a wonder drug to help them function in new time zones. Cephalon said on March 29 that the Food and Drug Administration (FDA) had declined to approve use of its alertness drug, Nuvigil, to treat jet lag. Had the drug been approved, it would have been the first prescription drug specifically designated for jet lag. Nuvigil is a slightly longer-lasting form of Cephalon’s biggest seller, Provigil. Both drugs are approved to treat the excessive sleepiness associated with narcolepsy, sleep apnea, and shift work. In a statement, Cephalon did not elaborate about why the FDA had rejected its application, other than to say that the agency had questioned the “robustness” of the results of questionnaires that asked participants in the company’s clinical trial how sleepy they felt after taking the drug. Source: New York Times 3/29/10

Genzyme is Facing FDA Fines

Federal regulators are preparing to fine Genzyme Corp. and step up supervision of the company’s troubled Allston drug manufacturing plant after a string of embarrassing quality problems at the facility. The firm said that the Food and Drug Administration (FDA) notified it on March 23 that the agency plans to take “enforcement action’’ against the company. The action comes four months after the company said some of the drugs processed at the plant were contaminated by bits of steel, rubber, and fiber. Last June, Genzyme temporarily suspended much of its Allston operations after it detected a virus in the plant and was forced to launch a massive decontamination effort. Source: Boston Globe 3/25/10

Opinion: FDA's New Policies Threaten Innovation

Writing in the Los Angeles Times, Henry I. Miller, a physician, molecular biologist, fellow at Stanford University's Hoover Institution, and former Food and Drug Administration (FDA) official, says that zeal has replaced science and common sense at the agency. Among other unwelcome developments, Miller notes that the FDA reversed a sound policy that required prior legal review of warning letters sent to companies; increased the amounts of data that will need to be obtained and submitted to regulators for medical devices to an extent that threatens innovation in the industry; and increased the kinds and amounts of "user fees" that companies must pay just to get the FDA to review their applications, which he terms nothing more than a discriminatory tax on pharmaceutical companies that will ultimately be passed along to patients. Source: Los Angeles Times 3/25/10

Medical Breakthroughs Sprang from One Woman’s Cells

Sixty years after Henrietta Lacks died of cervical cancer, her cells live on in laboratories around the globe. Collected by Johns Hopkins researchers as she was being treated, the cells grew incessantly--and they've since helped scientists make blockbuster medical advances, including cancer treatments and the polio vaccine. Decades passed before anyone told Lacks' relatives of her enduring gift to modern science. And though the advances that have come from her cells are worth millions, her family never received a cent. Lacks' story, illuminated in the book The Immortal Life of Henrietta Lacks by Rebecca Skloot, raises important and unresolved issues about the bioethics of modern tissue research, experts say: Are consent rules strong enough? Are people entitled to any compensation if pieces of their bodies turn out to be worthy of a medical advancement? Source: Los Angeles Times 3/23/10

FDA Panel Mostly Backs Much-Debated Knee Device

Federal health experts said on March 23 that a device for repairing knees that was subject to a contentious three-year review by the Food and Drug Administration (FDA) is likely safe and effective, despite major shortcomings with company studies. The findings by the FDA's independent panel of orthopedic experts came despite the government's highly critical review of Regen Biologic's device published ahead of the meeting. The head of FDA's medical device division said the agency would make a decision on the device in coming weeks. The agency's options include leaving it on the market as is, pulling it, or it could call for additional testing. The meeting was a rare case of the FDA reassessing the safety of a device it previously approved for patients. Source: Associated Press/Washington Post 3/23/10

Drug Trials Have Excluded Gays

Clinical trials have all sorts of scientifically valid criteria to determine who can try out an experimental medicine--such as age, gender, and whether the person smokes or has a certain disease. In at least a few dozen cases, however, trials have excluded participants based on a criterion that has raised concern: sexual orientation. Researchers at Fox Chase Cancer Center identified 37 recent trials that were limited to heterosexual patients. The analysis was published in a letter in the New England Journal of Medicine. The trials in question generally had something to do with sexual function, sex drive, or relationships. There was no indication that people running the trials intended to discriminate against gays or lesbians, one author said. Source: Philadelphia Inquirer 3/22/10 For more information, see the ACRP Wire.

Many Avandia Defenders Have Drug Company Ties

Virtually all of the experts who wrote favorably about GlaxoSmithKline Plc's troubled diabetes drug Avandia had financial ties to drug makers, a finding that shows the need for reform of such relationships, U.S. researchers said in March. A team at the Mayo Clinic in Rochester, Minn., pored through more than 200 scientific studies and commentaries that offered positive opinions about the drug after a May 2007 study suggested Avandia significantly increased the risk of heart attacks. They found that 94 percent of the authors who defended the drug, known generically as rosiglitazone, had ties to drug companies, and nearly half had financial ties that presented a conflict of interest. Source: Reuters/Yahoo! News 3/18/10

Breast Cancer Study Aims to Speed Drugs, Cooperation

Researchers launched a unique collaboration in March aimed at getting cancer drugs to the market more quickly in which three companies (Abbott Laboratories, Amgen, and Pfizer Inc) will cooperate with the U.S. government and nonprofit groups to test five experimental breast cancer drugs. The five-year, $26 million study called Investigation of Serial Studies to Predict your Therapeutic Response with Imaging and Molecular Analysis, or I-SPY2, will aim to use DNA to match the best drug to each patient and to more quickly toss out approaches that do not work or are too toxic. Unusually, the companies agreed to share information on using genes to predict how well a patient will respond as part of the Biomarkers Consortium, which includes the U.S. Food and Drug Administration and the National Institutes of Health. Source: Reuters/Yahoo! News 3/17/10

In Cancer Fight, Teenagers Don’t Fit In

Experts say that since teenagers tend not to ask adults for help or confide about embarrassing physical changes, they are likely to receive their cancer diagnoses much later in the course of their illness than younger children. This usually means they will require more aggressive and protracted treatments that can lead to lifelong side effects. While overall survival rates are as high as 70 to 80 percent, depending on the type of cancer, teenagers have not benefited from the huge advances in survival made by younger children and much older adults in recent decades. They are also far less likely to participate in clinical trials, which offer the most up-to-date therapies: Fewer than one in five adolescents with cancer are treated in a clinical trial, according to some estimates, compared to well over half of younger children. Source: New York Times 3/15/10

Few U.S. Studies Compare One Drug to Another

Comparing medical treatments to find the best and the cheapest may be a pillar of U.S. healthcare reform efforts, but very little such research is being done, according to a report published on March 9. Most of the so-called comparative effectiveness research is done at academic institutions or by other noncommercial enterprises, and less than 20 percent of such studies examine the safety of treatments, researchers reported in the Journal of the American Medical Association. "Most of the comparative effectiveness studies we reviewed simply tested whether medication 'x' is better than medication 'y,' rather than addressing fundamental questions such as: How can we use this medication more effectively? When is this medication better than surgery? Which among two effective approaches is the safest?" said Dr. Danny McCormick of Harvard Medical School in Boston, who led the study. Source: Reuters/Yahoo! News 3/9/10 For more information from the ACRP Wire, click here.

Biogen, Roche Halt Trial of Drug

Biogen Idec Inc. and its Swiss partner Roche Holding AG in March said they halted clinical trials of a new drug being tested on patients with rheumatoid arthritis and lupus after an outside monitoring panel warned its safety risks outweighed the benefits, resulting in an unspecified number of deaths. The drug, called ocrelizumab, caused "serious and opportunistic infections, some of which were fatal" in some of the 2,400 patients being treated in trials in more than 30 countries, according to the independent data and safety monitoring board for the two diseases. Neither Roche nor Biogen Idec would say how many patients have died from infections related to the experimental drug. Source: Boston Globe 3/9/10

Roche's Avastin Fails in Stomach Cancer Trial

Roche Holding's cancer drug Avastin missed its main target in a late-stage trial when used with chemotherapy to treat patients with advanced stomach cancer, the Swiss drug maker said on February 23. Avastin failed to meet its target of extending overall survival in patients treated with the drug in combination with chemotherapy when compared with the same chemotherapy treatment plus placebo in the trial, Roche said. Roche, the world's largest maker of cancer drugs, said no new safety issues were observed in the AVAGAST Phase III trial, and that Avastin's broad development program in other tumor types would continue. Source: Reuters/Yahoo! News 2/23/10

Massachusetts Doctor Pleads Guilty to Research Fraud

A doctor accused of faking research for a dozen years in published studies that suggested after-surgery benefits from painkillers including Vioxx and Celebrex pleaded guilty on February 22 to one count of federal healthcare fraud. An attorney for Dr. Scott Reuben said the anesthesiologist will have to repay $361,932 in research grants and forfeit assets worth at least $50,000 as penalty for his conduct following a plea hearing in U.S. District Court. Prosecutors alleged the former chief of acute pain at Baystate Medical Center in Springfield sought and received research grants from pharmaceutical companies but never performed the studies. They said he fabricated patient data and submitted information to anesthesiology journals that unwittingly published it. Reuben's attorneys said that Reuben was suffering from a serious a bipolar disorder that was only accurately diagnosed in 2008 and had no financial motive when he committed the offenses. Source: Associated Press/Washington Post 2/22/10

FDA: No New Conclusions on GlaxoSmithKline's Avandia Yet

The U.S. Food and Drug Administration (FDA) is reviewing data on possible heart risks with GlaxoSmithKline Plc's diabetes drug Avandia, but has not reached any new conclusions, the agency said on February 22. The FDA will hold a public meeting in July 2010 to discuss the risks and benefits of the drug, but said in the meantime doctors and patients should continue to use Avandia as directed. Democratic Representative Rosa DeLauro, in a statement posted on her website, urged the FDA to "remove Avandia from the market until a truly independent, science-based advisory panel can evaluate the safety and effectiveness of the drug." Avandia came back in the spotlight after two U.S. senators on February 20 released a report on the drug and internal FDA documents. The documents included a 2008 memo from two FDA drug safety reviewers who recommended pulling the drug from the U.S. market after they concluded it was more dangerous to the heart than the rival drug Actos by Takeda Pharmaceutical Co Ltd. Source: Reuters/Yahoo! News 2/22/10

Target Cancer: A Roller Coaster Chase for a Cure

Dozens of “targeted” drugs are emerging from the laboratory, rooted in decades of research and backed by unprecedented investment by pharmaceutical companies, which stand to profit from drugs that prolong life even by weeks. But putting them to their truest test falls to a small band of doctors committed to running experimental drug trials for patients they have no other way to heal. At a time when cancer still kills one in four Americans, it is a job that requires as much hubris as heart. To chronicle the trial of the drug known as PLX4032 is to ride a roller coaster of breakthroughs and setbacks at what many oncologists see as a watershed moment in understanding the genetic changes that cause cancer. A three-part series of articles in the New York Times on PLX4032 research starts here (2/22/10) and continues here (2/23/10) and here (2/24/10).

Boston Scientific Plans Job Cuts

Medical device maker Boston Scientific Corp., still grappling with the aftermath of a giant acquisition and big legal settlements, in February said it will eliminate up to 1,300 jobs worldwide as part of a restructuring move unveiled by the company’s new chief executive. The job cuts, disclosed as the company posted a $1.1 billion fourth-quarter loss, came nine days after Boston Scientific agreed to pay more than $1.7 billion to competitor Johnson & Johnson to resolve three longstanding patent disputes involving competing heart stents, the largest legal settlement in the 30-year history of Boston Scientific. Boston Scientific said it would cut 1,000 to 1,300 jobs, a reduction of 8 to 10 percent, in an effort to reduce annual expenses by $200 million to $250 million over the next two years. Source: Boston Globe 2/11/10 

FDA Budget Draws Cries of "Not Enough"

The Food and Drug Administration (FDA) was sitting pretty in early February, winning a significant budget increase while many other federal agencies faced the prospect of cut or frozen funding as the Obama administration confronts a 13-figure deficit. However, a coalition of public interest advocates, patient groups and healthcare industry interests regulated by the FDA had a swift response: It's not enough. "We are disappointed in the president's budget request and...will seek to increase" the budget, said Steven Grossman, deputy executive director of Alliance for a Stronger FDA. The group lists seven former FDA commissioners and many of the largest and most influential consumer, food, and pharmaceutical trade groups among its 180 members. Source: Los Angeles Times 2/11/10 

Opinion: The Damage of the Anti-Vaccination Movement

Writing in the Los Angeles Times, Michael Fumento, director of the Independent Journalism Project, notes: The doctor who launched the modern anti-vaccine movement acted "dishonestly and irresponsibly," Britain's General Medical Council has ruled. But fear not. Dr. Andrew Wakefield is still a hero to his many acolytes. And others, with curious credentials, fight on to terrify parents into not getting their children inoculated. ..."One person's research set us back a decade, and we're just now recovering from that," Mark Sawyer, a pediatrician and infectious disease specialist at Radey Children's Hospital in San Diego, told me in an interview. But are we recovering? Anti-vaccination groups have popped up like toadstools after rain (there are more than 180 on the Web), while older ones...were reinvigorated. Source: Los Angeles Times 2/5/10 

FDA Aims at Doctors’ Drug Pitches

The Food and Drug Administration (FDA) has cracked down on one of the most widely quoted cosmetic doctors, sending shudders through the ranks of opinion leaders in fashion publishing and vanity medicine. The agency recently sent a warning letter to Dr. Leslie Baumann, a well-known dermatologist and clinical researcher, citing the doctor for expressing premature enthusiasm in the media about Dysport, an injectable antiwrinkle drug the FDA had not yet approved. This is believed to be the first time the agency has warned an individual investigator—a medical researcher who oversees a clinical trial—for apparently promoting an unapproved drug. Baumann was an investigator on a clinical trial for Dysport and promoted it well before the drug’s approval last April. Source: New York Times 2/1/10

Amphastar Pharmaceuticals Files New Appeal in FDA Conflict-of-Interest Case

A new appeal in a conflict-of-interest controversy involving the Food and Drug Administration's (FDA's) handling of the deadly heparin contamination crisis of 2008 has shed more light on the convoluted and costly maneuvering that can break out when billions of dollars in profits are at stake. The latest round began when Amphastar Pharmaceuticals Inc. said it would appeal the FDA's rejection of a complaint. The privately held drug maker alleged that Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, had a conflict of interest. Amphastar contended that Woodcock's conflict should have barred her from taking part in FDA deliberations over applications by Amphastar and a rival drug maker to market generic versions of heparin, a widely used blood thinner. What appears to worry Amphastar—and raises a corner of the curtain on drug industry's arcane maneuvering—is not just that the FDA, perhaps influenced by Woodcock, might oppose the company's application to market generic heparin, it's that Amphastar, which filed its application two years earlier than the other firm, might see both applications approved at the same time—meaning that Amphastar would not get the head start it says it deserves. Source: Los Angeles Times 1/21/10

MS Pills Show Promise and Risk

Tests of the first two oral drugs developed for treating multiple sclerosis (MS) show that both cut the frequency of relapses and may slow progression of the disease, but with side effects that could pose a tough decision for patients. The new studies tested two types of pills. Cladribine, made by Merck Serono, is already sold to treat a rare blood cancer. For MS, it would be taken eight to 10 days a year. Fingolimod is a daily MS pill being developed by Novartis. The research found that patients on the pills were about half as likely to suffer relapses of symptoms as those who took dummy pills or a commonly prescribed shot for MS. However, they also found both drugs significantly lowered immune defenses that allowed latent herpes viruses to rage in some patients—in one study, two people died of unchecked herpes infections. Treatments on the market that have had success in reducing recurrence of MS symptoms involve daily or regular injections, so a pill is appealing. Source: Associated Press/Yahoo! News 1/20/10

Researchers Find Study of Medical Marijuana Discouraged

Despite the Obama administration’s tacit support of more liberal state medical marijuana laws, the federal government still discourages research into the medicinal uses of smoked marijuana. That may be one reason that—even though some patients swear by it—there is no good scientific evidence that legalizing marijuana’s use provides any benefits over current therapies. Lyle E. Craker, a professor of plant sciences at the University of Massachusetts, has been trying to get permission from federal authorities for nearly nine years to grow a supply of the plant that he could study and provide to researchers for clinical trials, but the Drug Enforcement Administration has refused. Marijuana is the only major drug for which the federal government controls the only legal research supply and for which the government requires a special scientific review. Source: New York Times 1/19/10

Benefits of Testosterone, HGH on Aging Unclear

In a quest to look younger, be healthier, and feel more vital later in life, increasing numbers of men are turning to testosterone and human growth hormone (HGH). Use of both hormones is controversial. It is unclear whether the issues associated with aging in men—decreased sex drive, less energy, cognitive impairment, and reduced muscle mass—are due to a decrease in testosterone or to other changes that come with aging. Long-term studies on effectiveness of the therapy are lacking, and the few small, shorter-term trials that exist have produced mixed results. One of the largest testosterone trials to date is being planned to take place at 12 medical centers across the country. Eight hundred men with low testosterone will receive either testosterone or a placebo for a year and be monitored for changes in vitality, sexual and cognitive function, anemia, bone density, and cardiovascular health. Meanwhile, HGH has not been approved by the Food and Drug Administration for treating elderly individuals experiencing physical decline, but it is being used "off label" for this purpose. Again, research on the safety and benefits of HGH is mixed, and the National Institute on Aging says there is no conclusive evidence that the hormone can reduce the physical decline that comes with age. Source: Los Angeles Times 1/18/10

True Story that Unfolded at Genzyme Inspired Film "Extraordinary Measures"

Harrison Ford is the brilliant scientist; Brendan Fraser, the father desperately trying to save his children from an incurable disease; and Zymagen is the biotechnology company that swoops in at the right moment. The Hollywood movie "Extraordinary Measures" is based loosely on a true story that took place more than a decade ago at Genzyme Corp., but like many “inspired by’’ films, this tale has a back story not found on the silver screen. John Crowley, a real-life Harvard graduate, built up a company, Novazyme Pharmaceuticals, to create a treatment for Pompe disease after two of his children were diagnosed with the rare enzyme deficiency. Genzyme, which acquired Novazyme and its drug research in 2001, isn’t mentioned in the film by name, but the film does include the fictional Zymagen. Genzyme later successfully created Myozyme, a treatment for Pompe, using its own in-house research. The film takes liberties with the way actual events unfolded, and many names were changed as characters were combined and timelines condensed. Source: Boston Globe 1/16/10

Could Vioxx Cousin Prevent Religious Fast Headache?

Every year, millions of observant Jews fast on their holiest day, Yom Kippur, and millions of Muslims fast for the month of Ramadan, and every year, as many as 40 percent of those who fast develop serious headaches. Now, a team of researchers in Israel, reporting in the journal Headache, thinks it has a solution in the form of a cousin of Vioxx (rofecoxib) called etoricoxib (Arcoxia). Researchers recruited more than 200 volunteers before Yom Kippur in October 2008. Just before the holiday, half of them took etoricoxib, and half were given placebo pills. Among the 195 study participants who responded to a survey after the holiday, about 36 percent who took etoricoxib developed headaches, compared to about 68 percent who took the placebo. Those who took etoricoxib also had less severe headaches, and they had an easier time fasting. Source: Reuters/Yahoo! News 1/14/10

Life Science Firms Pitch Optimism

Biotechnology, pharmaceutical, and medical device company leaders from around the world converged in San Francisco for the 28th annual J.P. Morgan Healthcare Conference in January to tell their stories—and to try to raise money--at the life sciences industry’s leading investment forum. This year’s event drew more than 6,500 bankers, analysts, and other industry insiders. Clinical trials have yielded encouraging data over the past year in areas ranging from cancer and diabetes to virology and immunology, and while the window for initial public offerings has been closed, companies have been raising more money in follow-on offerings than any time in the past decade—about $6 billion in 2009. Source: Boston Globe 1/14/10

Clinical Trial Chiefs More Likely Tied to Industry

Cancer researchers who have the greatest ability to influence research are also the researchers with the greatest financial ties to the pharmaceutical and biotechnology industries, according to a report released in January. In a study, researchers found that cancer researchers who design clinical trials, analyze or interpret the data, or play other key scientific roles are four times more likely to have financial ties to industry than their counterparts who have lesser roles in these studies, such as recruiting study subjects or collecting the data. The findings stem from a look back at 235 drug trials published between January 2006 and June 2007 in the Journal of Clinical Oncology. Sixty-four percent of the authors—1,881 of 2,927—said they performed at least one key role in the study and 842 (29 percent) reported at least one financial tie. This relationship was present among both industry-sponsored and non-industry-sponsored studies, although it was stronger for industry-sponsored trials. Source: Reuters 1/11/10

Grant Money Could Speed Stem Cell Cures

Dr. Karen Aboody estimates that she has cured several hundred mice of a cancer of the central nervous system called neuroblastoma using a stem cell treatment. To hear Aboody tell it, that was the easy part. "People are curing mice right and left," said the City of Hope neuroscientist. The real challenge is convincing the Food and Drug Administration (FDA) to let her try this on people with brain tumors. Reams of safety data must be amassed to satisfy the FDA. Scientists struggle to navigate all that red tape. Many don't even try. Now the California Institute for Regenerative Medicine has stepped in--with an $18 million grant financed by state taxpayers. Aboody's windfall is just one manifestation of the state agency's changing mission, galvanized by the hiring in 2008 of a director with a track record of moving discoveries from lab to clinic. The institute is moving to fill a void known as the "valley of death"—a point at which projects are typically too commercial to vie for federal funds, yet too risky to entice private investors. Source: Los Angeles Times 1/10/10

A Drug’s Second Act: Battling Jet Lag

It seemed like the offer of a lifetime—earn $2,500 by flying to France aboard a private luxury jet, but as the fine print made clear, there would be no Eiffel Tower or chateaux, no foie gras or Bordeaux. Travelers were confined to a laboratory in either Toulouse or Rouffach with electrodes attached to their heads, testing whether a drug could keep their jet-lagged bodies awake. That drug, Nuvigil from Cephalon, could become the first medicine specifically approved by the Food and Drug Administration to combat jet lag. The clinical trial involved 427 healthy adults who were flown from the East Coast of the U.S., departing in the early evening and arriving in France around 7 a.m. local time. Each was given either Nuvigil or a placebo pill after arrival and on the next two mornings. At 10 a.m., noon, 2 p.m., and 4 p.m. each of those days, participants went to bed in a dark, quiet room. As soon as they fell asleep, as measured by brain waves, they were prodded awake. Those who got the placebo took an average of only 3.4 minutes to nod off on the first day, 6.2 minutes on the second day, and 8.2 minutes on the third, but those who got the highest dose of Nuvigil stayed awake for an average of 9.7 minutes the first day, 13.8 minutes the second day, and 14.8 minutes the third. Source: New York Times 1/7/10

FDA Drug Approvals Mostly Flat in 2009

New drugs cleared by the Food and Drug Administration (FDA) last year kept pace with 2008, suggesting a much-touted push for drug safety has not slowed down approvals. The FDA's new leaders did step up early warnings about potential drug safety issues and citations to companies that violate safety regulations. Drug approvals inched higher to 26 first-of-a-kind prescription medicines last year, from 25 in 2008, according to figures from Washington Analysis, an investment research group. The 2009 totals suggest a moderate approach to regulation from FDA, despite drug industry concerns that recently appointed Obama administration officials would result in fewer drug approvals. Source: Associated Press/Yahoo! News 1/5/10

Study: Ginkgo Does Not Slow Cognitive Decline of Aging

For years, practitioners of alternative medicine have been touting the benefits of ginkgo, especially for maintaining brain health, but a new study finds that the centuries-old nostrum does little to slow the cognitive decline of aging. Researchers at six universities across the U.S. report that elderly people taking ginkgo supplements showed no notable differences in scores on brain function tests from people taking placebo pills. The study is the largest and, with a mean follow-up of six years, also the longest to investigate the effects of ginkgo on the brain. The more than 3,000 elderly volunteers between the ages of 72 and 96 were tested repeatedly using crude screening measures as well as more detailed, computerized assessments of their mental function. Source: Time/Yahoo! News 12/30/09

Studies Spotlight Problems in FDA Device Approvals

Two new studies find shortfalls in the Food and Drug Administration's (FDA's) approval process for heart devices such as pacemakers and stents. Safety targets often weren't clearly spelled out in the research submitted by device makers and important patient information was missing, according to one study conducted by researchers from the FDA and Boston's Beth Israel Deaconess Medical Center. A separate analysis by researchers at the University of California, San Francisco, found heart devices frequently got the FDA's blessing based on research done outside the United States in small groups of patients. Many device studies lacked standards most scientists expect: randomization and a clear goal. Source: Associated Press/Yahoo! News 12/29/09

Tool in Cystic Fibrosis Fight: A Registry

In the 1950s, children with cystic fibrosis usually died before they reached kindergarten age, but today, many people with the disorder are living well into their 30s and beyond. These patients owe their lives, at least in part, to a carefully updated nationwide collection of personal profiles called a patient registry. The Cystic Fibrosis Foundation, in Bethesda, Md., manages the electronic registry, which tracks treatments, results, and health status on patients from more than 100 centers around the country. Researchers say that by identifying the most effective treatments for each patient, it has played a crucial part in doubling the average life expectancy of the estimated 30,000 cystic fibrosis patients in the United States. Now the idea is being widely copied. Advocates for patients with an array of intractable diseases like Parkinson’s, lupus, and juvenile diabetes are among the latest to begin developing registries, and the National Institutes of Health is promoting a linked network of registries for rare diseases with fewer than 200,000 patients. Source: New York Times 12/22/09

U.S. Rolls Back AIDS Drug Prevention Trial in Botswana

U.S. officials said on December 17 that they will give up on a trial in Botswana that was trying to show whether it is possible to prevent HIV infections by taking a daily pill because too few people are being infected. There are also problems keeping track of people enrolled in the trial, so it will be adjusted to show instead how well people can stick to the routine, the team at the U.S. Centers for Disease Control and Prevention said. The trial of 1,200 people was testing a daily pill that combined two HIV drugs. It was using Gilead Sciences Inc's Truvada, a combination of two drugs called tenofovir and emtricitabine. The idea is that a daily low dose of the drugs, which interfere with the ability of the virus to replicate, could also lower the risk of infection. Similar trials are under way in the United States, South Africa, Thailand, Brazil, Peru, Kenya, Uganda, and elsewhere. Source: Reuters/Yahoo! News 12/17/09

Drug Makers Step Up Lobbying On Healthcare Bill

The U.S. Senate has resumed debate on a popular proposal to allow U.S. citizens to buy cheaper drugs from foreign countries, which led to a last-minute lobbying push by drug makers earlier in December and bogged down negotiations over a heathcare reform bill. The fight over the imported drugs proposal poses a particularly difficult political challenge for President Obama, who cosponsored a similar bill when he was in Congress and who included funding for the idea in his first budget. The amendment would allow pharmacies and wholesalers to import U.S.-approved medications from Canada, Europe, Australia, New Zealand, and Japan, where drug costs are much lower because of price controls. However, the pharmaceutical industry has responded with a fierce lobbying campaign aimed at killing the proposal, focusing on Democratic senators from states with large drug and research sectors. Source: Washington Post 12/14/09

A Prescription for Snooping

When your doctor writes you a prescription, that's just between you, your doctor, and maybe your health insurance company--right? Wrong. As things stand now, the pharmaceutical companies that make those prescription drugs are looking over the doctor's shoulder to keep track of how many prescriptions for each drug the physician is writing. By obtaining data from pharmacies and health insurers, the drug companies learn the prescribing habits of thousands of doctors. The identity of patients is not disclosed in such data, but knowing in detail what individual doctors are prescribing enables drug makers to fine-tune their messages when sales reps call on doctors. What worries some government officials and patient advocates is that keying sales tactics to an individual doctor's prescribing preferences--known as data mining--may distort decision-making and fuel prescribing of new, high-cost drugs. Source: Los Angeles Times 12/14/09

Menopause, as Brought to You by Big Pharma

Millions of American women in the 1990s were told they could help their bodies ward off major illness by taking menopausal hormone drugs. Some medical associations said so. Many gynecologists and physicians said so. Respected medical journals said so, too. Along the way, television commercials positioned hormone drugs as treatments for more than hot flashes and night sweats. One commercial about estrogen loss by the drug maker Wyeth featured a character discussing research into connections between menopause and heart disease, Alzheimer’s disease, and blindness. Now, more than 13,000 people have sued Wyeth over the last seven years, claiming in courts across the country that its menopause drugs caused breast cancer and other problems. The suits also assert, based on recently unsealed court documents, that Wyeth oversold the benefits of menopausal hormones and failed to properly warn of the risks. The drug giant Pfizer, which absorbed Wyeth and its hormone drugs in a merger this year, says that Prempro is a safe, federally approved drug, and that Wyeth acted responsibly by including a clear warning about a breast cancer risk on Prempro labels and by updating the warning as new evidence emerged. Pfizer plans to appeal every product liability case on menopausal drugs it loses. Source: New York Times 12/13/09

FDA Has Yet to Make Safety Changes Post-Vioxx

The U.S. Food and Drug Administration (FDA) still hasn't restructured its staff to better monitor drug safety, more than three years after experts recommended key changes in the wake of the Vioxx scandal. That's according to congressional investigators who found that the FDA has yet to follow through on changes suggested in 2006 to help the agency detect problems with drugs taken by millions of Americans. Those recommendations came after the embarrassing and dangerous episode with Vioxx, a blockbuster pain drug the FDA approved in 1999, only to pull from the market in 2004 after linking it to heart attack and stroke. Agency officials have made some changes to drug oversight, according to a Government Accountability Office report, but the FDA continues to give the bulk of its decision-making power to scientists who approve new drugs, rather than those who monitor the side effects of drugs on the market. The FDA said it intends to give its Office of Surveillance and Epidemiology more responsibilities, but only after its nearly 200 employees gain the experience and resources needed to take on those tasks. Source: Associated Press/Yahoo! News 12/9/09

FDA Staff Urge More Antipsychotic Review in Kids

U.S. drug reviewers recommended that regulators further study the effects in children of a group of medicines known as atypical antipsychotics, a report released on December 4 said. Food and Drug Administration (FDA) staff said a recent study of health insurance claims found children treated with the drugs "were much more likely to experience an adverse metabolic effect than adults, and the likelihood was directly correlated with age." Metabolic effects can include abnormal weight gain, diabetes and increases in cholesterol and blood pressure. The report said an FDA analysis showed increased reporting of metabolic problems with Eli Lilly & Co.'s Zyprexa and AstraZeneca's Seroquel. The FDA review also included Bristol-Myers Squibb Co. and Otsuka Pharmaceutical Co.'s Abilify, Johnson & Johnson's Risperdal, and Pfizer Inc.'s Geodon. Source: Reuters/Yahoo! News 12/4/09

U.S. Approves First "Ethical" Human Stem Cell Lines

The U.S. government approved the first 13 batches of human embryonic stem cells on December 2, enabling researchers using them to get millions of dollars in federal funding as promised by President Barack Obama in March. The batches, known as lines, were made by two researchers at Harvard University and Rockefeller University using private funds. The National Institutes of Health set up a panel to decide which stem cell lines met strict ethical restrictions. The cells, for instance, have to have been made using an embryo donated from leftovers at fertility clinics, and parents must have signed detailed consent forms. Another 96 lines are under consideration and more approvals can be expected soon. Source: Reuters/Yahoo! News 12/2/09

Pfizer Deal Poses Challenge to Genzyme

Pfizer Inc., mounting a direct challenge to Genzyme Corp., says it will spend up to $110 million to license the global rights to a treatment being developed for the rare genetic disorder Gaucher disease. Pfizer’s alliance with Israeli biotechnology start-up Protalix BioTherapeutics Inc. to eventually sell the drug, which is based on plant cell technology, takes aim at Genzyme’s top-selling product, Cerezyme, which also treats Gaucher disease. Beyond the licensing deal, Pfizer says it wants to use its partnership with Protalix as a template to move into the lucrative market of treating rare diseases. “Historically, people thought big pharma companies wouldn’t be interested in that market because their focus was on blockbuster drugs that treat many more people,’’ says biotechnology analyst Phil Nadeau. “This could signal a new interest by big pharma in these rare drugs.’’ Source: Boston Globe 12/2/09

FDA Panel Backs Pfizer's Enhanced Vaccine for Kids

Federal health experts said on November 17 that an updated version of Pfizer's best-selling anti-infection vaccine is safe and effective for infants and toddlers, despite company studies that failed to meet certain goals. The Food and Drug Administration's (FDA's) panel of vaccine experts voted 10-1 in favor of Pfizer's Prevnar 13 to protect against pneumococcal disease. The FDA is expected to make its approval decision by December 30. The new vaccine reduces risk of infection from 13 varieties of the disease, which causes thousands of cases of ear infection, meningitis, and pneumonia each year. The new version adds protection against six additional strains to the seven covered by the current vaccine. FDA reviewers noted that company studies failed to meet preselected goals for three types of pneumococcal disease, but Pfizer scientists argued that the missed statistical targets were a result of comparisons between the new vaccine and the original Prevnar. The FDA panel recommended long-term safety tracking for the new vaccine, and Pfizer has agreed to a 43,000-patient follow-up study. Source: Associated Press/Washington Post 11/18/09

Vaccines on Horizon for AIDS, Alzheimer's, Herpes

Malaria. Tuberculosis. Alzheimer's disease. AIDS. Pandemic flu. Genital herpes. Urinary tract infections. Grass allergies. Traveler's diarrhea. You name it, the pharmaceutical industry is working on a vaccine to prevent it. Many could be on the market in five years or less. Contrast that with five years ago, when so many companies had abandoned the vaccine business that half the U.S. supply of flu shots was lost because of factory contamination at one of the two manufacturers left. Vaccines are no longer a sleepy, low-profit niche in a booming drug industry. Today, they are starting to give ailing pharmaceutical makers a shot in the arm, as they look for ways to bolster slowing prescription medicine sales amid intensifying generic competition and government pressure to cut down prices under the federal health overhaul. Source: Associated Press/Yahoo! News 11/18/09

European Union Trial Rules Stall Research

European clinical trial guidelines meant to make trials safer and more efficient are actually slowing down studies that could help patients, and even dissuading researchers from launching trials at all, according to an opinion published online in PLoS Medicine. The European Union's (EU's) Clinical Trials Directive, adopted in 2004, says that trials evaluating investigational medicinal products should follow Good Clinical Practice (GCP), an international quality standard for human clinical trials. According to Alex McMahon of the University of Glasgow in Scotland and his colleagues, who wrote the opinion piece, the stringent definition of GCP employed in the directive makes sense for trials sponsored by biopharma companies, which are generally developing new medicines, but not for trials run by academic medical centers, which more often are studying already-licensed drugs in order to fine tune their use in the clinic. Although the regulatory suggestions outlined in the directive are not legally binding in most EU countries, the authors write, academic centers and regulatory agencies are needlessly enforcing them, and this over-interpretation of the rules is dramatically slowing academic clinical work. Source: The Scientist 11/17/09

Breaching a Barrier to Fight Brain Cancer

Getting drugs into the brain has always been a major challenge in treating tumors and other neurological diseases, because the blood-brain barrier, a natural defense system, keeps many drugs out. However, a safety study at NewYork-Presbyterian/Weill Cornell is combining old technologies in a new way to open the barrier and deliver extraordinarily high doses of Avastin straight to glioblastomas—without soaking the rest of the brain in the drug and exposing it to side effects. The technique might also be useful for brain metastases, and could also deliver other drugs and might eventually be used to treat neurological disorders like multiple sclerosis or Parkinson’s disease, if suitable therapies are developed. The new technique uses microcatheters to spray chemotherapy directly onto tumors or areas from which they have been removed. “This will substantially alter the way that chemotherapy is given in the future,” said Dr. John Boockvar, the brain surgeon who devised the trial. “But we have to prove that at certain doses, nobody gets hurt.” Source: New York Times 11/17/09

New Study Questions Effectiveness of Popular Cholesterol Drugs

A widely prescribed and expensive cholesterol drug is not as effective as niacin, a cheap vitamin, in helping to unclog coronary arteries in people already taking statins, the standard medicines used to lower cholesterol, according to a new study. The research, which appears in the New England Journal of Medicine, is sending rumbles through the medical community because it is the third recent study to raise questions about the effectiveness of Zetia and its sister drug, Vytorin, highly profitable pharmaceuticals made by Merck & Co. Although the drugs have been shown to reduce cholesterol, there is no evidence that they prevent heart attacks, strokes and other cardiovascular problems. Top Merck executives are vigorously defending their drugs and have dismissed the new research as limited. Merck has a large-scale clinical trial of 15,000 patients under way that is not expected to yield results until at least 2012. Source: Washington Post 11/16/09

Seeking a Shorter Path to New Drugs

What’s smaller than a breadbox, can cost $800 million or more, and takes more than eight years to research and develop? A new medicine, according to data from the Tufts Center for the Study of Drug Development in Boston--and that’s just for traditional drugs made by using old-fashioned chemical molecules. Developing new biopharmaceuticals--those high-tech wonder drugs fashioned from living cells--can cost $1.2 billion, according to the center. Those costs aren’t only for the drug that has successfully made it through the testing gamut to your medicine cabinet, but also for all earlier iterations that failed. This linear, trial-and-error method is no longer a sustainable model for big pharmaceutical companies. Now big-picture thinkers, within the industry and outside it, are re-examining every stage of drug development--from molecule to market--in an effort to foster faster innovation. It’s a holistic approach, called “systems thinking,” that originated in methods that engineers used to streamline projects in the aeronautics and automotive industries. Source: New York Times 11/15/09

Review: Reports on Pfizer Drug Studies Misleading

Analysis of a dozen published studies testing possible new uses for a Pfizer Inc. epilepsy drug found that reporting of the results was often misleading, indicating the medicine worked better than internal company documents showed. According to the report, when a company-funded study's primary finding wasn't favorable, that result was usually buried and something else positive was highlighted, without disclosing the switch. The documents used in the review were obtained by lawyers suing Pfizer for refunds on prescriptions paid for by insurers and consumers. The lawyers, who are seeking class action status for the cases, claim Pfizer concealed evidence the epilepsy drug Neurontin didn't work for those unapproved uses, including nerve pain, migraines, and bipolar disorder. Pfizer disputes the report's conclusions, saying the company never "attempted to mislead the medical community about the effectiveness" of the drug for certain uses. Source: Associated Press/Yahoo! News 11/11/09

Drug Industry Presses FDA to Allow More Online Ads

As federal regulators take their first tentative steps toward policing the wild west of medical information online, pharmaceutical companies are pressing their case to market drugs via Google, Twitter, and other websites. The Food and Drug Administration (FDA) will convene a two-day meeting beginning November 12 to hear the drug industry's position on Internet marketing. The agency has agreed to consider developing rules for online advertising after companies complained that the current guidelines for traditional media--which require a detailed list of possible side effects--have left them hamstrung on the Web. Source: Associated Press/Yahoo! News 11/11/09

Partnering for Global Health

Scientists from companies such as Novartis, GlaxoSmithKline, and Merck & Co. have started collaborating with experts across the nonprofit and academic sectors to send new drugs and vaccines into the pipeline. As this partnership model evolves, more companies appear ready to take on an active role in researching treatments that will probably never make a profit. Only a few years into their alliances, the partners are starting to see successes, ranging from reformulating a malaria drug for children to discovering compounds that show promise in treating tuberculosis. However, rapid growth in the drug and vaccine pipeline is overshadowed by questions about who is going to pay for the expensive late-stage clinical trials, registration, manufacturing, and distribution of products with no profits. Source: C&EN (Chemical & Engineering News) 11/9/09

New Vaccine Offers Hope in Africa's Malaria Battle

There is new hope for treating malaria, the mosquito-borne disease that kills a million African children every year: A vaccine that appears to be able to prevent the disease in about 50 percent of children is now undergoing the final stage of testing. If regulators determine the vaccine is safe, it could be on the market in three to five years--the first vaccine against a human parasite. Malaria is also prevalent in parts of Asia, the Middle East, and Central and South America. This vaccine was developed specifically for Africa, however, and will only prevent the African strain of the disease. Experts say it would be a historic advancement. More than $500 million has been spent on the combined efforts by drug maker GlaxoSmithKline and the PATH Malaria Vaccine Initiative, which is funded by the Bill & Melinda Gates Foundation. The Phase III testing is being done at 11 sites in seven African countries on 16,000 children under the age of 18 months. Source: Associated Press/Yahoo! News 11/3/09

FDA Won't Accept Merck's Application for New Drug

U.S. regulators have refused to accept drug maker Merck & Co.'s application for a new combination cholesterol pill that once looked sure to be a blockbuster: a combination of Pfizer Inc.'s Lipitor and Zetia, a cholesterol pill Merck sells with Schering-Plough Corp. The two drug makers have been partners on cholesterol medicines since 2000 and are about to merge. Merck applied to the Food and Drug Administration (FDA) in September for approval of the combo pill, but Merck said the FDA refused to file the application and instead is requiring more data on the manufacturing and stability of the drug. Normally, when companies seek approval of an experimental drug, the FDA accepts the application and spends months reviewing it; if the data is deemed inadequate or otherwise problematic, the agency usually requires further information and sometimes additional patient studies. In this case, the FDA would not file the application. Source: Reuters/Yahoo! News 11/2/09

Human Genome, GlaxoSmithKline Lupus Drug Works in Second Trial

Human Genome Sciences Inc said its experimental lupus drug Benlysta was successful in a second large clinical trial, paving the way for approval of the first new treatment for the disease in 50 years. The results from the latest clinical trial were not quite as convincing as those from an initial study, but still looked good enough to secure a green light from regulators and ensure doctors use the drug widely, industry analysts said on November 2. The new trial showed patients given a high dose of Benlysta experienced a statistically significant improvement in symptoms compared with those taking a placebo. Hopes for Benlysta have been growing since July, when it was shown to work in a first clinical trial--to the surprise of many experts who had been skeptical, given the previously poor track record of new lupus treatments. Source: Reuters/Yahoo! News 11/2/09

Geron, FDA Agree on Spinal Injury Drug Trial

Stem cell research company Geron Corp. in late October said it reached an agreement with U.S. health regulators that may enable it to restart the early-stage trials of its cell therapy to treat complete thoracic spinal cord injury. In August, the U.S. Food and Drug Administration (FDA) placed a clinical hold on Geron's investigational new drug application after some of the animals developed cysts in the injury site. While the early-stage trial still remains on clinical hold, a recent agreement with the FDA outlines what is necessary to move the spinal cord injury program forward. Geron, which expects to reinitiate the early-stage trial in the third quarter of 2010, said the FDA has advised that positive data from an ongoing preclinical study using its product can be used to support both release of the clinical hold and the drug's expansion to cervical patients. However, an analyst said, "A great amount of work still needs to be done before any patient is ultimately enrolled into the study." Source: Reuters 10/30/09

Bending the Rules of Clinical Trials

In her New York Times article about a case in which she wanted to help a patient enroll in a trial for which the patient was not eligible, Pauline W. Chen, MD, writes, "It turns out that I am not the only doctor who has considered bending the admissions criteria of a clinical trial for a patient. There is an essential conflict for doctors involved in clinical research. As collaborators in research, they want to obtain information that is valid and able to be generalized for all future patients; but as doctors working within a patient-doctor relationship, they need to focus only on improving the condition of the patient before them. ...Now, a recently published survey reveals that it may not be the patients who are most vulnerable [in such situations], but the trials themselves. In the current issue of the bioethics journal IRB: Ethics & Human Research, investigators from four different institutions surveyed over 700 clinicians involved in clinical trials and found that 90 percent believed that ignoring certain entry criteria was acceptable if a patient could, in their estimation, benefit from the trial. In addition, over 60 percent of those surveyed also believed that researchers should deviate from study rules if doing so might improve a patient’s care." Source: New York Times 10/29/09

Report Says FDA Fails to Follow Up on Unproven Drugs

The Food and Drug Administration (FDA) has allowed drugs for cancer and other diseases to stay on the market even when follow-up studies showed they didn't extend patients' lives, say congressional investigators. A report due out October 26 from the Government Accountability Office (GAO) also shows that the FDA has never pulled a drug off the market due to a lack of required follow-up about its actual benefits--even when such information is more than a decade overdue. When pressed about that policy, agency officials said they have no plans to get more aggressive. The GAO says the FDA should do more to track whether drugs approved based on preliminary results actually have lived up to their promise. The FDA responded that the report paints an overly negative picture of its so-called "accelerated approval" program, which is only used to approve drugs for the most serious diseases. Source: Associated Press/Washington Post 10/26/09

Research Uproar at a Cancer Clinic

Two years after becoming vice president for research at the biggest hospital in the university town of Urbana, Ill., Suzanne Stratton, PhD, said she had finally seen enough. She had clashed repeatedly with a doctor who oversaw the local patients enrolled in more than 130 federally sponsored cancer studies, but that Stratton said was often putting patients and science at risk. In a meeting with Carle Foundation Hospital administrators late last year, Stratton cited an outside audit that had found “major deficiencies” in 12 of 29 experiments being overseen by that doctor, potentially endangering patients or skewing the studies’ results. She says her bosses responding by firing her, but federal officials have corroborated many of the shortcomings she found and are continuing an investigation with implications for the nation’s cancer research effort that go far beyond the Carle Cancer Center, because they have never conducted a systematic review of the National Cancer Institute's community clinical oncology program of which the center is a member. Source: New York Times 10/23/09

Full Results Show AIDS Vaccine is of Modest Help

Fresh results from the world's first successful test of an experimental AIDS vaccine confirm that it is only marginally effective, and suggest that its protection against HIV infection may wane over time. Yet the findings are exciting to scientists, who think that blood samples from the trial may show how to make a vaccine that does a better job. The results also hint that the vaccine may work better in the general population than in those at higher risk of infection, such as gay men and intravenous drug users. Last month, researchers announced that a two-vaccine combination cut the risk of becoming infected with HIV by more than 31 percent in a trial of more than 16,000 volunteers in Thailand. Full results, published online October 20 by the New England Journal of Medicine and presented at a scientific conference in Paris, include two additional analyses that merely suggest the vaccine is beneficial, rather than providing definitive proof. An analysis of just the 12,452 participants who received all six shots exactly on schedule showed that there were 86 infections--36 in the vaccine group and 50 in the placebo group. Source: Associated Press/Yahoo! News 10/20/09

Opinion: Have Faith in an AIDS Vaccine

Seth Berkley, president and chief executive of the International AIDS Vaccine Initiative, writes in the New York Times..."[A] storm has erupted over the announcement last month that an experimental AIDS vaccine tested in Thailand proved modestly effective. It was billed as a major scientific advance--the long-awaited hard evidence that it is possible to inoculate people against AIDS. But now the trial has been called into question in a way that is overblown and possibly destructive. ...Although the candidate [drug pairing] tested in the Thai trial did not prove to be a vaccine ready for the market, it may provide an unprecedented opportunity to learn how an AIDS vaccine can work. ...[T]he [current] controversy over statistical significance is exaggerated. Whether you consider the first or second analysis, the observed effect of the Thai candidates was either just above or below the level of statistical significance. Statisticians will tell you it is possible to observe an effect and have reason to think it’s real even if it’s not statistically significant. And if you think it’s real, you ought to examine it carefully. Even if the Thai vaccine regimen turns out, on examination, to have had no real benefit, researchers will still learn from the trial, as they do from every study." Source: New York Times 10/19/09

Swine Flu Shots Revive a Debate About Vaccines

Anti-vaccinators, as they are often referred to by scientists and doctors, have toiled for years on the margins of medicine, but an assemblage of factors around the swine flu vaccine is giving the anti-vaccine movement a fresh airing, according to health experts. The factors include confusion over how the vaccine was made, widespread speculation about whether it might be more dangerous than the virus itself, and complaints among some healthcare workers in New York about a requirement that they be vaccinated. Websites, Twitter feeds, talk radio, and even elevator chatter are awash with skeptics criticizing the vaccine, largely with no factual or scientific basis. The most common complaint is that the vaccine has been newly formed and quickly distributed without the benefit of clinical trials; in fact, the swine flu vaccine was made using the same techniques as seasonal flu shots over the last two decades, and a small number of clinical trials were conducted this year to determine the adequate dose. Source: New York Times 10/16/09

Placebo Effect is in the Spine as Well as the Mind

It's not all in the mind--the so-called placebo effect is real and reaches right down to the spine, German scientists said in October. The finding may help in the hunt for better ways to tackle pain and other disorders. Using modern imaging technology the researchers found that simply believing a pain treatment is effective actually dampens pain signaling in a region of the spinal cord called the dorsal horn, suggesting a powerful biological mechanism is at work. The ability of sham medicines with no active ingredient to produce real clinical benefits has long perplexed doctors and frustrated drug makers. Patients are typically given either an experimental drug or a dummy in clinical trials and the fact that those on placebo often get better, too, makes it hard to determine whether a new drug is working. Source: Reuters/Yahoo! News 10/15/09

The Nocebo Effect: The Flip Side of Medicine's Placebos

In clinical trials, every drug response is in fact assumed to be at least partially due to the placebo effect. However, the confounding thing about the benefits of the placebo is that the effect is often not beneficial at all. Consider the negative placebo response, called the nocebo effect. A nocebo response occurs when the suggestion of a negative effect of an intervention leads to an actual negative outcome. In double-blind clinical trials of antidepressants, even those participants receiving a sugar pill report side effects like gastrointestinal discomfort if investigators have warned them at the outset that those effects are likely. In a new paper published in the journal Pain, researchers found that clinical trial participants have reported a wide variety of nocebo-fueled medical complaints, including burning sensations outside the stomach, sleepiness, fatigue, vomiting, weakness, and even taste disturbances, tinnitus, and upper respiratory tract infection. What's more, these nocebo complaints aren't random; they tend to be specific to the type of drug that patients believe they may be taking. Source: Time/Yahoo! News 10/13/09 See also "Placebos are Getting More Effective. Drug Makers are Desperate to Know Why."

HIV/AIDS Vaccine Trial Questioned as Experts Check Data

It is an emotional cycle familiar to most AIDS vaccine researchers: the high of finally making measurable headway against HIV, followed by the crushing low of discovering that the virus has once again found a way to elude them. It happened again on October 10, when researchers learned that the first ever successful AIDS vaccine turned out not to be the triumph they had originally hoped. In September, scientists from the National Institutes of Health and the U.S. Army announced the results of an AIDS vaccine study in Thailand involving more than 16,000 volunteers. The data showed that the new vaccine had protected 31 percent of inoculated participants from becoming infected with HIV. However, a closer look at a subset of the study's volunteers now reveals that the vaccine in fact protected only 26 percent of the people who received it. The difference is small, but critical, because the new success rate falls below the threshold for statistical significance. That means that the odds of being protected from infection by the AIDS vaccine may be no better than chance. Source: Time/Yahoo! News 10/13/09

Pfizer Halts Enrollment in Lung Cancer Trial

Pfizer Inc has halted patient enrollment in a late-stage lung cancer trial of its experimental drug, figitumumab, for safety reasons. The halt was recommended by independent safety monitors who found more serious adverse events, including deaths, with patients receiving figitumumab. Currently enrolled patients may continue their treatment in consultation with their physicians. As of September 30, 681 patients were enrolled in the Phase III study out of a target of 820. The company notified all clinical trial investigators and regulatory agencies and will work closely with the safety monitoring committee on analyzing the data before issuing further guidance. Another Phase III lung cancer study evaluating figitumumab in combination with Roche and OSI Pharmaceuticals' Tarceva is continuing to enroll new patients. Source: Reuters/Yahoo! News 10/9/09

Cocaine Vaccine Study Offers Hope for Addiction Treatment

Researchers announced in October the early trial results of a new vaccine to fight cocaine addiction--a compound that, by vaccine standards, was only mildly effective, but served as an important proof of concept that vaccination against addiction could work. "Vaccines are one of our top priorities," says Dr. Nora Volkow, director of the National Institute on Drug Abuse, which helped fund the study and will finance a larger study of the cocaine vaccine next year. The vaccine stimulates the immune system to create antibodies that bind to drug molecules and prevent them from entering the brain; it vaccine produced a strong antibody response in 38 percent of the 55 people who received five doses, and when the antibody levels were highest, these participants used significantly less cocaine than either a placebo group or those who did not produce sufficient antibodies. Source: Time/Yahoo! News 10/9/09

Government Orders Columbia University to Tell Patients "True Nature" of Drug Study

Ten years ago, more than 200 open-heart surgery patients were part of a two-year medical study at Columbia University that government regulators now say was carried out with ethical and regulatory mistakes and may have caused harm to some patients. The study was testing a commonly used intravenous surgical fluid that previous studies had shown could cause hemorrhaging at high doses. At least two patients in the study died shortly after receiving the fluid, and more than two dozen others required transfusions, according to documents submitted to the federal government by the hospital. In the past decade, Columbia has conducted three separate internal reviews of the study. The reviews raised serious questions about the drug trial’s design, management, and oversight, but they concluded that there was no evidence that the fluid caused deaths or other medical problems for the patients and that there was no need to provide the patients with additional information about the study. Now, federal regulators have decided not to accept that conclusion. They have taken the rare action of demanding that Columbia track down the patients and their families, and acknowledge that they never were informed about the “true nature” of the drug study, the risks they faced, or the consequences of their participation. Source: Flesh and Stone/Huffington Post Investigative Fund 10/8/09

New Diabetes Drug Hits Target in Late-Stage Study

An experimental diabetes drug from Bristol-Myers Squibb and AstraZeneca met its main target in a late-stage study, achieving significant reductions in glycosylated hemoglobin levels. When added to the common diabetes pill metformin, dapagliflozin cut both levels of both glycosylated hemoglobin and fasting plasma glucose in patients with Type 2 diabetes after 24 weeks, hitting both its main and secondary targets. It is the first late-stage data on the once-daily pill dapagliflozin, a member of a class of drugs known as SGLT2 inhibitors and designed to block reabsorption of glucose to lower elevated blood sugar levels. If approved, it could be the first such drug to reach the market and the companies have said they could file for approval in late 2010 or early 2011. The study assessed results from 546 patients aged 18 to 77 who had Type 2 diabetes that was inadequately controlled with metformin alone. Source: Reuters/Yahoo! News 10/2/09

FDA Questions Safety of GlaxoSmithKline Kidney Cancer Drug

Federal regulators said in early October that an experimental kidney cancer drug from GlaxoSmithKline may cause liver problems, potentially outweighing its ability to slow the disease. The company wants the Food and Drug Administration (FDA) to approve its pazopanib pills for advanced kidney cancer, a rare but deadly form of the disease. In documents posted online, FDA reviewers noted three deaths related to liver damage with the drug, as well as elevated levels of enzymes that often predict liver damage. Since 2005, the FDA has approved five new treatments for the disease, including Pfizer's Sutent and Bayer's Nexavar. GlaxoSmithKline did not compare its drug to these treatments because they were not widely available when the company began designing a trial in 2005. Instead, the company compared pazopanib to placebo. Results from that 435-patient study showed patients taking the drug experienced a five-month halt in the progression of their cancer, although they did not live significantly longer. Source: Associated Press/Yahoo! News 10/1/09

Pancreatic Cancer: Hard to Diagnose, Even Harder to Treat

When actor Patrick Swayze died in September of pancreatic cancer, it put a famous face on a grim disease. This relatively rare cancer remains one of the most lethal. Only two drugs are Food and Drug Administration-approved for its treatment: gemcitabine (marketed as Gemzar) and erlotinib (Tarceva). The paucity of effective chemotherapy drugs is not for lack of trying, doctors say. However, many in the field have become discouraged. Such promising drugs as Avastin, axitinib, and aflibercept have failed Phase III trials in the past few years. A current Phase III trial is testing nab-paclitaxel, a drug coated with a protein that binds to a protein in the tumor cells, in combination with Gemzar. Source: Los Angeles Times 9/28/09

FDA Reveals It Fell to a Push by Lawmakers

The Food and Drug Administration (FDA) said on September 24 that four New Jersey congressmen and its own former commissioner unduly influenced the process that led to its decision last year to approve a patch for injured knees, an approval it is now revisiting. The agency’s scientific reviewers repeatedly and unanimously over many years decided that the device, known as Menaflex and manufactured by ReGen Biologics Inc., was unsafe because the device often failed, forcing patients to get another operation. However, after receiving pressure from four Democrats from New Jersey, agency managers overruled the scientists and approved the device for sale in December. All four legislators made their inquiries within a few months of receiving significant campaign contributions from ReGen, which is based in New Jersey, but all said they had acted appropriately and were not influenced by the money. Dr. Andrew C. von Eschenbach, the drug agency’s former commissioner, said he had acted properly. Source: New York Times 9/25/09

A World First: Vaccine Helps Prevent HIV Infection

For the first time, an experimental vaccine has prevented infection with the AIDS virus, a watershed event in the deadly epidemic and a surprising result. The vaccine—a combination of two previously unsuccessful vaccines—cut the risk of becoming infected with HIV by more than 31 percent in the world's largest AIDS vaccine trial of more than 16,000 volunteers in Thailand, researchers announced September 24. The study was sponsored by the U.S. Army and the National Institute of Allergy and Infectious Diseases, and conducted by the Thailand Ministry of Public Health. It tested the two-vaccine combination in a "prime-boost" approach, in which the first one primes the immune system to attack HIV and the second one strengthens the response. They are ALVAC, from Sanofi Pasteur, and AIDSVAX, held by a nonprofit, Global Solutions for Infectious Diseases. Source: Associated Press/Yahoo! News 9/24/09

Space-Based Vaccine May Go to Human Trials

A vaccine created from research in space may soon be put to the test in human trials for the first time. The Astrogenetix company, based in Austin, Texas, has begun applying for approval to begin testing its space-designed salmonella vaccine on humans. The company created its vaccine candidate based on research conducted on NASA's space shuttle flights. Astrogenetix has sent up experiments on 23 shuttle missions so far, and plans to continue with the six remaining missions on NASA's current schedule. The key to new vaccines lies in an important difference in the way things grow in space. Many bacteria and viruses have been shown to grow more quickly in the microgravity of space, as well as become more virulent or more infectious. The changes that occur in weightlessness help speed up experiments, and help point researchers toward the causes of virulence, both in space and on Earth. Source: Space.com/Yahoo! News 9/23/09

Wyeth Sues FDA to Block Rival Generic Antibiotic

Drug maker Wyeth on September 23 sued the Food and Drug Administration (FDA) to block the sale of a generic rival to its intravenous antibiotic Zosyn, claiming the generic is not an equivalent product and could harm critically ill patients. Wyeth filed a federal lawsuit against the FDA, seeking both a temporary restraining order and a preliminary injunction. The lawsuit seeks to prevent Orchid Chemicals & Pharmaceuticals Ltd. of India from selling a generic version of Zosyn that the FDA approved last week. Wyeth's head of global medical affairs, Joe Camardo, said patient safety is at stake, and that Orchid's product could lead to preventable medical errors. The problem is that with an older formulation of Zosyn, which Wyeth claims is what Orchid is using, Zosyn can mix with intravenous solution in a way that causes a chemical reaction that inactivates the antibiotic, limiting how much patients would get. Source: Associated Press/Yahoo! News 9/23/09

New Gene-Targeted Drug Offers Hope in Skin Cancer

Scientists offered new hope in the fight against the most deadly type of skin cancer in September, as an early-stage clinical trial showed an experimental drug dramatically shrank tumors. Paul Chapman from the Memorial Sloan-Kettering Cancer Center in New York said 70 percent of patients with a particular gene mutation saw their tumors shrink when given the new pill, called PLX4032. Of the 27 patients evaluated in the small Phase I study, two responded completely and signs of their disease disappeared. Preliminary findings with the new drug were first reported in June, but the data from more patients have boosted confidence in its prospects. Chapman and his colleagues are planning a Phase II trial of 90 patients starting at the end of 2009, and a large international Phase III trial involving several hundred patients is planned to start in late 2009 or early 2010. PLX4032, which Roche is developing with privately held U.S. biotech company Plexxikon, is the latest in a growing group of gene-targeted drugs that oncologists believe will drive future cancer treatment. Source: Reuters/Yahoo! News 9/23/09

Pill Beats Hopes in Breast Cancer Test

Women with breast cancer lived significantly longer without their disease getting worse when treated with Bayer and Onyx Pharmaceuticals' cancer pill Nexavar, researchers said in September. Nexavar, a targeted anticancer drug sold as a liver and kidney cancer therapy in more than 70 countries, beat analysts' expectations in the breast cancer trial, increasing the time patients lived without their disease progressing by 74 percent. The Phase II trial showed that Nexavar, when combined with standard chemotherapy agent Xeloda, halted tumor growth for 2.3 months more than Xeloda alone, giving patients 6.4 progression-free months--a result analysts described as impressive. Analysts said Nexavar's promise in early trials may open up opportunities for doctors to prescribe it off-label before it undergoes the final-stage trials that usually precede a license. Source: Reuters/Yahoo! News 9/23/09

New Drugs May Attack Flu, But Not in Time

Besides the vaccines aimed at preventing flu, new drugs are on the way to treat it once it strikes. However, it is not clear whether these drugs will arrive in time to make a difference in thwarting the H1N1 flu pandemic. New drugs are needed, researchers say, because there are now only four approved flu drugs that work by only two different mechanisms. Furthermore, many strains of influenza are already resistant either to the two older drugs, amantadine and rimantadine, or to Tamiflu, a somewhat newer drug. The H1N1 pandemic strain, or swine flu, is resistant to the two older drugs. There have been only sporadic cases of resistance to Tamiflu so far, but if such resistance spread, the only effective drug left would be Relenza. Source: New York Times 9/22/09

Buying Binge Hits Biotech Firms

Buy or be bought. That appears to be the new mantra for the Massachusetts life sciences industry, where the strengthening economy and rising stock market have sparked a small wave of acquisitions. On September 21 alone, Waltham’s Inverness Medical Innovations Inc. agreed to purchase a Seattle company, while Cambridge’s Biogen Idec Inc. launched a hostile tender offer for a California company. With another biotech company, Sepracor Inc. of Marlborough, agreeing earlier in September to be sold to a Japanese pharmaceutical giant, the scramble for merger partners is on. Although each deal is different, several factors are driving the bump in takeovers among biotech, medical device, and diagnostic firms that had been holding off on mergers. Small companies need cash, big ones are seeking to expand their product lines, capital markets are loosening, and early fears about a health care overhaul leading to price controls or patent restrictions now seem unfounded. Together, these factors have unleashed a spurt of pent-up deal demand that is likely to continue, industry insiders said. Source: Boston Globe 9/22/09

Failed Cancer Vaccine May Help Some

A therapeutic cancer vaccine from Oxford BioMedica that failed in a clinical study may still help a subset of patients, researchers said in September. Shares in the company plunged as much as 18 percent in early trade as investors reacted to the reiterated trial failure rather than the fact the drug, TroVax, may help some patients, analysts said. The data in fact show TroVax works if patients most likely to respond are preselected via a blood test. The original failure prompted French drug maker sanofi aventis to hand back rights to TroVax in April. The latest data suggested it may yet have a future and could kindle renewed interest from potential pharmaceutical partners. Although TroVax did not show a significant survival advantage in the overall population studied, patients with a good prognostic profile receiving interleukin-2 therapy did live longer, results presented at a major cancer conference showed. Source: Reuters/Yahoo! News 9/22/09

Medical Editors Push for Crackdown on Ghostwriting

The scientific integrity of medical research has been clouded in recent years by articles that were drafted by drug company-sponsored ghostwriters and then passed off as the work of independent academic authors. However, the leading medical journals have continued to rely largely on an honor system of disclosure to detect such potential bias, asking authors to voluntarily report any industry ties or contributors to their manuscripts. Now, in light of recently released evidence that some drug makers have gone to great lengths to turn scientific articles into marketing vehicles for their products, some influential medical editors are cracking down on industry-financed ghostwriting, and they are getting help from some members of Congress. Over the last few years, international associations of medical journal editors have developed stricter disclosure criteria for authors of and contributors to scientific manuscripts. Drug companies say they are about to put these publication principles into effect for clinical trials. Source: New York Times 9/18/09 See also "Ghostwriting is Called Rife in Medical Journals" New York Times 9/11/09

FDA Chief Calls for More Regulatory Science

The U.S. must bolster study on how to best craft regulations that bring drugs, medical devices, and vaccines to market, the commissioner of the Food and Drug Administration (FDA) stressed in a speech delivered on September 16 in Philadelphia. Margaret Hamburg, speaking at the annual Regulatory Affairs Professional Society conference to approximately 850 representatives from pharmaceutical companies, biotechs, and medical device manufacturers, said, "Just as biomedical research has evolved in the past decades, regulatory science--the science and tools we use to assess and evaluate product safety, efficacy, potency, quality, and performance--must also evolve." Source: The Scientist 9/17/09

Merck, British Charity to Jointly Develop Vaccines

Drug maker Merck & Co. and Britain's largest charity, the Wellcome Trust, said in September that they are starting a not-for-profit partnership to create affordable vaccines against diseases common in poor countries. The joint research venture will develop new vaccines for diseases with unmet need and work to improve existing vaccines, such as by finding lower-cost production methods or tweaking them to make them stable at room temperature. Merck will have the first rights to license any vaccines developed by the partnership, which will consult with experts around the world to pick initial projects. One priority, though, will be a vaccine against Group A Streptococcus, which kills more than 500,000 people a year worldwide. The venture will be based in India and will have about 60 researchers and developers on staff, including some from Merck. It is expected to begin operating sometime next year. Source: Associated Press/Yahoo! News 9/17/09

Where Cancer Progress Is Rare, One Man Says "No"

Politicians and researchers have predicted for nearly four decades that a cure for cancer is near, but cancer death rates have hardly budged and most new cancer drugs cost a fortune while giving patients few, if any, added weeks of life. For this collective failure, the man atop the nation’s regulatory agency for new cancer drugs increasingly--and supporters say unfairly--gets the blame: Dr. Richard Pazdur. Patient advocates have called Pazdur, director of the Food and Drug Administration’s (FDA's) cancer drug office, a murderer, conservative pundits have vilified him as an obstructionist bureaucrat, and guards are now posted at the agency’s public cancer advisory meetings to protect him and other committee members. In 10 years at the FDA, Pazdur has helped to loosen approval standards for cancer medicines and made it easier for dying patients to get experimental drugs. However, he demands that drug makers prove with near certainty that their products are beneficial. Critics say that Pazdur’s resolve has cost thousands of lives and set back the pace of discoveries. Source: New York Times 9/16/09

Lilly Shuffles Staff, Cuts Thousands of Jobs

Eli Lilly announced in September that it would eliminate approximately 5,500 positions as part of a massive reorganization strategy that is expected to net the firm reduce $1 billion in costs by 2011. This move will drop the company’s headcount from approximately 40,500 to 35,000. The company has not revealed where the cuts will come from or how many will be real layoffs and how many will simply be reduced positions. The company also revealed plans to follow the lead of companies like Pfizer and Johnson & Johnson and segment its pharmaceutical model into business units (for diabetes, established markets, emerging markets, oncology, and animal care), and created a central developmental program to boost production of late-stage drugs. Source: PharmExec Direct 9/14/09

Government Approves New Swine Flu Vaccine

The U.S. Food and Drug Administration approved the new swine flu vaccine on September 15, a long-anticipated step as the government works to get vaccinations under way in October. Health and Human Services Secretary Kathleen Sebelius announced the vaccine's approval to Congress, saying she hopes to get the first limited supplies distributed early in October. The bulk of vaccine will start arriving October 15, and Sebelius said it should be available at 90,000 sites around the country. The government has ordered 195 million doses for now, but may order more if needed, she said. The vaccine is made by CSL Ltd. of Australia; Switzerland's Novartis; Sanofi-Pasteur of France; and Maryland-based Medimmune. Source: Associated Press/Yahoo! News 9/15/09

FDA Panel Backs GlaxoSmithKline's Cervical Vaccine for Women

Drug maker Merck likely will face U.S. competition for its vaccine Gardasil, after federal experts recommended rival GlaxoSmithKline's Cervarix also be approved to prevent the virus that causes most cervical cancers. The Food and Drug Administration's (FDA's) panel of vaccine experts voted overwhelmingly on September 9 that Cervarix appears safe and effective for girls and women ages 10 to 25. If the FDA follows the group's advice as it usually does, GlaxoSmithKline would begin competing against Merck's Gardasil, which has controlled the U.S. market since 2006. However, Merck won its own small victory at the meeting, as the same panel recommended Gardasil be expanded to prevent genital warts in boys, a new use for a vaccine that already posts sales of more than $1 billion. Source: Associated Press/Yahoo! News 9/9/09

Stem Cell Company Faked Success

A stem cell company is in hot water with the U.S. Securities and Exchange Commission (SEC) for falsely representing an early-stage experimental stem cell therapy as nearing human trials. The SEC on September 8 filed charges against CellCyte, based in Bothell, Wash., as well as the company's former CEO and former chief scientific officer, who were involved in the alleged deception, according to Forbes.com. "The company...tried to take advantage of the hype over stem cells to give the false impression that they were on the verge of clinical trials, when really it was just an early-stage project that was going to require years of additional research and testing," SEC staff attorney Steven Buchholz told The Scientist. Source: The Scientist 9/9/09

Pfizer to Pay Record $2.3 Billion Penalty Over Promotions

Federal prosecutors hit Pfizer Inc. with a record-breaking $2.3 billion in fines on September 2 and called the world's largest drug maker a repeating corporate cheat for illegal drug promotions that plied doctors with free golf, massages, and resort junkets. Announcing the penalty as a warning to all drug manufacturers, Justice Department officials said the overall settlement is the largest ever paid by a drug company for alleged violations of federal drug rules. The allegations surround the marketing of 13 different drugs, including big sellers such as Viagra, Zoloft, and Lipitor. Source: Associated Press/Yahoo! News 9/2/09

Taking Risk for Profit, Industry Seeks Cancer Drugs

Virtually every large pharmaceutical company seems to have discovered cancer, and a substantial portion of the smaller biotechnology companies are focused on it as well. Together, the companies are pouring billions of dollars into developing cancer drugs. About 860 cancer drugs are being tested in clinical trials; more than twice the number of experimental drugs for heart disease and stroke combined, nearly twice as many as for AIDS and all other infectious diseases combined, and nearly twice as many as for Alzheimer’s and all other neurological diseases combined. However, only a trickle of new cancer drugs make it to market. Source: New York Times 9/2/09 See also "Lack of Study Volunteers Hobbles Cancer Fight," another article in this New York Times series.

Patients Gather for the Largest Clinical Study Ever of a Rare Disorder

In an attempt to unravel some of the unknowns of a potentially lethal genetic disease that makes milk poison to infants, 35 patients gathered in Boston in August for a two-day battery of tests and to meet with an international panel of doctors. It was part of the largest-ever comprehensive clinical study of adults with the disease, called galactosemia. The study--primarily funded by the patient advocacy group, Parents of Galactosemic Children, and made possible by the willingness of patients to pay their way to Boston--is just one example of the ways in which families and patient groups are driving scientific research on rare diseases. Source: Boston Globe 8/31/09

Half of Health Workers Might Reject Swine Flu Shot

About half of Hong Kong's health workers would refuse the swine flu vaccine, new research shows, and experts say that the trend would likely apply worldwide. Doctors and nurses are on the swine flu front lines--and if they become infected, they may not only spread the disease to patients, but their absence from work could cripple health systems. The World Health Organization recommends countries vaccinate their health workers. "A good argument can be made that health workers have an ethical obligation to be vaccinated, not to protect themselves, but to protect their patients," said George Annas, a bioethics expert at Boston University. "But if they don't believe that vaccine to be safe and effective, it will be a hard sell." Source: Associated Press/Yahoo! News 8/25/09

Research Trove: Patients’ Online Data

Since the Internet’s earliest days, patients have used the Web to share experiences and learn about diseases and treatments. But now advocates say that online communities have the potential to transform medical research--especially into rare diseases that lack the number of patients needed for large-scale studies and rarely attract research financing from the drug industry. Patients’ everyday experiences in living with an illness represent an enormous source of untapped data; aggregated, those data could generate new hypotheses and avenues for research. Several private companies are now collecting patient data and genetic information online to use in recruiting patients for clinical trials, conducting research internally, or to sell to drug and biotechnology companies. Source: New York Times 8/25/09

End of Road for Biopure

Biopure Corp., the Cambridge, Mass. company that was working to develop a human blood substitute, has officially expired. In July, the struggling biotech filed for Chapter 11 bankruptcy protection, and in late August it said an auction of the company’s assets had been completed. The firm was once one of the state’s most promising biotechs. Hemopure, the blood substitute made from cow hemoglobin, long seemed on the verge of emerging as a life-saving breakthrough. It had the potential to be used in emergency situations--such as at accident scenes or on battlefields--when supplies of human blood might be limited. However, Biopure could never clear regulatory hurdles that would have turned the substitute into a mainstream medical product. Source: Boston Globe 8/25/09

Opinion: Stigma of Lung Cancer Stymies Research Funds

Kim Norris, president of the Lung Cancer Foundation of America, writes in the Los Angeles Times that lung cancer, "the leading cause of cancer deaths in the United States, kills more people than breast, prostate, colon, and pancreatic cancers combined and is considered one of the most significant effects of tobacco use on public health. Yet lung cancer remains the most under-funded and under-researched cancer... Because lung cancer still carries the stigma that somehow you brought the disease on yourself, tobacco taxes rather than direct funding remain the perennially popular government source of funding to make up for the shortfalls in general spending. The bottom line is that all cancer victims should be treated equally regardless of personal habits; all cancers are the direct result of genetics, environment, and behavior." Source: Los Angeles Times 8/24/09

Why Racial Profiling Persists in Medical Research

Experts within the research community say a small but stubborn streak of racial profiling has long persisted in the medical literature, borne out in studies that attribute health disparities between blacks and whites not to socioeconomics or access to healthcare alone, but also to genetic differences between the races--a concept that implies that a biological category of race exists. The controversy resurfaced in July with the publication of a study in the Journal of the National Cancer Institute in which researchers analyzed more than 19,000 patients who participated in clinical trials involving treatments for a variety of cancers. The paper found that all other factors being equal, black patients had on average a significantly lower cancer survival rate than whites. The conclusion that race was at fault, critics quickly responded, was flawed. This is a uniquely American phenomenon, experts say. In other countries, information about race is usually not available to medical researchers, as it is not collected in census data or in birth and death certificates. Source: Time/Yahoo! News 8/22/09

Activists Say Poor Nations' Access to Affordable Drugs Stymied

Activists in the U.S., as well as Thailand and other developing countries, have accused the Obama administration of blocking greater access to affordable drugs in a bid to win the pharmaceutical companies' support for its healthcare overhaul. Organizations such as Doctors Without Borders and Oxfam International said they had hoped President Obama would join them in the fight to make generic drugs available to combat disease in impoverished countries. Source: Los Angeles Times 8/20/09

Study: Lung Cancer Pill Works for Some Patients

Four years after the government severely restricted its use, the lung cancer drug Iressa may be poised to make a comeback: A study concludes it can slow the deadly disease better than standard chemotherapy in certain patients. The research is the first to show Iressa can be more effective than chemotherapy as a first-line treatment, and some experts are hopeful it will prompt the Food and Drug Administration to allow wide use of the drug, made by AstraZeneca PLC. Others are doubtful. Although the study shows an effect on cancer growth over one year, the drug's impact on long-term survival is still in question. Also, the study was done in Asia, and the drug seems to work best with specific patients--Asians, women, and nonsmokers who carry a specific gene mutation. Source: Associated Press/Yahoo! News 8/19/09

FDA Warns Johnson & Johnson On Antibiotic Trials

The Food and Drug Administration (FDA) has warned Johnson & Johnson that it did not properly monitor two human tests of its antibiotic drug candidate ceftobiprole, which is intended to treat complicated skin infections like MRSA. The agency has twice delayed approval of ceftobiprole, citing similar problems. It said Johnson & Johnson violated the protocols of its own study, by, for example, not making sure patients were storing the drug properly when using it at home. The FDA also said Johnson & Johnson failed to document some doses given to patients, did not conduct thorough examinations of all patients, enrolled some patients who did not meet its own eligibility criteria for the study, and hired unqualified investigators. Source: Associated Press/Philly.com 8/19/09

Cerezyme Rival Available in Trials

For the second time in two weeks, federal regulators moved recently to counter the ongoing supply shortage of Cerezyme, Genzyme Corp.’s treatment for Gaucher disease, by letting doctors prescribe a rival drug before it has won approval for commercial use. Protalix BioTherapeutics of Israel in August said that the Food and Drug Administration (FDA) approved a treatment protocol allowing the company to make its drug available on a wider basis in clinical trials. Protalix is set to complete its trials of the drug in September and by year’s end file an application to market the product, called prGCD. Earlier in August, the FDA approved a similar plan for velaglucerase alfa, another rival Gaucher disease treatment that is in clinical trials. That treatment was developed by British drug maker Shire PLC’s human genetic therapies division. Source: Boston Globe 8/18/09

Tests Begin on Drugs that May Slow Aging

It may be the ultimate free lunch--taking a drug that tricks the body into thinking it is on a calorically restricted diet that yields freedom from disease and an extended healthy life span, all without eating one fewer calorie. It sounds too good to be true, and maybe it is, yet such drugs are now in clinical trials. Even if they should fail, their development represents a new optimism among research biologists that aging is not immutable, that the body has resources that can be mobilized into resisting disease and averting the adversities of old age. This optimism, however, is not fully shared; evolutionary biologists have strong reasons to suppose that human life span cannot be altered in any quick and easy way. Source: New York Times 8/18/09

White House Deal with Drug Firms Draws Flak

An $80 billion deal with the drug industry that the White House thought would add momentum to its campaign for national healthcare reform has instead provoked a political tempest, frustrating and bewildering some of the president's most important allies. As complaints rolled in, the administration offered varying, sometimes contradictory explanations of the deal. Under the deal, the Pharmaceutical Research and Manufacturers of America agreed to provide $80 billion in cost savings over 10 years. Source: Los Angeles Times 8/14/09

Abbott Expanding Study of its Popular Stent

Abbott Laboratories Inc. is expanding a study of Xience V, its top-selling heart stent, by more than 2,000 patients, who may also be put in a second, landmark stent study of more than 25,000 patients meant to find the best way to prevent potentially fatal blood clots. The bigger study aims to determine the optimum time people should take blood-thinning medicines after they undergo angioplasty to clear out a blocked artery and implant a stent; its first patients are scheduled to be enrolled this fall. The large study was designed and is being overseen by the Harvard Clinical Research Institute, and involves the four big U.S. stent makers--Johnson & Johnson, Boston Scientific Corp., Abbott, and Medtronic Inc.--plus four large drug makers that sell anticlotting medicines--Bristol-Myers Squibb Co., sanofi aventis SA, Eli Lilly & Co., and Daiichi Sankyo. Source: Associated Press/Yahoo! News 8/13/09

Drug Chief at the FDA is Accused of Conflict

The inspector general of the Department of Health and Human Services is investigating a conflict-of-interest allegation involving the official in charge of drug approvals at the Food and Drug Administration (FDA), the agency said. The investigation of Janet Woodcock, the director of the FDA's Center for Drug Evaluation and Research, stems from an ethics complaint filed by Amphastar Pharmaceuticals Inc., a California company that says it has been delayed in its six-year effort to win approval for a generic version of Lovenox, a multibillion-dollar blood thinner. In its complaint, Amphastar alleges that its competitor had special access to Woodcock at critical times in the prolonged approval process, which is ongoing. Source: Wall Street Journal 8/12/08

FDA Medical Device Regulator Resigns

Daniel Schultz, the U.S. Food and Drug Administration's (FDA's) top medical device regulator, said on August 11 that he is resigning. The departure follows internal dissent over device approval decisions that the regulator's critics said were too friendly to industry. A group of nine device division employees wrote to the House Energy and Commerce Committee last October to complain that the division's leaders had approved devices despite formal safety and efficacy concerns raised by FDA. They also alleged some scientists who objected to the decisions faced retaliation from leaders of the device division. Source: Wall Street Journal 8/12/09

U.S. Rules Aim to Help Dying Patients Access Drugs

The U.S. government finalized rules on August 12 meant to make it easier for seriously ill patients to gain access to unapproved medicines when they have run out of other options. The regulations spell out more clearly which patients are eligible for special access to experimental medicines, the Food and Drug Administration (FDA) said. They also clarify when manufacturers can charge patients for the drugs. The Abigail Alliance for Better Access to Developmental Drugs, an advocacy group, said the FDA needs to go further to significantly boost access. Source: Reuters/Yahoo! News 8/12/09

Volunteers Lining Up for Swine Flu Vaccine Test

Hundreds of Americans in eight cities are lining up for experimental swine flu shots in a race to get a vaccine out in case the new flu virus regains strength this fall and winter. Also under way are separate studies by five flu vaccine manufacturers under contract with the government. Participants will be given different combinations of two swine flu vaccines made by drug makers Sanofi Pasteur and CSL Limited and a seasonal flu vaccine. The study sites are at Baylor College of Medicine in Texas, Children's Hospital Medical Center in Cincinnati, Emory University, Group Health Cooperative in Seattle, St. Louis University, University of Iowa, University of Maryland School of Medicine, and Vanderbilt University. Source: Associated Press/Yahoo! News 8/11/09

Adaptive Evolution: Once-Rare Type of Clinical Trial is Taking Off

Adaptive trial design goes against the sacred rule of the randomized double-blind, placebo-controlled trial: In order to avoid influencing the trial’s outcome, no one should know who is getting which treatment. However, in an increasingly common approach, a trial can be altered in various ways while it is still in progress, based on a peek at interim data—which of necessity means unblinding the data before the trial’s completion. In the past few years, pharmaceutical companies have adopted the approach in full force. Too often, critics caution, companies wrongly assume the approach is an easy fix to common clinical trial woes when, in fact, the changes can ultimately cost time and money, not save them. Source: The Scientist August 2009

Opinion: Clinical Trials, Wrapped in Red Tape

Sally Satel, a psychiatrist and resident scholar at the American Enterprise Institute, writes, "It’s Christmas in August for hopeful scientists. The National Institutes of Health is now sending out its annual 'priority scores,' the indicators of whether grant requests will likely receive financing from the agency. And hearts are beating faster than ever, as the federal stimulus package has poured an additional $8.2 billion into the institutes’ budget specifically for research. However, for those grant-winners whose studies will involve human volunteers, another big hurdle remains: federal ethics regulations. No one denies the need to shield human subjects from undue risk. But current regulations have become so stringent and unwieldy that the ethics oversight system often impedes the kind of careful research we should be promoting." Source: New York Times 8/7/09

Sanofi Pasteur Starts Testing Swine Flu Vaccine

French drug maker Sanofi Pasteur began human trials of its swine flu vaccine in early August in about 2,000 people in the United States, becoming the third company conducting clinical trials on swine flu vaccines. The company makes about 40 percent of the world's flu vaccine, and has applied to the U.S. Food and Drug Administration for fast-track licensing for the vaccine. Meanwhile, Swiss pharmaceutical Novartis AG said that it had started testing its swine flu vaccine in about 6,000 people in Britain, Germany, and the U.S. Last month, Australian company CSL also began testing its vaccine in Australia. Source: Associated Press/Yahoo! News 8/7/09

Contract Research on the Rise

The global economic crisis has got most businesses pinching pennies just to stay afloat, and the drug development industry is no exception, but at least one area of the biotechnology sector--contract research organizations (CROs)--is on the rise. Data from the past two years show an annual growth rate of nearly 17 percent. Last year, despite the economic recession, the CRO market grew to nearly $20 billion--29 percent of the $74 billion drug development budget. The number and depth of the connections between CROs and drug developers has grown so much, they now have their own matchmaking website; finding partnerships, which used to take four to six weeks, can now take as little as two days, its maker claims. Source: The Scientist 8/5/09

Sometimes it Takes an Experiment to Make You Well

At any given time, according to ClinicalTrials.gov, more than 70,000 clinical trials are going on worldwide. There is an excellent chance that you will be faced with a decision about whether to participate in clinical research or a clinical trial at some point in your life. The choice you make may be for yourself or for a loved one, such as your child or a parent who has lost decision-making capacity. The trial may involve a drug, surgery, or new device. Or it could be testing a new method of diagnosis. There might also be studies that compare available marketed drugs to see which is most beneficial. In the end, your decision comes down to a simple question: Is the uncertainty of the trial and treatment you’ll undergo worth pursuing over established options? The answer will not always be clear. Source: Parade 7/26/09

Novartis Starts Testing Swine Flu Vaccine

Swiss drug maker Novartis has begun injecting its swine flu vaccine into people in the company's first human tests, a spokesman said on August 5. The vaccine is being tested in a year-long trial of 6,000 people of all ages in Britain, Germany, and the United States, and will likely be on the market before the trial finishes. A person in Britain became the first to get the swine flu vaccine, about 10 days before Novartis made the announcement. Meanwhile, Sanofi-Pasteur, which makes about 40 percent of the world's flu vaccines, said that it expected to start testing its swine flu vaccine within days in the U.S. and Europe. Source: Associated Press/Yahoo! News 8/5/09

U.S. Stops Viagra Trial Against Sickle Cell

The U.S. National Institutes of Health said in late July that it had stopped a trial of the drug sildenafil, saying it caused serious medical problems in sickle cell patients. The trial was looking at the safety and effectiveness of the Pfizer drug for pulmonary hypertension in patients with sickle cell disease, the National Heart, Lung, and Blood Institute (NHLBI) said. Patients without sickle cell disease taking the drug—which is approved under the Revatio brand name for pulmonary hypertension—are not at risk, the NHLBI said. Sildenafil also is sold under the name Viagra as a treatment for erectile dysfunction or impotence. The researchers found that 38 percent of patients taking Revatio had serious adverse effects, such as painful sickle cell "crises," compared to 8 percent of patients getting a placebo. "No deaths have been associated with the drug in the clinical trial," the NHLBI said. Source: Reuters/Yahoo! News 7/28/09

Lobbyists Battle Over Drug Sales

As Congress struggles with a massive healthcare overhaul, several lobbying powerhouses—including the pharmaceutical industry and the nation's largest advocacy group for retirees—are locked in a contentious fight over the future of biotechnology drugs. Both sides have spent heavily to sway lawmakers in the debate over how long to keep the expensive drugs exempt from generic competition. President Obama is pushing for seven years of exclusivity as he looks to trim costs to help pay for his healthcare plan—five years less than what the industry wants. Source: USA Today 7/28/09

Europe Fast-Tracking Swine Flu Vaccine

In a drive to inoculate people against swine flu before winter, many European governments say they will fast-track the testing of a vaccine, arousing concern among some experts about safety and proper doses. The European Medicines Agency is accelerating the approval process for swine flu vaccine, and countries such as Britain, Greece, France, and Sweden say they'll start using the vaccine after it's greenlighted—possibly within weeks. In Europe, flu vaccines are usually tested on hundreds of people for several weeks or months, to ensure the immune system produces enough antibodies to fight the infection. However, to ensure swine flu vaccine is available as soon as possible, the agency is allowing companies to skip testing in large numbers of people before the vaccine is approved. Source: Associated Press/Yahoo! News 7/26/09

More Cost Cuts Sought From Drug Industry

The pharmaceutical industry has remained relatively unscathed so far in Washington's effort to overhaul the nation's healthcare system, but it is too soon for drug makers to declare victory. Despite the much publicized 10-year, $80 billion cost-saving promise the drug industry made to President Obama and the chairman of the Senate Finance Committee in June, some House leaders do not think the drug makers have given enough. Chief among the Congressional critics of the drug industry is Henry A. Waxman, chairman of the House Energy and Commerce Committee. For starters, Waxman wants the drug industry to give up an additional $63 billion over the next decade. Source: New York Times 7/23/09

Researchers to Implant Pig Cells in Diabetics

A New Zealand biotech company began a trial in July of an experimental treatment for diabetes in which cells from newborn pigs will be implanted into eight human volunteers. Living Cell Technologies hopes the cells may be able to delay the effects of Type 1 diabetes, including blindness, premature coronary illness, and limb amputation resulting from poor blood circulation. Some scientists have warned that implanting pig cells has risks. Others say it is too soon to begin testing on humans because no animal trials were conducted. Source: Associated Press/Yahoo! News 7/23/09

Vaccine Shows Promise Against Typhoid Fever

A typhoid vaccine proved effective in the slums of India, where it not only helped prevent infection in children who received it, but also those in close contact who were unvaccinated, a new study found. Researchers recruited 37,673 slum-dwellers aged 2 years or older in Calcutta. Study participants randomly received a dose of the typhoid vaccine or an inactivated hepatitis A vaccine and were followed for two years. Overall, the vaccine was 61 percent effective. Protection was higher (80 percent) among immunized young children. Source: Associated Press/Yahoo! News 7/22/09

Government Calls for Volunteers to Test Swine Flu Shots

The race is on: The U.S. government and vaccine makers are seeking thousands of volunteers, from babies to the elderly, to roll up their sleeves for the first swine flu shots—to test whether a new vaccine really will protect against this novel virus before its expected rebound in the fall. On July 22, the National Institutes of Health tapped a network of medical centers around the country to begin a series of studies, with the first shots to go into the arms of healthy adults, of any age, in early August. If there are no immediate safety concerns, such as allergic reactions, testing quickly would begin in children as young as 6 months. Source: Associated Press/Yahoo! News 7/22/09

Epix Pharmaceuticals is Latest Biotech to Fall

Epix Pharmaceuticals Inc. has become the second Massachusetts biotechnology company in the past week to fold, raising fresh concerns about the health of the sector. The cash-starved company, which had been working on treatments for Alzheimer’s disease and other ailments, on July 21 confirmed it was moving ahead with "an orderly liquidation" of its assets after failing to find an acquirer or obtain additional financing to stay afloat. The week before, Biopure Corp. filed for Chapter 11 bankruptcy protection. Biopure, which was developing a human blood substitute, plans to sell most of its remaining assets to OPK Biotech LLC of Delaware. Source: Boston Globe 7/22/09

Costly Drugs Known as Biologics Prompt Exclusivity Debate

A bitter Congressional fight over the cost of superexpensive biotechnology drugs has come down to a single, hotly debated number: How many years should makers of those drugs be exempt from generic competition? The trick is to allow competition without undermining the financial incentives the pharmaceutical industry needs to undertake the risky job of developing the next drugs for cancer and other diseases. Trade groups call for an exclusivity period that would last 12 to 14 years from the time the Food and Drug Administration approves a drug for sale. However, consumer groups, insurers, employers, and generic drug companies say anything more than five years would eviscerate any potential savings from the new competition. Source: New York Times 7/22/09

Australia Starts First Swine Flu Vaccine Trials

The world's first human trials of a swine flu vaccine have begun in Australia, drug company officials said on July 22, with the aim of controlling the virus that has so far killed more than 700 worldwide. Two biotechnology companies have started injecting adult volunteers in the southern city of Adelaide with their vaccines. Adelaide-based Vaxine began trials Monday with 300 subjects, and Melbourne's CSL has 240 people in its seven-month trial, which started Wednesday. The companies say their trials are the first tests of a swine flu vaccine on humans. Medical experts warned against rushing the vaccines through trials. Source: Associated Press/Yahoo! News 7/22/09

Rx for Change (Legislation): "Comparative Effectiveness" in Healthcare Debated 

Earlier this year, President Obama and Congress decided to spend more than a billion healthcare dollars for something known as "comparative effectiveness" research. The research involves side-by-side studies to determine which medical treatments work best. The findings help policymakers decide which treatments are most effective for each healthcare dollar spent. Yet as Congress struggles to craft a healthcare overhaul plan that will cut costs, lawmakers on both sides of the aisle are balking at using comparative effectiveness to limit coverage or reimbursements. Source: National Public Radio 7/21/09

Rx for Change (Patients): No Easy Answers on Health Effects Of Comparing Care

The Obama administration and Congress are moving forward with plans to evaluate the strengths and weaknesses of the various medical treatments for common health conditions, despite concerns of some lawmakers and the drug and device industry that it will lead to rationed healthcare. But how the government uses this comparative effectiveness research and how it might benefit—or complicate—the decision-making of average people and their doctors are also matters of some debate. Source: National Public Radio 7/21/09

Lupus Drug Succeeds in Trial

Human Genome Sciences Inc. said on July 20 that its experimental lupus drug had succeeded in a late-stage clinical trial, shocking many who had written the product off. The results showed that 57.6 percent of patients who took a high dose of the drug, Benlysta, in a 52-week trial—the first of two requested by U.S. regulators—experienced an improvement in their symptoms, compared to 43.6 percent who took a placebo. The result was statistically significant and met the main goal of the clinical trial. The drug is not expected to treat all patients, but is aimed at a subgroup who have mild-to-moderate disease, because an earlier trial failed to meet its main goal. However, a subset of patients did very well, and the Phase III trials were designed in conjunction with the U.S. Food and Drug Administration to test those patients most likely to benefit from the drug. Source: Reuters/Yahoo! News 7/20/09

South Africa Stops Funding for AIDS Vaccine Research

South Africa launched a high-profile AIDS vaccine trial on 36 healthy volunteers on July 20, yet its leading scientist says the government has halted its support for the research project. Professor Anna-Lise Williamson, of the University of Cape Town, said that South Africa's Department of Science and Technology has decided to stop funding her research, and that the utility Eskom's contract for funding ended last year and was not renewed. "For vaccine development presently, the South African AIDS Vaccine initiative has no money," she said as the trial to test the vaccine's safety began with an event attended by American health officials who gave technical help. The vaccine is manufactured at the U.S. National Institutes of Health. Williamson's vaccine also is being tested in a trial of 12 volunteers in Boston that began earlier this year. Source: Associated Press/Yahoo! News 7/20/09

Fight for Swine Flu Vaccine Could Get Ugly

An ugly scramble is brewing over the swine flu vaccine—and when it becomes available, Britain, the United States, and other nations could find that the contracts they signed with pharmaceutical companies are easily broken. Experts warn that during a global epidemic, which the world is in now, governments may be under tremendous pressure to protect their own citizens first before allowing companies to ship doses of vaccine out of the country. That does not bode well for many countries, including the United States, which makes only 20 percent of the flu vaccines it uses, or Britain, where all of its flu vaccines are produced abroad. Source: Associated Press/Yahoo! Health 7/16/09

FDA Debars Two Clinical Investigators

More than six years after being convicted of fraud, two medical researchers were recently barred from working as clinical investigators by the U.S. Food and Drug Administration (FDA). The two researchers, Mary Sawaya of Ocala, Fla., and Dr. Allyn Norman of Williamsville, N.Y., were convicted in unrelated cases. Sawaya, who was convicted in 2003 of the felony offense of making a false statement to a federal agency, was permanently debarred. Norman was debarred for five years, resulting from a 2002 conviction of the misdemeanor offense of failure to maintain adequate and accurate records relating to the use of investigational new drugs. Source: CenterWatch 7/15/09

Government Can Take the Life Out of Drug Ads

Writing in the Los Angeles Times, business columnist Dan Neil notes, "Big Pharma experienced acute dizziness and irritability last month when House Ways and Means Chairman Charles B. Rangel (D-N.Y.) proposed rescinding the tax deduction for direct-to-consumer prescription drug advertising. ...I, for one, do not fear for the drug ad tax deduction. The pharmaceutical and health products industry disgorged more than $234 million upon Capitol Hill last year.... I'm sure Big Pharma's voice will be heard. And yet there are darker clouds on the horizon for direct-to-consumer drug ads, in the form of new rules about the presentation of 'risk information'.... Not only would advertisements have to fully explicate serious side effects, the nature of adverse reactions, the risk of dependence, dangerous drug interactions, and so on, but all of that would also have to be communicated in the most direct, unambiguous, and, if you will, artless form possible." Source: Los Angeles Times 7/14/09

If Red Wine's Good, are Resveratrol Pills Even Better?

In August 2003, when scientists first revealed the life-extending powers of trans-3,4,'5-trihydroxystilbene—also known as resveratrol—its earthly form had all the allure of an apple in the garden of Eden. Red wine can deliver as much as 1.5 mg of the plant compound resveratrol per four-ounce serving. Today, remarkable claims for resveratrol supplements abound, but the business of selling the supplement touted as an "anti-aging miracle" rests on a foundation of science that is as unstable and incomplete as it is promising. In fact, the marketing frenzy surrounding resveratrol is a prime example of how science can be distorted when it is mingled with hope, amplified for buzz, and spun for profit. A dozen human trials on resveratrol (or on drugs derived from it) are currently under way or have recently been completed; only two are in the third and final phase of testing, in which an agent's effectiveness in treating or preventing a specific disease is measured. Source: Los Angeles Times 7/13/09

FDA Clears Eli Lilly's Blood Thinner Effient

The Food and Drug Administration (FDA) on July 10 approved a highly anticipated blood thinner from Eli Lilly, although the drug must carry the agency's sternest warning because of its bleeding risks. The approval makes Lilly's Effient (prasugrel) the first real competition to the blood thinner Plavix, the world's second-best selling medication made by sanofi-aventis and Bristol-Myers Squibb. The FDA delayed its decision on Effient multiple times during an 18-month review, as agency staffers weighed the drug's benefits versus its risks. A study of more than 13,000 patients conducted by Lilly found that Effient prevents more heart attacks than Plavix, but also causes more internal bleeding. Lilly developed Effient with Japanese drug maker Daiichi Sankyo Co. The two companies will share revenue. Source: Associated Press/Yahoo! News 7/10/09

Omega-3 No Match for Alzheimer's, Study Finds

Omega-3 fatty acid supplements did nothing to slow memory declines in people with mild-to-moderate Alzheimer's disease, but a study in healthy people with slight memory complaints did show promise, U.S. researchers said in July. The findings from an 18-month, government-backed study suggest taking supplements of docosahexenoic acid, or DHA—an omega-3 fatty acid—does not arrest Alzheimer's in people who have already developed the mind-robbing disease. "These trial results do not support the routine use of DHA for patients with Alzheimer's," Dr. Joseph Quinn of Oregon Health and Sciences University in Portland, who led the study, said in a statement. But a six-month company study that looked at people whose memory was slipping just a bit found Martek Biosciences Corp.'s DHA supplements helped restore some of the mental acuity they had lost. Taken together, the findings, along with other studies, suggest treating Alzheimer's must begin early in the disease process, before sticky amyloid plaques begin forming toxic clumps in the brain. Source: Reuters/Yahoo! News 7/12/09

Final Rules Out for Government Stem Cell Research

The government issued final rules on July 6 expanding taxpayer-funded research using embryonic stem cells, easing scientists' fears that some of the oldest batches might not qualify and promising a master list of all that do. The National Institutes of Health said it deems those old stem cell lines eligible for government research dollars if scientists can prove they met the spirit of new ethics standards. Any newly made stem cells must come with documentation that the woman or couple who donated the original embryo gave full informed consent. Source: Associated Press/Yahoo! News 7/6/09

Experiment Seeks to Head Off Type 1 Diabetes

A bold quest has begun to create a vaccine-like therapy for Type 1 diabetes. One research effort at Children's Hospital of Pittsburgh has reached an early experimental stage: The vaccine approach is being tested to be sure that it is safe in humans before moving on to kids newly diagnosed with this deadliest form of diabetes. Scientists increasingly hope to control Type 1 diabetes by curbing the rogue immune cells that cause it, before patients become completely dependent on daily insulin injections to survive. The novel Pittsburgh vaccine--made from patients' own blood--is among a handful of possible immune therapies being tested around the country. Source: Associated Press/Google 6/30/09

Drug Firms' Medical Staffs Say What Salespeople Can't

Pharmaceutical companies are barred by the Food and Drug Administration (FDA) from promoting unapproved drug uses, called off-label use, but they are using employees called "medical science liaisons," who are often physicians and pharmacists, as a legal way to discuss those uses. Such liaisons are considered medical staff rather than sales staff, and have greater freedom than salespeople as they visit doctors' offices to discuss the science behind a medicine, including unapproved uses. The FDA permits drug companies to respond to unsolicited requests for information from doctors, including off-label data, if they provide truthful, nonpromotional material. Source: Wall Street Journal 6/26/09

U.S. Swine Flu Cases May Have Hit 1 Million

Swine flu has infected as many as 1 million Americans, U.S. health officials said in late June, adding that 6 percent or more of some urban populations are infected. The estimate was no surprise to the experts who have been closely watching the virus. The number is from mathematical modeling, based on surveys by health officials. Regular seasonal flu sickens anywhere from 15 million to 60 million Americans each year. The United States has roughly half the world's swine flu cases. More than 25 million doses of seasonal flu vaccine and as many as 60 million doses of swine flu vaccine could be available by September. Source: Associated Press/Yahoo! News 6/26/09

Groups Urge FDA to Release Information On Rejected Drugs

The U.S. Food and Drug Administration (FDA) should make more information available to the public, even on drugs and devices that never make it to the U.S. market, consumer advocates told the health agency in late June. However, industry representatives cautioned that findings or data containing confidential company information could harm competition if made widely available. While the FDA often provides public details on products that win its approval, doctors and consumers could benefit from similar disclosure on those it rejects, several advocates and former FDA staff reviewers said at a public meeting to discuss ways the agency can make its regulatory decisions more clear. Source: Reuters/Yahoo! News 6/24/09

Venezuela Set to Tear Up Pharma Patents

Venezuela has announced plans to invalidate the patents on a number of medicines and allow local firms to produce cheap generic copies of them, under a reform of the country’s intellectual patent laws. The proposals were announced on national television by the Minister of Trade, Eduardo Saman, who said the decision had been taken because the needs of Venezuelan patients with diseases such as cancer and HIV/AIDS had to take precedence over the interests of powerful drug makers. Source: PharmaTimes 6/23/09

German Research Points Way to Better Anxiety Drug

German scientists believe they may have found a better anxiety drug that can counteract panic attacks without the side effects caused by existing treatments like Valium. The new compound, XBD173, appears to avoid the problems of other treatments by targeting a different biological "don't panic" button, making it a good potential candidate for use as a safe and fast-acting anxiety pill. Researchers published details of their experimental drug in the journal Science, including encouraging results from an initial Phase I clinical study, which was sponsored by Swiss drugmaker Novartis. Source: Reuters/Yahoo! News 6/18/09

Zicam Not Alone in Side Effect Reports

The unsettling little secret of Zicam Cold Remedy finally spilled out. Though widely sold for years as a drug for colds, it was never tested by federal regulators for safety like other drugs. And that was perfectly legal--until scores of consumers lost their sense of smell. One little word on Zicam's label explains all this: "homeopathic." An Associated Press analysis of the Food and Drug Administration's side effect reports found that more than 800 homeopathic ingredients were potentially implicated in health problems last year. Complaints ranged from vomiting to attempted suicide. Source: Associated Press/Yahoo! News 6/17/09

Disease of Rich Extends its Pain to Middle Class

The rising tide of gout--an extremely painful arthritis of the big toe and other joints--is leading the pharmaceutical industry to rediscover what it had considered a disease of the past. Companies are now racing to improve upon decades-old generic drugs that do not work well for everyone. Already this year, the Food and Drug Administration has approved the first new gout drug in more than 40 years, another new drug is under review, and several others are being tested in clinical trials. Various studies suggest that the number of gout cases in this country has as much as doubled in the last three decades. Source: New York Times 6/13/09

WHO Declares First 21st Century Flu Pandemic

The World Health Organization (WHO) declared an influenza pandemic on June 11 and advised governments to prepare for a long-term battle against an unstoppable new flu virus. The United Nations agency raised its pandemic flu alert to phase 6 on a six-point scale, indicating the first influenza pandemic since 1968 is under way. Countries from Australia to Chile to the United States are reporting that the new swine flu virus is "crowding out" seasonal flu, becoming the predominant influenza strain. The virus disproportionately makes younger people sick. Some 57 percent of U.S. cases were among people aged 5 to 24, and 41 percent of those hospitalized were in this younger age group. Source: Reuters/Yahoo! News 6/12/09

Novartis Produces First Batch of H1N1 Vaccine

Swiss pharmaceuticals company Novartis AG said on June 12 that it had successfully produced a first batch of swine flu vaccine weeks ahead of expectations. WHO says drug makers will likely have vaccines approved and ready for sale after September. Novartis said it would use the first batch of vaccine for preclinical evaluation and testing. It is also being considered for clinical trials, the company said. Source: Associated Press/Yahoo! News 6/12/09

Study Suggests Drug is Safe for Morning Sickness

For the first time, a large study shows that pregnant women who suffer morning sickness are not risking harm to their babies if they take a certain antinausea drug. The result may lead more doctors to prescribe the drug metoclopramide and women to feel less guilty about using it during their baby's crucial first few months of development, experts said. Currently, no drugs are approved in the U.S. for morning sickness. The study looked back at nearly 82,000 births in Israel, where metoclopramide is commonly used. It found no difference in birth defects or other problems in newborns of women whether or not they took the drug, sold as Reglan and in generic form. Source: Associated Press/Yahoo! News 6/10/09

$2.5 Billion Later, No Alternative Medicine Cures

Ten years ago, the government set out to test herbal and other alternative health remedies to find the ones that work. After spending $2.5 billion, the disappointing answer seems to be that almost none of them do. Echinacea for colds. Ginkgo biloba for memory. Glucosamine and chondroitin for arthritis. Black cohosh for menopausal hot flashes. Saw palmetto for prostate problems. Shark cartilage for cancer. All proved no better than placebos in big studies funded by the National Center for Complementary and Alternative Medicine. The lone exception: ginger capsules may help chemotherapy nausea. As for therapies, the government is funding studies of purported energy fields, distance healing, and other approaches that have little if any biological plausibility or scientific evidence. Source: Associated Press/Yahoo! News 6/10/09

Government Probes Chelation-Heart Disease Study

Heart attack survivors are again being enrolled in a controversial federal study of an alternative treatment while the government investigates whether they were told enough about possible health risks. The $30 million study, with 1,500 participants so far, is one of the largest alternative medicine experiments ever launched. It tests high doses of vitamin and mineral supplements and chelation, a treatment used for lead poisoning that has not been proved safe or effective for heart disease. Researchers suspended enrollment last August, when the federal Office for Human Research Protections began a probe into whether the people in the study were being fully informed of risks and adequately protected. Source: Associated Press/Yahoo! News 6/10/09

FDA Releases List of Potential Drug Risks

The Food and Drug Administration (FDA) on June 4 listed two dozen drugs that it is at an early stage of reviewing for potential safety problems. The agency is checking the smoking cessation drug Chantix for possible risk of accidental injury, vision impairment, and other issues, and sleep disorder drugs Nuvigil and Provigil for a potential of serious skin reactions. Other drugs listed included orlistat, a weight-loss drug that is under evaluation for liver toxicity, and overactive bladder drug Detrol, under investigation for reports of a serious skin reaction. The FDA posted the list here. Source: Reuters/Yahoo! News 6/4/09

South Africa Leads Hunt for TB Vaccine

Trials for a new vaccine for tuberculosis (TB) start in South Africa in July. Scientists across the world are seeking a new vaccine against the contagious lung disease, which is largely under control in developed countries but still haunts the poor in the developing world. Up to one in three people have it globally. With more resources and research committed by international and philanthropic organizations, optimism for a new TB vaccine has reached heights last seen when the current TB vaccine was developed in the 1920s. Source: Reuters/Yahoo! News 6/4/09

Drug Agency May Reveal More Data on Actions

The Obama administration has announced that it is setting up a task force within the Food and Drug Administration (FDA) to recommend ways to reveal more information about FDA decisions, possibly including the disclosure of now secret data about drugs and devices under study. “Many people have been harmed over the last decade because the FDA has treated clinical trial results of drugs and devices as trade secrets,” said Dr. Steven Nissen, a cardiologist who has campaigned for the release of such information. Source: New York Times 6/2/09

AstraZeneca, Merck Collaborate on Cancer Treatment

AstraZeneca PLC and Merck & Co. have said they will jointly develop a pair of drugs meant to hit cancer with a one-two punch, part of a growing trend of combination cancer treatments. The companies said they will combine efforts with Merck's MK-2206 and AstraZeneca's AZD6244, both of which are still in very early development. The drug candidates are currently in separate early-stage and mid-stage studies involving people. Under the deal, the companies will jointly evaluate the combination in a Phase I clinical trial. Source: Associated Press/Yahoo! News 6/1/09

Deep Brain Stimulation is Expanding its Reach to New Patients

Until recently, deep brain stimulation was approved in the U.S. only to treat certain movement disorders, but now use of the technique seems set to mushroom. This year, the FDA granted a humanitarian device exemption for the treatment to be used in severe cases of obsessive-compulsive disorder. Large clinical trials are also in the works for cases of epilepsy and depression, and experimental studies are exploring the treatment for obesity, traumatic brain injury, severe chronic pain, Alzheimer's disease, anorexia, tinnitus, and addiction. Source: Los Angeles Times 6/1/09

Immune Therapies Finally Working Against Cancer

After 30 years of false starts, doctors apparently are finding success with a new way to fight cancer: using the immune system via cancer vaccines. At a conference in May, researchers said one such vaccine kept a common form of lymphoma from worsening for more than a year. That is huge in this field, where progress is glacial and success with a new treatment is often measured in weeks or even days. Experimental vaccines against three other cancers—prostate, melanoma, and neuroblastoma—also gave positive results in late-stage testing in recent weeks. Source: Associated Press/Yahoo! News 5/31/09

Companies Starting Work on H1N1 Vaccine

Companies are starting preliminary work on a vaccine for the new H1N1 flu and should begin clinical trials in the coming months, but the new vaccine will not be ready for widespread use until October, U.S. health officials said in late May. In coming weeks, companies will start making "candidate lots" of vaccine for use in clinical trials to answer a number of questions, including whether one or two injections will be needed, whether vaccinating different age groups yields different results, and whether an adjuvant is needed. Source: Reuters/Yahoo! News 5/28/09

U.S. Sets Aside $1 Billion for Flu Vaccine

The U.S. government is setting aside $1 billion to launch development of an H1N1 swine flu vaccine. The money will be used both for clinical studies this summer and for the production of two bulk ingredients that will be placed in a federal stockpile, the Department of Health and Human Services (HHS) said on May 22. Novartis AG will receive $289 million, Sanofi Aventis SA will receive $191 million, and GlaxoSmithKline PLC will be given $181 million to produce H1N1 vaccine ingredients. Another $150 million will go to these manufacturers and others to produce pilot lots of vaccine and to pay for the clinical studies in which they will be used. The clinical studies will determine whether the vaccine is safe, the proper dose, and whether adjuvants are needed. Source: Wall Street Journal 5/23/09

Study: Diabetes Drug Lowers Amputation Risk

A drug originally intended to lower diabetic patients' cholesterol instead reduced their risk of so-called minor amputation by 36 percent, a new analysis of research published four years ago has found. Researchers in Australia, Finland, and New Zealand studied almost 10,000 patients aged 50 to 75 with Type 2 diabetes. About half of the patients were given fenofibrate, a drug available generically under a variety of names. The other half received a placebo. After five years, 115 patients had at least one lower limb amputation because of diabetes. The study originally aimed to see if fenofibrate prevented heart disease, but it did not. Source: Associated Press/Yahoo! News 5/21/09

New Institute to Focus on Rare Diseases

A unique new institute will look for ways to treat rare and neglected diseases and take the first and riskiest steps toward bringing new drugs to market, U.S. health officials said in May. Congress has provided $24 million a year for five years to start the Therapeutics for Rare and Neglected Diseases Program at the National Institutes of Health (NIH). The program will use taxpayer money to get drugs through the most costly and dangerous phase of development, known as the "Valley of Death" because so many fail there. It will publish details of failures as well as successes to guide other researchers, the NIH said. The NIH estimates that up to 90 percent of all potential drugs fail to make it from the lab into human volunteers for safety testing. Source: Reuters/Yahoo! News 5/20/09 See also the ACRP Wire 5/28/09

Opinion: All the World’s a Laboratory

According to Andrea Meyerhoff and Paul Lietman, both of whom are on the faculty at Johns Hopkins University School of Medicine, "Disease outbreaks like the swine flu epidemic present an unparalleled opportunity...to gain knowledge to better protect the public. The spreading H1N1 influenza...provides the chance to learn more about the drugs we are counting on using against it. ...Clinical trials could easily be set up to include patients who show up for medical attention 72 or 96 or more hours after their symptoms appear. The results could be used to plot a curve of risk over time to guide patients and doctors in the use of anti-influenza drugs. ...Data from real-time clinical trials could enable us to not only provide better care for flu patients and their families, but also better manage resources. Source: New York Times 5/20/09

WHO: No Swine Flu Vaccine Available for Months

Drug manufacturers will not be able to start making a swine flu vaccine until mid-July at the earliest, weeks later than previous predictions, the World Health Organization (WHO) said on May 19. It will then take months to produce a new vaccine. According to vaccine experts convened by WHO, swine flu virus is not growing very fast in laboratories, making it difficult for scientists to get the key ingredient they need for a vaccine, the "seed stock" from the virus, the agency reported. Source: Associated Press/Yahoo! News 5/19/09

A Long Search for a Universal Flu Vaccine

Scientists and vaccine manufacturers are hard at work on a so-called universal flu vaccine that would work against all types of flu. The goal is to provide protection for years, if not a whole lifetime, against all seasonal flu strains and pandemic strains, making flu inoculation much more like that for measles and polio. Unfortunately, a universal vaccine will not be ready soon enough to combat a possible pandemic from the new strain of swine flu that has already sickened thousands of people. The most advanced of the vaccines have been tested only in small clinical trials. It is likely to take several more years to show if the vaccines really work. Source: New York Times 5/19/09

Fighting for a Last Chance at Life

At a time when terminally ill patients have more access to medical research than ever before, many are campaigning for the chance to be treated with drugs whose safety and effectiveness is not yet known, and for which there is little consensus on how and when to provide them to dying patients. The Food and Drug Administration does not want patients to bypass clinical trials, which require that some participants receive a placebo to determine reliably whether a drug works. Some patient advocates are lobbying for laws and policies that would sanction what has become known as the “compassionate use” of experimental drugs by seriously ill patients who have run out of other options. Source: New York Times 5/17/09

GlaxoSmithKline to Begin Work on H1N1 Vaccine Soon

GlaxoSmithKline said on May 15 that it would begin work on a vaccine as soon as it received a supply of the virus from the World Health Organization (WHO). Health officials have been cautious about production of a vaccine against the so-called swine flu for fear it would interfere with production of the normal seasonal influenza vaccine. However, company officials said they would complete their production of this year's seasonal vaccine in July and could immediately switch to producing the new vaccine. The vaccine would be available four to six months after the company receives the virus seed stock. The company said it would donate 50 million doses to the WHO for developing countries. Source: Los Angeles Times 5/16/09

FDA Approves Drug for Treatment of Aggressive Brain Cancer

The U.S. Food and Drug Administration (FDA) recently approved Genentech's Avastin (bevacizumab) to treat patients with glioblastoma multiforme (GBM) when this form of brain cancer continues to progress following standard therapy. Avastin is a monoclonal antibody that inhibits the action of vascular endothelial growth factor that helps form new blood vessels. These vessels can feed a tumor and provide a pathway for cancer cells to circulate in the body. The drug was first approved in 2004 to treat metastatic cancer of the colon or rectum, and has since been approved for treatment of nonsquamous, non-small cell lung cancer and metastatic breast cancer. In two clinical trials, about 25 percent of patients with GBM responded to Avastin with an average duration of response of about four months. Source: FDA News 5/8/09

1976 Swine Flu Response Haunts Health Officials

As U.S. health officials consider rolling out a plan to inoculate the nation against swine flu in the next several months, they are haunted by the events that unfolded the last time the government stepped in to head off a surprise flu outbreak. In the fall of 1976, dozens of Americans died within 48 hours of receiving a swine flu vaccine. Two months later, the campaign was abruptly stopped: More deaths had followed and hundreds were reporting serious side effects, including paralysis. Obama Administration officials say they are keenly aware of the history. Source: Washington Post 5/9/09

Obama Seeks FDA Budget Hike, Generic Drug Fees

President Barack Obama has proposed a 19 percent hike in the Food and Drug Administration's (FDA's) budget to strengthen inspections and increase the number of generic drugs on the U.S. market. The proposal seeks $36 million from manufacturers to review generic versions of drugs, and repeats a call to give the FDA power to clear cheaper generic versions of biotechnology drugs that can be difficult to make and cost tens of thousands of dollars a year. It also includes $5 million for the FDA to develop policies to allow Americans to buy drugs approved in other countries. Source: Reuters/Yahoo! News 5/7/09

Elsevier Published Six Fake Journals

Scientific publisher Elsevier produced six publications between 2000 and 2005 that looked like peer-reviewed medical journals, but did not disclose their corporate sponsorship, the company has admitted. Elsevier is conducting an "internal review" of its publishing practices after allegations came to light that the company produced a pharmaceutical company-funded publication, the Australasian Journal of Bone and Joint Medicine, without disclosing that it was sponsored by Merck. The publication's articles and reviews often presented data favorable to Merck's products. Source: TheScientist.com 5/7/09

FDA Backs Drug that Treats Diabetes via the Brain

Some Type 2 diabetics may soon be using a drug that helps control blood sugar via the brain. The FDA approved Cycloset, from VeroScience Inc., a new version of an old drug, bromocriptine, that has been formulated to require a low, quick-acting dose taken just in the morning. Cycloset is the first drug to win FDA approval under new guidelines that require better evidence that diabetes treatments are heart-safe. In a safety study involving 3,000 diabetics, those given Cycloset had 42 percent fewer heart attacks and other cardiovascular complications than those given a placebo. Source: Associated Press/PhysOrg.com 5/6/09

Opinion: Off-Label Uses of Drugs Should be Approved for Very Ill Patients

FDA and Congress should protect physicians' and patients' right to use FDA-approved drugs for off-label uses, Richard Epstein, a law professor at the University of Chicago, wrote in a recent Wall Street Journal opinion piece. He continued, "Clinical trials sometimes give way to educated guesses that a drug approved for one kind of tumor might treat a second... In many instances the result is failure. [However], when early results from clinical trials suggest favorable results on which the FDA is unwilling to act, off-label use begins in earnest." Source: Kaiser Daily Health Policy Report 5/5/09

Was the Alarm Over Swine Flu Justified?

Like a patient suffering from a particularly tenacious case of, well, the flu, the H1N1 virus seemed to gain ground and lose it over the weekend, leaving health officials still cautious, but hopeful that the disease might be on the wane. The number of confirmed infections continues to rise, with the World Health Organization (WHO) reporting 898 infections in 18 countries as of May 3, and the Centers for Disease Control tallying 226 confirmed cases in 30 states. The continuing spread led Homeland Security Secretary Janet Napolitano to predict on Sunday that the WHO might soon raise its pandemic alert level from phase 5 to the highest stage, phase 6, which would indicate that a full flu pandemic was under way. But there have been no deaths and few serious cases reported outside Mexico--and even there, the epicenter of the H1N1 outbreak, officials reported that the spread has slowed. As labs slogged through the backlog of suspected H1N1 flu cases, the number of confirmed cases and deaths dropped precipitously, indicating that the initial outbreak that so alarmed world health officials might not have been as serious as first feared. Source: Time/Yahoo! News 5/4/09

Anemia Drugs May Raise Death Risk in Cancer Patients

Two new studies provide more evidence that drugs such as Procrit and Aranesp, often used by cancer patients to fight anemia-linked fatigue, may boost the risk of death and serious adverse events such as blood clots. These drugs, called erythropoiesis-stimulating agents, have also been associated in prior studies with increased risk of heart attack, stroke, and tumor growth. The primary argument for the continued use of these drugs is that they help reduce the number of blood transfusions some cancer patients need, while improving quality of life. However, a coauthor of one paper, Dr. Anthony Reiman, from the University of Alberta, Canada, said his team is "supporting other groups that are recommending great caution in using these drugs for cancer patients, and in routine circumstances they may not be indicated. We hope the drugs would still be made available for people for whom transfusion isn't a good option--but those are very limited circumstances." Source: HealthDay/Yahoo! News 5/1/09

Roche Drug Avastin Fails Cancer Study

Cancer drug Avastin--a key Genentech asset now wholly owned by Roche Holding AG--has failed in a major study to prevent the recurrence of colon cancer in patients who have undergone surgery. The 2,700-patient study--evaluating Avastin plus chemotherapy for treating colon cancer immediately following surgery compared to chemotherapy alone--had been viewed as risky. The clinical trial had aimed to show the drug's ability to prevent colon cancer recurrence by wiping out microscopic cancer cells that may remain in the body after tumors have been removed by surgery, known as use in the "adjuvant setting." There are further ongoing Avastin adjuvant programs in early-stage colon, breast, and lung cancers. Source: Reuters/Washington Post 4/22/09

Electronic Health Records Raise Doubt

When Dave deBronkart, a tech-savvy kidney cancer survivor, tried to transfer his medical records from Beth Israel Deaconess Medical Center to Google Health, a new free service that lets patients keep all their health records in one place and easily share them with new doctors, he was stunned by what he found. Google said in error that his cancer had spread to either his brain or spine and listed an array of other conditions that he never had. A warning announced his blood pressure medication required "immediate attention." DeBronkart eventually discovered the problem: Some of the information in his Google Health record was drawn from billing records, which sometime reflect imprecise information plugged into codes required by insurers. Google Health and others in the fast-growing personal health record business say they are offering a revolutionary tool to help patients navigate a fragmented healthcare system, but some doctors fear that inaccurate information from billing data could lead to improper treatment. Source: Boston Globe 4/13/09

FDA to Check Safety of Old Devices

Federal regulators said in April that they would ask makers of some of the riskiest medical devices to prove that their products were safe and effective. The Food and Drug Administration (FDA) has for decades failed to fix its system for reviewing categories of devices that have been on the market since before the enactment of the medical device law in 1976. Some "legacy devices" were originally allowed on the market with minimal testing, and the agency never finished the process of reclassifying them and deciding which ones needed extensive testing before new versions could be approved. The 27 different types of devices left unexamined include artificial lung membranes, external defibrillators, and various pacemaker components. Source: New York Times 4/8/09 For more information, click here.

Doctors: Thyroid Drug Can Hurt Liver, Kill Kids

A pill used for thyroid disease can cause fatal liver failure in children and should no longer be used to treat them, two doctors warn. Over the past 60 years, reports have popped up linking the use of propylthiouracil (PTU) for Graves' disease in children to liver failure, sometimes fatal or requiring a liver transplant. PTU is also a primary treatment for adults with Graves' disease, but there appear to be fewer liver complications in adults. Doctors at Yale University School of Medicine noticed the problem in children and decided to do some research and publicize the issue. They estimate that five to 10 children die each year from complications of the drug. Source: Associated Press/Washington Post 4/8/09 

FDA Statement on the Voluntary Withdrawal of Raptiva from the U.S. Market

Genentech, the manufacturer of the psoriasis drug Raptiva (efalizumab), has announced that it has begun a voluntary, phased withdrawal of the product from the U.S. market. The company is taking this action because of a potential risk to patients of developing progressive multifocal leukoencephalopathy, a rare, serious, progressive neurologic disease caused by a virus that affects the central nervous system. By June 8, 2009, Raptiva will no longer be available in the United States. Prescribers are being asked not to initiate Raptiva treatment for any new patients. Source: FDA News 4/8/09

New TB Vaccine Passes Safety Trial

An experimental tuberculosis (TB) vaccine called MVA85A proved safe in a small Phase I clinical trial. The study included 12 people with latent TB infection, which, when reactivated, can cause full-blown TB. It's believed that about a third of the world's population has this latent infection. The participants, who had no other complicating factors, such as hepatitis or HIV, were given the vaccine and then followed for a year. The researchers concluded that the vaccine was safe, did not produce any immunopathology, and was immunogenic in the patients. The findings appear in the April 15 issue of the American Journal of Respiratory and Critical Care Medicine. Source: HealthDay News/Yahoo! News 4/8/09 

Prostate Cancer Drug Shows Promise in Early Testing

Early trials of a new drug for advanced prostate cancer are showing some success, researchers report. Of the first 30 patients treated with MDV3100, 13 showed declines of more than 50 percent in the levels of chemicals in the blood that indicate the presence of the cancer. However, the tests are only in Phase I and II. The preliminary findings, by a team led by Charles L. Sawyers of Memorial Sloan-Kettering Cancer Center in New York, are reported in the April 10 edition of the journal Science. The treatment is for prostate cancer that has metastasized, or spread to other areas of the body. Source: Associated Press/Yahoo! News 4/7/09 

Firms Tied to Some Doctors Who Set Policy

Virtually all of the psychiatrists who wrote the latest clinical guidelines for how to treat depression, bipolar disorder, and schizophrenia had financial ties to drug companies, according to preliminary findings by Boston-based researchers. The study is the first to examine potential conflicts of interest in the American Psychiatric Association panels that write the treatment guidelines widely used by practitioners. The guidelines focus heavily on medications, and give relatively little attention to nondrug treatments and how and when to phase out drugs prescribed for mentally ill patients, the authors wrote. They said three common diagnoses generate some $25 billion in drug sales per year. Source: Boston Globe 4/2/09 

Heads Up, Botox

The wrinkle smoother Botox has has made billions for Allergan, but it may soon face fierce competition if the Food and Drug Administration approves Reloxin, an injectable from France that is also made from the botulinum toxin. A dermatologist who did some of the clinical trials for Botox and Reloxin recently said on NBC’s “Today” show, “It’s time that we have something that lasts a little bit longer” than Botox. However, there are no good comparative trials on Reloxin and Botox. Source: New York Times 4/2/09

Experimental Vaccine Used in Ebola Exposure Case

On March 12, a scientist accidentally pricked her finger with a needle used to inject the deadly Ebola virus into lab mice. Within hours, members of a far-flung community of virologists, biologists, and others were tensely gathered in a trans-Atlantic telephone conference trying to map out a way to save her life. Less than 24 hours later, an experimental vaccine--never before tried on humans--was on its way to Germany from a lab in Canada. Within 48 hours of the accident, the scientist was injected with the vaccine. So far, so good. If she is still healthy by April 2, she can consider herself safe. Source: Associated Press/Yahoo! News 3/27/09 

Regulators Under Fire for Oversight of Human Trials

Federal regulators are coming under heavy fire from Congress for their failure to properly oversee institutional review boards (IRBs). At a Congressional hearing on March 26, the Government Accountability Office (GAO) detailed how it set up a fake company with a fake surgical adhesive to be used in a fake clinical trial. A for-profit IRB, Coast IRB of Colorado, reviewed materials for the fake surgical adhesive and decided it was okay to allow the device to be tested in humans. The GAO also set up a fake IRB that it registered without any problems with the U.S. Department of Health and Human Services. Source: Wall Street Journal/Dow Jones Newswires 3/26/09 For more media coverage and other details on this topic, click here.

Online Age Quiz is a Window for Drug Makers

According to its makers, more than 27 million people have taken RealAge, an Internet-based test that promises to help shave years off their age. The test asks 150 or so questions about lifestyle and family history to assign a “biological age,” how young or old your habits make you. Then, RealAge makes recommendations on how to get “younger.” But while RealAge promotes better living through nonmedical solutions, the site makes its money by selling better living through drugs. Pharmaceutical companies pay RealAge to compile test results of RealAge members and send them marketing messages. Source: New York Times 3/26/09 

Study: Few U.S. Hospitals Use Digital Records

U.S. hospitals have a long way to go to join the digital age. Fewer than 2 percent have abandoned paper medical charts and completely switched to electronic health records, a new national survey found. Another 8 to 11 percent of hospitals have basic electronic systems in place where at least one department has converted to digital. The sobering findings come as the Obama administration plans to spend $19 billion to help modernize medical-record keeping systems. The Bush administration had set a goal of 2014 for converting medical records to digital. Source: Associated Press/Washington Post 3/25/09 

Opinion: Protect Our Access to Medical Research

Nobel Prize winner Richard J. Roberts, a biochemist and molecular biologist, writing in the Boston Globe, says, "If you think this is the era of e-government and transparency, it's time to think again. Hard as it is to imagine, there's a move afoot in Congress to take away the public's free online access to tax-funded medical research findings. That would be bad for medical discovery, bad for patients looking for the latest research results, and another rip-off of the American taxpayer." He's referring to scientific journal publishers who are backing legislation to rescind the mandate that researchers who accept National Institutes of Health (NIH) funding must deposit their resulting peer-reviewed articles in the freely accessible NIH PubMed Central archive. Source: Boston Globe 3/23/09 

Drug Industry Advocates Join Chorus to Split FDA

As momentum builds to rework the nation's food-safety system after a salmonella outbreak linked to peanuts, the drug industry is hoping for a happy side effect: faster approvals for new medicines. Drug industry advocates are quietly allying with some of their longtime critics pushing to split the Food and Drug Administration (FDA) into two agencies, one for food safety and one for medical products. Drug executives see a chance to speed up drug approvals that have lagged amid a drought of new products, provided their regulator is no longer distracted by high-profile food-safety breakdowns. Source: Associated Press/Yahoo! News 3/22/09 

Experts to Advise Government on Spending for Comparative Effectiveness Research

The U.S. Department of Health and Human Services (HHS) has named 15 members to a panel that will advise the government on how to spend the $1.1 billion in federal stimulus funding dedicated to comparing the effectiveness of medical treatments. The stimulus package, signed into law by President Obama in February, includes $300 million for the Agency for Healthcare Research and Quality, $400 million for the National Institutes of Health, and $400 million for HHS to support comparative effectiveness research. The panel will hold its first public meeting on April 14. Source: Kaiser Daily Health Policy Report 3/20/09 

Drug Maker Told Studies Would Aid it, Papers Say

A Harvard child psychiatrist told Johnson & Johnson that planned studies of its medicines in children would yield results benefiting the company, according to court documents. Among the documents is a slide that Dr. Joseph Biederman used in a presentation to company executives; the slide referred to a proposed trial in preschool children of the antipsychotic risperidone, stating that the trial “will support [its] safety and effectiveness...in this age group.” Much of Biederman's work has been underwritten by drug makers for whom he privately consults. An inquiry revealed last year that he earned at least $1.6 million in consulting fees from drug makers from 2000 to 2007, but failed to report all but about $200,000 of this income to university officials. Source: New York Times 3/20/09 

U.S. Drug Sales Show Anemic Growth in 2008

Sales of prescription drugs in the United States rose an anemic 1.3 percent in 2008 to $291 billion in a continuing slowdown, as patients opted for cheaper generic versions or chose to go without treatment due to the economic downturn. In previous years, U.S. prescription drug sales rose 3.8 percent in 2007 and about 8 percent in 2006--those years themselves reflecting a slowdown from annual double-digit percentage sales growth often seen in earlier decades. The worrisome snapshot comes from information supplied by IMS Health Inc, which compiles market data on the pharmaceutical industry. Source: Reuters/Yahoo! News 3/19/09

Obama Picks FDA Chief

President Barack Obama on March 14 chose public health and biological threat expert Dr. Margaret Hamburg to run the troubled U.S. Food and Drug Administration (FDA). He selected Baltimore Health Commissioner Dr. Joshua Sharfstein to serve as Hamburg's principal deputy. Hamburg is a former New York City health chief who worked on policy issues in President Clinton's health department. The choice signals the FDA's priority under the Obama administration will be safety, and not necessarily speeding through drug approvals. Source: Reuters/Yahoo! News 3/14/09 

New Debate On How to Decide Best Health Treatments

Prominent health industry and patient advocacy groups are trying to reframe the debate over how medical treatment decisions are made. Spurred by $1.1 billion in the economic stimulus bill for "comparative effectiveness research," their coalition unveiled a campaign in March to keep "comparative effectiveness" from morphing into "cost effectiveness" that would deny treatments to patients who need them. It's a concern for drug and biotech companies, too, since they could lose out if a treatment they've developed is found to be less effective than a competitor's. Source: Associated Press/Yahoo! News 3/12/09 

Legislation Introduced to Create Regulatory Path for Generic Biotech Drugs

House Energy and Commerce Committee leaders in March introduced a bill (H.R. 1427) that would permit the Food and Drug Administration (FDA) to approve generic versions of biotechnology drugs. The bill would grant generic biotech drug manufacturers regulatory approval if they demonstrate, according to FDA standards, that there are no clinically meaningful differences between a generic and the existing biotech drug. Biotech companies have suggested that all biosimilars undergo comprehensive clinical trials in order to demonstrate their safety and effectiveness. Source: Kaiser Daily Health Policy Report 3/12/09 

European Union Red Tape "Blocks Drug Trials"

Red tape is severely hampering clinical research in the U.K. and inadvertently "killing people," leading researchers have warned. European legislation introduced in 2004 has led to fewer patients enrolled in clinical trials and has caused "huge delays" in research, they said. National Health Service bureaucracy is a further hurdle to treatments being quickly assessed, a team of U.K. academics added. Despite increased funding in clinical trials in the U.K., the number of trials being approved has stayed the same since 2004. Source: BBC News 3/12/09 See also Reuters/Guardian 3/12/09

Roche Wins Genentech with $46.8 Billion Offer

Roche Holding AG has struck a deal with Genentech Inc to acquire all outstanding shares in the U.S. biotech group for $46.8 billion. Buying Genentech will give Roche control of all revenues for big-selling cancer drugs Avastin and Herceptin, as well as absorbing an attractive portfolio of new medicines. The value of the oncology business could be boosted or somewhat diluted, depending on the results of key clinical trial results for Avastin due next month. Source: Reuters/Washington Post 3/12/09 

Doctor Accused of Faking Studies

Dr. Scott S. Reuben, an anesthesiologist at Baystate Medical Center in Springfield, Mass., whose research has influenced how doctors treat surgery patients for pain, has been accused of fabricating results in at least 21 published studies and, in some cases, even inventing patients. Physicians and journal editors said the allegations, if proven, could constitute one of the largest and longest-running cases ever of medical research fraud. Source: Boston Globe 3/11/09 

Obama Decision On Stem Cells Cheers Scientists

President Barack Obama's decision to lift the contentious Bush-era restraints on stem-cell research came with a larger message for all scientists: Follow the data, not political ideology. Researchers said the new president's message was clear: Science, which once propelled men to the moon, again matters in American life. Opponents saw it differently: a defeat for morality in the most basic questions of life and death. Source: Associated Press/Yahoo! News 3/10/09 

Merck Buying Schering-Plough in a $41.1 Billion Deal

Merck & Co. is buying Schering-Plough Corp. for $41.1 billion in a deal that gives Merck key new businesses, access to a promising pipeline of new products, and the chance to further cut costs, including eliminating about 16,000 jobs. Merck hopes the cash-and-stock deal, which would vault it to the world's No. 2 drug maker, helps it better compete in a drug industry facing slumping sales, tough generic competition, and intense pricing pressures. Source: Associated Press/Yahoo! News 3/9/09 

Combination Therapy Eases Fibromyalgia Symptoms

A review of previous clinical trial results shows that a multifaceted approach can be effective for treating fibromyalgia, German researchers report. German guidelines recommend "multicomponent treatment" of fibromyalgia: patient education or psychological therapy and exercise as second-line therapy for patients whose symptoms and restrictions in daily life are not sufficiently reduced by a single therapy, such as medication. Researchers examined pooled evidence from nine clinical trials of multicomponent therapy involving more than 1,100 patients and reported the results in the medical journal Arthritis and Rheumatism. Source: Reuters/Yahoo! News 3/3/09 

Sebelius, DeParle Named to Healthcare Posts

President Obama has named Nancy-Ann DeParle as director of the White House Office of Health Reform, rounding out the leadership of the team that will direct his administration's efforts to revamp the nation's ailing healthcare system. Obama also formally named Kansas Gov. Kathleen Sebelius as his secretary of health and human services. Sebelius and DeParle will be charged with helping to craft and sell the administration's ambitious effort to revamp the nation's healthcare system and extend access to the 46 million people in the country who lack coverage, while attempting to rein in runaway costs. Source: Washington Post 3/3/09 

Harvard Medical School in Ethics Quandary

Harvard Medical School is experiencing a full-blown movement by more than 200 students and sympathetic faculty who are intent on exposing and curtailing the drug industry's influence in their classrooms and laboratories, as well as in Harvard's 17 affiliated teaching hospitals and institutes. They say they are concerned that the same money that helped build the school's world-class status may in fact be hurting its reputation and affecting its teaching. Source: New York Times 3/3/09 

Massachusetts General Hospital to Use Genetics to Personalize Cancer Care

Cancer doctors at Massachusetts General Hospital plan within a year to read the genetic fingerprints of nearly all new patients' tumors, a novel strategy designed to customize treatment. The hope is to spare patients from the traditional hit-or-miss approach to cancer care, when expensive drugs with harmful side effects are often given without knowing whether they will work. Doctors will hunt for 110 abnormalities, carried on 13 major cancer genes, that can predict whether drugs already on the shelf or in development might thwart a patient's tumor. Source: Boston Globe 3/3/09

Study: Old Drugs Might Give Tuberculosis a One-Two Punch

Scientists may have found a way to deal drug-resistant tuberculosis (TB) a one-two punch using two old, safe antibiotics. The beta-lactamase enzyme in TB bacteria renders the penicillin family of antibiotics useless, but there are different antibiotics that can block it. A combination of one, named clavulanate, to drop TB's shield, and the antibiotic meropenem to attack the bacteria, worked well in the lab. Researchers from the National Institutes of Health and New York's Montefiore Medical Center are now planning patient studies in South Korea and South Africa. Source: Associated Press/Yahoo! News 2/26/09 

Acting FDA Commissioner Lays Out Plans to Ensure Quality

Acting Food and Drug Administration (FDA) Commissioner Frank Torti, at a meeting with the agency's Science Board, presented a series of plans intended to ensure the quality of science at the organization, Dow Jones reports. Torti's plans in part are based on ongoing efforts to strengthen the agency's staff with more qualified people through a fellowship program and other initiatives. He noted FDA also is focused on launching new clinical trial concepts that would help regulators make optimal evaluations on the safety of drugs and other medical products. Source: Kaiser Daily Health Policy Report 2/26/09 

FDA Says Firm Faked Generic Drug Tests

India's largest drug maker has falsified laboratory tests for generic drugs that had been approved for sale in the United States, officials at the Food and Drug Administration (FDA) say. The FDA cited the fraudulent laboratory tests as it took the unusual step of stopping its review of all pending applications from Ranbaxy Laboratories. The FDA is not seeking a recall, because regulators do not believe the drugs pose a health risk. The affected drugs include medications for high cholesterol and an antihistamine, but the FDA would not provide a specific list. Source: Washington Post 2/26/09 

FDA Consolidates at Former Navy Site

In the great chess game of managing the government’s properties, the Food and Drug Administration (FDA) has moved into the gap left a dozen years by the closing of the 662-acre Naval Surface Warfare Center compound in White Oak, Md., a suburb of Washington, D.C., with a large-scale consolidation of its previously scattered labs and offices. The $1.15 billion project--which includes 14 new buildings totaling 3.1 million square feet of labs and offices--is expected to become the anchor of a new biotech hub just outside the Capital Beltway. Source: New York Times 2/25/09 

Outsourcing of Drug Trials is Faulted

An article by Duke University researchers about the globalization of clinical trials, published in The New England Journal of Medicine, raises questions about the ethics of increasingly conducting studies outside the United States when they are meant to gather evidence for approving new drugs in the states. The authors suggest an ethical quagmire when drugs intended for wealthy nations are tested on people in developing countries, because the volunteers may be unduly influenced with promises of compensation or free medical care for their participation. Source: New York Times 2/19/09 

Report: FDA Scaled Back Enforcement at Labs

An independent watchdog group says the government has quietly scaled back enforcement of federal quality regulations at labs that develop medical devices. The Project on Government Oversight found that the Food and Drug Administration (FDA) has dramatically reduced inspections of "good laboratory practices" at facilities that do the earliest testing of medical devices. Such inspections declined from 33 in 2005, to seven in 2007, to just one last year, according a report the group released February 18. No inspections are planned for this year, the report said. Source: Associated Press/Yahoo! News 2/18/09

Push is On to Tailor Cancer Care to Tumor's Genes

The days of one-size-fits-all cancer treatment are numbered: A rush of new research is pointing the way to tailor chemotherapy and other care to what's written in a specific tumor's genes. However, as tantalizing as this personalized medicine is, gene testing is like the Wild West. Laboratories often introduce new tests at the first clues they might work, not waiting for final proof. Few tests so far have won the backing of major medical groups, making research studies a best bet for many patients. Source: Associated Press/Yahoo! News 2/16/09

Bone Drugs May Help Fight Breast Cancer

A drug of a class commonly used to combat bone loss may reduce by a third the chance that some breast cancers will spread or recur, a large study has found. The new study, published in the New England Journal of Medicine, involved 1,803 premenopausal women with tumors that were fueled by estrogen. As part of their treatment, all received drugs that shut down their ovaries, preventing them from making estrogen, along with drugs that stymie cancer cells from using estrogen to grow. Half also got the bone drug zoledronic acid, or Zometa, as an intravenous infusion twice a year for three years. Those who took the drug had a 36 percent reduction in cancer recurrences and metastases, compared to women who did not get it. Source: New York Times 2/12/09

Charles Grassley and the FDA

It all started with an $1,800 toilet seat cover. In that 1980s defense-spending scandal, U.S. Sen. Charles Grassley found a congressional calling: creating uproars that often transform public opinion and policy. In his nearly 30 years in the Senate, the Iowa Republican has gone after the FBI, the IRS, and others. But in recent years, Grassley has repeatedly turned his sights on wrongdoing by the pharmaceutical industry and its overseer, the U.S. Food and Drug Administration (FDA). "The pharmaceutical industry feels it's got a seat at the table, whereas the only person that should have a seat at the table across from the FDA is John Q. Public," he says. Source: Philadelphia Inquirer 2/8/09

Drug Makers' Push Boosts "Murky" Ailment

Two drug makers spent hundreds of millions of dollars last year to raise awareness of a murky illness, helping boost sales of pills recently approved as treatments and drowning out unresolved questions--including whether it's a real disease at all. Key components of the industry-funded buzz over the pain-and-fatigue ailment fibromyalgia are grants to nonprofit groups for medical conferences and educational campaigns. Many doctors and patients say the drug makers are educating the medical establishment about a misunderstood illness. But critics say the companies are hyping fibromyalgia along with their treatments, and that the grantmaking is a textbook example of how drug makers unduly influence doctors and patients. Source: Associated Press/Washington Post 2/8/09

Psychiatrists May Cut Some Ties to Drug Firms

Many psychiatrists have been studying the issue of financial ties to the drug industry and have proposed that the American Psychiatric Association cut back on medical education seminars funded by drug companies. Dr. Nada Stotland, president of the group that represents 38,000 doctors, said the proposal is one of several the association's board will take up in March to address concerns that psychiatrists have become too cozy with drug makers. Several drug makers last month stopped passing out pens and other items as part of a set of voluntary ethics guidelines, but getting U.S. doctors to give up free medical education, often at fancy resorts, will be a harder sell. Source: Reuters/Yahoo! News 2/8/09

In Colon Cancer Drug Study, More Wasn't Better

Doctors thought that combining two newer drugs that more precisely attack cancer would help people with advanced colon cancer. Instead, it made the cancer worse and made the patients more miserable, a study found. The surprising findings underscore the importance of doing rigorous studies before rushing to mix these pricey, new-generation drugs, the Dutch researchers and other experts said. The doctors tried combining Erbitux and Avastin because lab tests and an earlier small study had shown promising results. "This will stand out as a warning," said Dr. Cornelis Punt, the 755-patient study's leader at Radboud University Nijmegen Medical Center. "You have to do the randomized studies to see what really happens." Source: Associated Press/Yahoo! News 2/4/09

Pfizer Agrees to Pay $68 Billion for Rival Wyeth

The board of Pfizer, the world's largest drug maker, agreed to acquire a rival, Wyeth, for $68 billion, the companies announced in January. Pfizer also said that its net income for the fourth quarter dropped 90 percent from the period a year ago, citing a charge to resolve inquires into its off-label promotional practices. In its statement, it also said that it planned to cut its work force by about 10 percent and reduce the number of manufacturing sites. The company also said that it would cut its dividend. Source: New York Times 1/26/09

New Drug Firm Limits Prompt Fears of Falloff in Medical Meetings in Boston

Boston's status as a popular destination for medical industry meetings is at risk because of new regulations restricting financial relationships between pharmaceutical companies and doctors, according to industry executives. With strict new regulations set to take effect July 1, some meeting sponsors are considering pulling out of Boston. At least two large medical conferences already have withdrawn. The state's rules say that continuing medical education courses must comply with guidelines issued by the Accreditation Council for Continuing Medical Education. Source: Boston Globe 1/24/09

New Pill May Work for MS

German drug maker Merck Serono is one step closer to releasing the first pill to treat multiple sclerosis (MS), the company said in January. In a press statement, Merck said that patients taking cladribine tablets had a nearly 60 percent lower relapse rate than those on placebo pills. The two-year study included 1,326 MS patients who were randomly divided into three groups. Two groups received different doses of cladribine and one group received fake pills. Merck has already asked American and European drug regulators to fast-track the drug to the market. Source: Associated Press/Washington Post 1/23/09

U.S. Approves First Stem Cell Study for Spinal Injury

A U.S. biotech company plans to start the world's first study of a treatment based on human embryonic stem cells this summer. The company gained federal permission in January to inject eight to 10 patients with cells derived from embryonic cells, said Dr. Thomas Okarma, president and CEO of Geron Corp. of Menlo Park, Calif. The patients will be paraplegics, who can use their arms but can't walk. They will receive a single injection within two weeks of their injury. The study is aimed at testing the safety of the procedure, but doctors will also look for signs of improvement like return of sensation or movement in the legs, Okarma said. Source: Associated Press/Washington Post 1/23/09

Gifts to Doctors Must be Disclosed Under U.S. Bill

Drug and medical device manufacturers would need to publicly disclose all doctor payments and gifts exceeding $100 per year under U.S. legislation unveiled in January. Companies would face penalties as high as $1 million for knowingly failing to report the payments if the bill by Sens. Charles Grassley and Herb Kohl becomes law. The information would be posted online for public viewing. Grassley said he is considering if reporting requirements should also apply to industry payments to medical organizations, hospitals, pharmacy benefit managers, pharmacists and pharmacies, continuing medical education groups, and medical schools. Source: Reuters/Yahoo! News 1/22/09

WHO Gets Gates Grant for Child Medicines Research

The World Health Organization (WHO) said in January it had received a $9.7 million grant from the U.S.-based Bill & Melinda Gates Foundation for research into the production and dosage of medicines for children. The research, aimed at increasing the low number of "child size" medicines available around the world and especially in developing countries, will be carried out in cooperation with the United Nations children's agency UNICEF. Results will be given to the pharmaceutical industry, a WHO statement said. Source: Reuters/Yahoo! News 1/21/09

U.K. Firm to Launch Pioneering Stem Cell Trial

A British biotechnology company, working with a team of doctors in Scotland, is to launch a pioneering clinical trial to assess whether stem cell therapy can help patients left disabled by stroke. ReNeuron Group Plc said in January it had received approval from the U.K. Medicines and Healthcare Products Regulatory Agency to commence the clinical trial using its fetal stem cells. The move is a victory for the company, which has so far failed to win approval for similar tests from U.S. regulators. It also represents a boost for Britain's position as a leader in developing stem cell treatments. Source: Reuters/Yahoo! News 1/18/09

Scotland Trial to Use Stem Cells for Blindness

A two-year trial involving 20 patients with corneal blindness was set to begin in Scotland in January at the Princess Alexandra Eye Pavilion in Edinburgh and the Gartnavel General Hospital in Glasgow. The treatment uses the stem cells of dead adult donors, rather than embryonic stem cells, and if successful could help millions of people around the world who suffer from corneal blindness, around 80 percent of whom are elderly. A similar study by the University of Pennsylvania in the United States last year found that people with inherited blindness saw dramatic improvements in their vision when a corrective gene was injected into their eyes. Source: Agence France Presse/Yahoo! News 1/19/08

High-Risk Medical Devices Escaped Close Review

Some medical devices for sensitive uses have won government approval without a close scientific review, congressional investigators said in January. Although Congress ordered the Food and Drug Administration (FDA) years ago to resolve the issue, the agency approved 228 medical devices without a full-scale review from 2003-07, the Government Accountability Office said in a report. The root of the problem seems to be that the FDA never fully carried out the intent of a decades-old change in the law. The report urged the FDA to promptly resolve the problem, either by carrying out full reviews or reclassifying some devices as lower risk, if appropriate. Source: Associated Press/Yahoo! News 1/15/09 

Heart Risk Cited in Newer Antipsychotic Drugs

A widely used class of antipsychotic drugs that includes bestsellers Zyprexa, Risperdal, and Seroquel is just as likely--perhaps even more likely--to cause a fatal heart attack as older antipsychotic drugs like haloperidol, researchers reported in January. The findings, which run contrary to a long-standing belief, add to a growing drumbeat of criticism about this class of drugs, known as atypical antipsychotics. Last week British researchers reported in the journal Lancet Neurology that Alzheimer's patients given the drugs to control aggression were nearly twice as likely to die from any cause as patients who did not receive them. Source: Los Angeles Times 1/15/09 

Love the Long Eyelashes...Who's Your Doctor?

Allergan, the company that turned Botox into a blockbuster wrinkle smoother, plans to introduce Latisse, the first federally approved prescription drug for growing longer, lusher lashes, at the end of January. Allergan held a clinical trial with about 280 volunteers, half of whom used Latisse daily for 16 weeks. The study results were reviewed by the Food and Drug Administration, which approved the drug in late December. In that study, eyelashes typically grew 25 percent longer, 106 percent thicker and 18 percent darker. The product has the same formula as Allergan’s eye drops for glaucoma, called Lumigan. Source: New York Times 1/14/09 

FDA Guidelines Allow Drug Makers to Share Unapproved Drug Uses With Physicians

U.S. Food and Drug Administration (FDA) officials on January 12 posted guidelines online that allow pharmaceutical companies to cite peer-reviewed medical journal articles to promote drugs for unapproved uses to physicians. Drug makers are not permitted to promote products for off-label uses, but FDA allows companies to distribute articles regarding unapproved uses provided they appear in a peer-reviewed journal. In addition, FDA said the new guidelines are intended to discourage ghostwriting. Source: Kaiser Daily Health Policy Report 1/13/09 

In FDA Files, Claims of Rush to Approve Devices

An FDA official overruled front-line agency scientists and approved the sale of an imaging device for breast cancer after receiving a phone call from a congressman, according to internal agency documents. The legislator’s call and its effect on what is supposed to be a science-based approval process is only one of many accusations in a trove of documents regarding disputes within the agency’s office of device evaluation. The documents show that front-line agency scientists believe that FDA managers have become too lenient with the industry. Source: New York Times 1/13/09 

FDA is Lax on Oversight During Trials, Inquiry Finds

The FDA does almost nothing to police the financial conflicts of doctors who conduct clinical trials of drugs and medical devices in human subjects, government investigators are reporting. Moreover, the investigators say, agency officials told them that trying to protect patients from such conflicts was not worth the effort. In 42 percent of clinical trials, the agency did not receive forms disclosing doctors’ financial conflicts and did nothing about the problem, according to the investigation. Source: New York Times 1/12/09 For a follow-up New York Times editorial, "Not What We Call Due Diligence," click here; see also Associated Press/Austin American-Statesman 1/12/09 

Goats May be the New Old-Fashioned Drug Makers

Goats being raised on a farm in central Massachusetts have been genetically engineered to make a human protein in their milk that prevents dangerous blood clots from forming. GTC Biotherapeutics Inc. extracts the protein and turns it into a medicine that fights strokes, pulmonary embolisms, and other life-threatening conditions, and has asked the FDA to OK the drug, called ATryn. An expert panel voted overwhelmingly in January that it is safe and effective, putting it on the verge of becoming the first drug from a genetically engineered animal to be approved in the U.S. The agency is expected to make a final decision in early February. Source: Los Angeles Times 1/10/09 

Researchers Study Breast Cancer, Bone Marrow Link with Trial

At first, Chris Gunn was reluctant to participate in a clinical trial for breast cancer that required the removal of some bone marrow, but with some words of encouragement from a close friend, she agreed to the procedure. "I decided 'Why be selfish with the potential to help other people?'" Gunn said. "It was painless and I have never regretted it." Gunn is one of more than 400 people newly diagnosed in the early stages of breast cancer who are participating in a new clinical trial being conducted by the National Surgical Adjuvant Breast and Bowel Project, in conjunction with the University of Vermont Medical Center and Intermountain Healthcare. Source: The Spectrum 1/9/09 

FDA Backs Vytorin After Finishing Study Review

The U.S. Food and Drug Administration (FDA) said in January that patients should not stop taking Vytorin or other cholesterol-lowering drugs, based on its just-completed review of a controversial study that hammered Vytorin sales. The agency said it is sticking to its original position that medicines that reduce bad cholesterol benefit patients at risk of heart attack, stroke, or sudden death. For the past year, some experts have instead been saying that reducing cholesterol levels isn't necessarily enough to reduce those risks if there's no evidence the medicines control plaque buildup in arteries. Source: Associated Press/Yahoo! News 1/8/09 

With More Oversight on the Horizon, Drug Makers Work to Polish Image

The pharmaceutical industry, confronting sluggish growth, low prestige, and the prospect of more-aggressive government oversight, is moving on several fronts to burnish its image and align itself rhetorically with the health reform goals of President-elect Barack Obama and the Democratic Congress. The industry's lobbying arm plans to spend tens of millions of dollars on an advertising blitz promoting Obama-style health coverage for every American. Beginning this month, drug companies also will voluntarily submit to a host of marketing restrictions in an attempt to preempt stricter regulations that lawmakers in both parties are pursuing. Source: Washington Post 1/8/09