In the News

The Latest News (Posted February 1, 2012)...

FDA Staffers Sue Agency Over Surveillance of Personal E-mail

The Food and Drug Administration (FDA) secretly monitored the personal e-mail of a group of its own scientists and doctors after they warned Congress that the agency was approving medical devices that they believed posed unacceptable risks to patients, government documents show. The surveillance took place over two years as the plaintiffs accessed their personal Gmail accounts from government computers. Information garnered this way eventually contributed to the harassment or dismissal of all six of the FDA employees, a new lawsuit alleges. All had worked in an office responsible for reviewing devices for cancer screening and other purposes. In 2010, the agency told the Department of Health and Human Services’ (HHS) inspector general that the scientists and doctors had improperly disclosed confidential business information about the devices, but the inspector general’s office declined to pursue an investigation, finding no evidence of criminal conduct. It also said that the doctors and scientists had a legal right to air their concerns to Congress or journalists. FDA officials sought a second time that year to initiate action against the scientists and doctors. The inspector general, after consulting with federal prosecutors, declined the second request, as well. The FDA scientists and doctors, all of whom worked for the agency’s Office of Device Evaluation, said they first made internal complaints beginning in 2007 that the agency had approved or was on the verge of approving at least a dozen radiological devices whose effectiveness was not proven and that posed risks to millions of patients. Frustrated, they also brought their concerns to Congress, the White House, and the HHS inspector general. Source: Washington Post 1/29/12

Avastin May Fight Early Breast Cancers

Surprising results from two new studies may reopen debate about the value of Avastin for breast cancer. The drug helped make tumors disappear in certain women with early-stage disease, researchers found. Avastin recently lost approval for treating advanced breast cancer, but the new studies suggest it might help women whose disease has not spread so widely. These were the first big tests of the drug for early breast cancer, and doctors were cautiously excited that it showed potential to help. In one study, just over one third of women given Avastin plus chemotherapy for a few months before surgery had no sign of cancer in their breasts when doctors went to operate, versus 28 percent of women given chemo alone. In the other study, more than 18 percent on Avastin plus chemo had no cancer in their breasts or lymph nodes at surgery versus 15 percent of those on chemo alone. A big caveat, though: The true test is whether Avastin improves survival, and it's too soon to know that—both studies are still tracking the women's health. The drug also has serious side effects. Source: Associated Press/Yahoo! News 1/25/12

Panel Sidesteps Controversy on Draft for Comparative Effectiveness Research

The Patient-Centered Outcomes Research Institute (PCORI)—created by the health law to help determine the most effective medical treatments—released its draft priorities and research agenda in January, but it did not single out any specific diseases, treatments, or procedures to study. Instead, the nongovernmental institute that will oversee billions in research funding “specifies a set of questions and topics” in five broad categories. Those are: comparing various medical options, improving the healthcare system, improving communication of research findings to patients and clinicians, addressing health disparities, and finding ways to improve research methods. Although comparative effectiveness research has been around for a long time, the new panel has drawn opposition from some Republican lawmakers who fear it will be used to ration care. By not naming specifics, PCORI sidesteps controversy that might arise from selecting certain diseases or treatments, but not others. Rather than PCORI deciding up front which diseases or treatments deserve top priority, researchers will submit questions they want to answer in their funding applications. Still, PCORI will ultimately need to select which research projects to finance. Source: Kaiser Health News 1/23/12

Embryonic Stem Cells Appear to Restore Some Vision to Legally Blind Patient

For the first time, an experimental treatment made from human embryonic stem cells has shown hints of helping someone, apparently restoring some vision for at least one and possibly two women losing their sight to incurable forms of progressive blindness. Because the women were the first to volunteer for an experiment designed primarily to test the safety of injecting an embryonic stem cell therapy into people, scientists are being cautious. However, the surprisingly quick improvement in a 51-year-old Los Angeles graphic artist whose vision was inexorably fading caught everyone involved off-guard. “This report provides the first evidence that embryonic stem cells may be safe—and that it really works,” said Robert Lanza, chief scientific officer at Advanced Cell Technology, which is sponsoring the closely watched experiment. The blindness trial is one of only two government-sanctioned attempts to try an embryonic stem cell therapy on people, and the first to document any evidence that the approach might be working. Source: Washington Post 1/23/12

U.S. Consumers Tell Insurers to Cover Experimental Drugs

When your health insurance provider denies an experimental treatment or a high-cost drug, how much are you willing to pay for the care you believe you need? Researchers at Harvard's School of Public Health recently asked residents of the United States, Britain, Italy, and Germany whether they would give up high-cost experimental treatments to decrease overall healthcare costs. The results, released in late December, showed that 62 percent of Americans "oppose decisions by the government or health insurance plans" to deny care if those entities determine that the benefits of that care do not justify its costs. Respondents in each country responded similarly. In fact, the only major difference between Americans and those surveyed in Europe was that a majority of Americans do not support a government body that decides whether programs should "pay for or provide prescription drugs and medical or surgical treatments if they think they cost too much." The survey found that "Americans have the lowest level of trust--34 percent--in national government to make the right healthcare decisions." Source: Reuters/Yahoo! News 1/23/12

Editorial: Who Else is Paying Your Doctor?

It took longer than expected, but the Obama administration is finally poised to enact badly needed regulations requiring that the manufacturers of drugs, medical devices, and medical supplies disclose all payments they make to doctors or teaching hospitals. The information, which would be posted on a government website, will allow patients to decide whether they need to worry about any possible conflicts of interest. Such payments can be for legitimate research and consulting, but there is also a lot of cash being spread around to pay for doctors’ travel and entertainment or for gifts or modest meals for a prescribing doctor’s staff. Some prominent doctors and researchers receive hundreds of thousands or even millions of dollars a year in exchange for providing advice to a company or giving lectures on its behalf. About a quarter of all doctors take some cash payments from drug or device makers and nearly two-thirds accept meals or food gifts. Analysts contend that even seemingly trivial gifts can influence doctors to prescribe expensive drugs that may not be best for a patient’s health or pocketbook. The proposed rules were issued on December 14 and are subject to comment until February 17, after which they will be revised and issued in final form. Manufacturers could be fined up to $150,000 a year for failing to report payments and up to $1 million a year for “knowingly” failing to report. The new rules should give a welcome boost to otherwise spotty efforts by some companies, medical centers, scientific journals, states, and ethical codes to eliminate, minimize, or at least disclose financial interests that might cloud medical judgments. The existence of the website could deter some questionable payments, and it could help patients decide which doctors to rely on. Source: New York Times 1/20/12

Earlier News Items...

U.S. Wants Effective Alzheimer's Treatment by 2025

The U.S. government is setting what it calls an ambitious goal for Alzheimer's disease: Development of effective ways to treat and prevent the mind-destroying illness by 2025. The Obama administration is developing the first National Alzheimer's Plan to find better treatments for the disease and offer better day-to-day care for those afflicted. A newly released draft of the overall goals sets the 2025 deadline, but doesn't provide details of how to fund the necessary research to meet that target date. A committee of Alzheimer's experts began a two-day meeting on January 17 to help advise the government on how to finalize the plan. Source: Associated Press/Yahoo! News 1/17/12

Red Wine Researcher Accused of Falsifying Data

A University of Connecticut researcher known for his work on red wine's benefits to cardiovascular health falsified his data in more than 100 instances, university officials said in January, and nearly a dozen scientific journals are being warned of the potential problems after publishing his studies in recent years. The researcher, Dr. Dipak Das, conducted some studies of resveratrol, an ingredient in red wine that has shown potential for promoting health. However, Das is not considered a major figure in the field, and the new allegations are not expected to make a material difference to resveratrol research, which is being conducted extensively around the world with encouraging results from many labs. University officials said their internal review found 145 instances over seven years in which Das fabricated, falsified, and manipulated data, and the U.S. Office of Research Integrity has launched an independent investigation of his work. Source: Associated Press/Yahoo! News 1/11/12

China Seeks to Stop Unapproved Stem Cell Treatments

China has ordered a halt to all unapproved stem cell treatments and clinical trials, state media reported in January, as Beijing seeks to rein in the largely untested stem cell therapies now on offer across the country. The Ministry of Health will stop accepting new applications for stem cell programs, a ban that will last until July and comes as China begins a one-year program to regulate the sector better, Xinhua cited a ministry spokesman as saying. A growing number of hospitals and clinics in large cities in China have been offering stem cell therapies for treatment of diseases ranging from cancer and Alzheimer's to spinal cord injuries, treatments that are backed by little or no scientific evidence and which are considered at best experimental. Source: Reuters 1/10/12

India’s Drug Trials Fuel Consent Controversy

Since India eased guidelines for conducting drug trials in 2005, the number of Indians participating has shot up to 150,000 from close to zero, as international drug companies take advantage of lower costs here. But questions about the consent process have fueled fears that many Indians are entering the trials without knowing the risks. In the wake of the recent controversies, the Indian Council for Medical Research invited public feedback on draft guidelines about compensation for injuries that occur during clinical research. “India is emerging as a hub for drug trials, and Indian patients are like guinea pigs,” said C.M. Gulhati, editor of the Monthly Index of Medical Specialities journal. “The ethical review panels are bogus. The drug control authority approves almost all the trial applications without rigorous scrutiny. And poor, unsuspecting patients get duped, while doctors and hospitals earn money.” Source: Washington Post 1/1/12

Avastin Disappoints Against Ovarian Cancer

Avastin, the blockbuster drug that just lost approval for treating breast cancer, now looks disappointing against ovarian cancer, too. Two studies found it did not improve survival for most of these patients and kept their disease from worsening for only a few months, with more side effects. The Genentech drug has won approval in Europe for advanced ovarian cancer, but its maker has no immediate plans to seek the same approval in the United States. Avastin can still be sold for some colon, lung, kidney. and brain cancers. The new research was aimed at adding ovarian cancer to the list. One study involved nearly 1,900 women with advanced ovarian cancer given one of three treatment combinations. The time until the disease got worse was a median of 10 months in those given just chemotherapy; adding Avastin improved that by just one to four months for the other two groups. In the other study, more than 1,500 ovarian cancer patients were given chemo with or without Avastin. The drug kept cancer at bay just one to two months longer than chemo alone did, with more cases of high blood pressure. There was a trend toward improved survival for those on Avastin, but the difference was too small to say the drug was responsible. Source: Associated Press/Yahoo! News 12/28/11

New Fee Coming for Medical Effectiveness Research

Starting in 2012, the government will charge a new fee to your health insurance plan for research to find out which drugs, medical procedures, tests, and treatments work best, but what will Americans do with the answers? The goal of the research, part of a little-known provision of President Barack Obama's healthcare law, is to answer such basic questions as whether that new prescription drug advertised on TV really works better than an old generic costing much less, but in the politically charged environment surrounding healthcare, the idea of medical effectiveness research is eyed with suspicion. The insurance fee (starting at $1/year per person) could be branded a tax and drawn into the vortex of election-year politics. The Patient-Centered Outcomes Research Institute—a quasi-governmental agency created by Congress to carry out the research—has yet to commission a single head-to-head comparison, although its director is anxious to begin. Source: Associated Press/Yahoo! News 12/28/11

Elevation of the Chimp May Reshape Research

When Dr. Francis S. Collins, the head of the National Institutes of Health (NIH), announced in December that the government would halt all new grants for research on chimpanzees, it was, in a sense, a profound step. The NIH is the source of a river of money that flows into labs around the country where animals in the millions are used in the cause of increasing knowledge and alleviating human suffering. The report that prompted Dr. Collins’s announcement came from the Institute of Medicine, one of the National Academies. It was commissioned by the NIH, and sets criteria for how to decide when it is necessary to use chimpanzees in biomedical research and in less invasive genomic and behavioral and cognitive research. For biomedical experiments, it states that there must be no other animal or laboratory method suitable for the research; that it must be unethical to do the research on humans; and that giving up the use of chimpanzees must “significantly slow or prevent important advancements to prevent, control, and/or treat life-threatening or debilitating conditions.” Source: New York Times 12/20/11

Chimp Research Rarely Warranted, Says U.S. Expert Panel

Use of chimpanzees in government-funded medical research should be strictly limited, reserved only for studies where no suitable alternative is available or where testing in people would be unethical, a U.S. expert panel said on December 15. The Institute of Medicine said studies in chimpanzees should only be allowed in cases where not studying them would hinder progress in treatments for life-threatening or debilitating conditions. The report was commissioned by the U.S. Congress, which has been considering legislation that would ban research on chimpanzees and other great apes, following the lead of the European Union, which issued a ban on research use of all great apes in 2010. The report does not address use of chimpanzees in research done by industry, but the chair of the panel has urged companies to take up the same standard. Source: Reuters/Yahoo! News 12/15/11

U.S. Panel Urges Protection of Human Research Subjects

A bioethics panel convened in the wake of a Guatemalan sex disease scandal urged the U.S. government on December 15 to consider compensating victims who are harmed by participating in future research. The President's Bioethics Commission was authorized by Barack Obama after revelations last year that 1,300 people were exposed to venereal disease as part of macabre research led by an American in Guatemala in the 1940s. Eighty-three people died as a result. In its final report, the commission urged greater transparency, easy-to-understand warnings about the potential dangers of participating in studies, and a continued focus on high ethical standards in U.S. federally funded research. "The commission is confident that what happened in Guatemala in the 1940s could not happen today," said commission chair Amy Gutmann. "We also are confident that there is room for improvement in protecting human subjects from harm--avoidable harm--and unethical treatment." Source: Agence France Presse/Yahoo! News 12/15/11

Opinion: Free the FDA

According to Daniel Carpenter, a professor of government at Harvard, [t]he unilateral decision [in December] by Kathleen Sebelius, the secretary of health and human services, to block the Plan B One-Step contraceptive pill from being sold to adolescents without a prescription is shocking in more ways than one. Not only was it unexpected, but for the first time in American history, a cabinet secretary--and by extension, a president--has overruled a drug approval decision by the Food and Drug Administration (FDA). The precedent risks placing the real power for drug approval not just with a cabinet secretary, but with the White House itself. The possibilities opened by this decision are frightening. A radical pro-business secretary could now, in principle, bypass the clinical trial system and the FDA approval process and decide to approve a drug. A different secretary, one distrustful of the pharmaceutical industry, could stop a drug despite strong scientific support behind it. The solution is to do something that many FDA watchers have been proposing for at least a decade: Take the FDA out of the Department of Health and Human Services and make it an independent agency, like the Fed. Source: The New York Times 12/13/11  See also "Is HHS Using Scientific Standards?" from PharmTech Talk

FDA Revisits Safety of Newer Birth Control Drugs

Birth control drugs that were heavily promoted as having fewer side effects and the ability to clear up acne and other hormonal bothers are under new scrutiny from safety regulators. Research suggesting that newer birth control formulations are more likely to cause blood clots than older drugs has prompted the Food and Drug Administration (FDA) to consider new safety measures in meetings in December. The increased risk is slight, but significant because blood clots can cause heart attacks, strokes, and blockages in lungs or blood vessels, which can be fatal. Yaz, its Bayer precursor Yasmin, and similar drugs use a version of a female hormone that appears to reduce side effects found in older drugs, including bloating and mood swings. On December 6, a judge unsealed several court documents suggesting Bayer may have withheld data from FDA about the blood clots risks of its drugs. FDA also is reviewing research on clot risks associated with Johnson & Johnson's weekly Ortho Evra patch, which uses a different version of the female hormone progestin than the pills under scrutiny. Source: Associated Press/Yahoo! News 12/6/11

FDA Sees Huge Opportunities in Opening Up Drug Data

Regulators and drug makers need to find ways to make more clinical data openly available, since vital knowledge about fighting disease is often locked away in confidential databases, the head of the U.S. drugs watchdog said on December 5. Food and Drug Administration (FDA) Commissioner Margaret Hamburg said opening up data to public scrutiny needed to be done selectively, given legitimate concerns among companies over commercial confidentiality, but more could still be done. In particular, making detailed data available on how experimental drugs have performed in clinical tests, which are then filed with regulators, could prevent scientists from pursuing dead-end areas of research, she said. Campaigners have long pushed for the FDA to disclose full explanations and accompanying data when it rejects new drugs, but the agency's hands are tied by the need to protect company confidentiality. Source: Reuters/Yahoo! News 12/5/11

FDA Guides Companies in Ways to Design Trials for Artificial Pancreas

The Food and Drug Administration (FDA), under pressure from some law makers, issued draft guidance in early December that outlines how companies should construct outpatient clinical trials on the artificial pancreas, a medical device that combines two other devices to monitor and control blood sugar levels. The technology has seen very positive results in an impatient setting, and is already undergoing outpatient testing in many countries in Europe. An artificial pancreas has the potential to dramatically improve the health and quality of life for individuals with diabetes. Jeff Shuren, director of the FDA’s Center for Devices and Radiological Health, said that the 64-page draft guidance was designed to be flexible. For instance, companies can choose among several options as they design trials. Device manufacturers have some flexibility in choosing the number of patients to be studied, the length of a trial, and the indicators that will be judged to determine if the product works safely and effectively. Source: CQ HealthBeat 12/1/11

Pfizer's Blockbuster Drug Lipitor Goes Generic

Pfizer's patent on the best-selling drug of all time, the cholesterol-lowering medication Lipitor, expired on November 30, opening the path to generic competitors for America's most popular medication. Lipitor came on the market in 1997, and has raked in some $100 billion for Pfizer even in a crowded market that includes various other cholesterol-lowering statins, many of which have already gone generic. U.S.-based Watson Pharmaceuticals immediately announced its launch of a generic version of Lipitor, atorvastatin calcium, under an exclusive supply and distribution agreement with Pfizer, whereby Pfizer manufactures the drug and Watson sells it, sharing net sales with Pfizer until 2016. Indian-based Ranbaxy, which was also expected to release its generic in the next six months, still awaits the green light from U.S. authorities due to delays over problems with quality control and some of their Indian factories. Source: Agence France Presse/Yahoo! News 12/1/11

Ex-Executives Get Nine Months for Fatal Bone Cement Trial

Two former Pennsylvania medical device company executives have been sentenced to nine months in prison for unapproved medical trials that left three people dead. Former Synthes North America President Michael Huggins and former Senior Vice President Thomas B. Higgins were the first of four executives sentenced. They pleaded guilty to misdemeanors as "responsible corporate officers." U.S. District Judge Legrome Davis called the actions of company officials "egregious." Prosecutors say the executives bypassed safety rules and let surgeons test bone cement in frail patients, even though animals had died almost instantly in earlier tests. The other defendants are former Vice President Richard Bohner and ex-Director of Regulatory Affairs John Walsh. Source: Associated Press/Yahoo! News 11/21/11

U.S. Revokes Roche's Avastin for Breast Cancer

U.S. health officials on November 18 revoked the authorization of Roche's Avastin for breast cancer treatment, saying it concluded the drug had "not been shown to be safe and effective for that use." Avastin will still remain on the market as an approved treatment for certain types of colon, lung, kidney, and brain cancer, the U.S. Food and Drug Administration (FDA) said in a statement. The latest move followed the recommendation of an expert panel that said the drug, also known under the generic name bevacizumab, carries risks such as severe high blood pressure and hemorrhage and does not prolong overall survival in women suffering from breast cancer. The FDA had accepted the expert report that Avastin was not an effective treatment for breast cancer, but Roche decided to appeal. Avastin, which is marketed in the United States by the firm Genentech for its Swiss parent Roche, was approved for metastatic breast cancer in February 2008 under the FDA's accelerated approval program. The program provides early access to promising new drugs to treat serious or life-threatening conditions while clinical trials to confirm their efficacy are conducted. In the case of Avastin, the accelerated approval was based on promising results from one study that suggested it could extend the lives of women with advanced breast cancer. The company said it will start a new Phase III study of Avastin in combination with paclitaxel in previously untreated metastatic breast cancer, and will evaluate a potential biomarker that may help identify which people might derive a more substantial benefit from Avastin. Source: Agence France Presse/Yahoo! News 11/18/11

Drugs for Unmet Needs May Get Faster Approval Under Senate Plan

Congressional talks to renew the fees drug makers pay to fund Food and Drug Administration (FDA) reviews include proposals to fast-track evaluations of medicines for conditions with no approved cures, according to two U.S. Senate aides. Plans by Senator Kay Hagan (D-N.C.) and the Biotechnology Industry Organization in Washington would let manufacturers bypass customary clinical trial requirements before bringing products to market, according to drafts of both proposals. The proposals are factoring in Congressional talks to reauthorize the fee system pharmaceutical companies use to fund FDA reviews, said the aides, who aren’t authorized to speak publicly. While the FDA occasionally grants exemptions, “the experience of many biotechnology companies is that the criteria for utilizing this type of regulatory flexibility are unclear and unpredictable,” according to the biotech industry group’s document, a copy of which was obtained by Bloomberg. Diseases that may be targeted are rare disorders affecting fewer than 200,000 patients in the U.S. About 360 drugs exist for 7,000 such conditions, according to the National Organization for Rare Disorders. Source: Bloomberg News 11/16/11

Embryonic Stem Cell Trial for Paralysis is Halted

The first-ever trial using human embryonic stem cells to treat paralysis has been halted due to high costs, and the company will focus instead on new cancer treatments, Geron Corp. said on November 15. The California-based biotech firm, which had bypassed federal funding to get its controversial trial off the ground in October 2010, said it was also cutting 66 full-time jobs, or 38 percent of its workforce. "Geron plans to close the GRNOPC1 trial for spinal cord injury to further enrollment," the company said in a statement, adding that the decision was made after a "strategic review" of costs and "regulatory complexities." The study was meant to include up to 10 people in the first-ever trial of embryonic stem cell therapy on humans. In Geron's Phase I trial, which is meant primarily to gauge safety, the therapy showed no harmful effects on the four patients enrolled so far, the company said, adding that the "therapies have held and continue to hold great promise." Geron intends to focus instead on a pair of cancer treatments that are in Phase II studies. Source: Agence France Presse/Yahoo! News 11/15/11

"Triumph" Seen in Drug Trial for Lung Disease

A new gene-targeted treatment for cystic fibrosis (CF) has shown promise for treating a small number of those affected by the incurable and life-shortening lung disease, U.S. researchers said in early November. Published in the New England Journal of Medicine, the results of the Phase III trial using the drug ivacaftor showed it could improve lung function by acting against the defect which causes the disease. Made by the U.S.-based Vertex Pharmaceuticals, the drug works to restore the balance of salt and water on the surface of the patient's airways. The process is crucial for CF patients because their bodies produce a thick, sticky mucus that makes it hard to breathe. However, the treatment studied only applies to about four percent of the 70,000 people worldwide who suffer from the hereditary disease, because it targets a specific gene mutation known as G551D. More than 90 percent of patients have another mutation, called 508-CFTR. Source: Agence France Presse/Yahoo! News 11/2/11

Questioning Privacy Protections in Research

Hoping to protect privacy in an age when a fingernail clipping can reveal a person's identity, federal officials are planning to overhaul the rules that regulate research involving human subjects. However, critics outside the biomedical arena warn that the proposed revisions may unintentionally create a more serious problem: sealing off vast collections of publicly available information from inspection, including census data, market research, oral histories, and labor statistics. Organizations that represent tens of thousands of scholars in the humanities and social sciences scrambled to register their concerns before the late October deadline for public comment on the proposals. Every institution that receives money from any one of 18 federal agencies must create an ethics panel, called an institutional review board. More than 5,875 boards have to sign off on research involving human participants to ensure that subjects are fully informed, that their physical and emotional health is protected, and that their privacy is respected. Although only projects with federal financing are covered by what is known as the Common Rule, many institutions routinely subject all research with a human factor to review. The changes in the ethical guidelines--the first comprehensive revisions in more than 30 years--were prompted by a surge of health-related research and technological advances. Researchers in the humanities and social sciences are pleased that the reforms would address repeated complaints that medically oriented regulations have choked off research in their fields with irrelevant and cumbersome requirements. However, they were dismayed to discover that the desire to protect individuals' privacy in the genomics age resulted in rules that they say could also restrict access to basic data, like public opinion polls. Source: New York Times 10/24/11

Lilly Withdraws Sepsis Drug From Market

Eli Lilly and Co. said it is withdrawing its sepsis drug Xigris from all markets after it failed to help patients live longer in a clinical trial. "While there were no new safety findings, the study failed to demonstrate that Xigris improved patient survival and thus calls into question the benefit-risk profile of Xigris and its continued use," Timothy Garnett, Lilly's chief medical officer, said in a statement October 25. Approved in the United States in 2001 and in Europe in 2002, Xigris never reached its initial lofty sales expectations. The clinical study of Xigris, known as PROWESS-SHOCK, began in March 2008 as a condition for continued market authorization in Europe. Results of the 1,696-patient study showed Xigris did not meet the main goal of a statistically significant reduction in 28-day all-cause mortality in patients with septic shock. Patients currently receiving Xigris treatment should stop, while doctors should not start any new patients on the drug, Lilly said. Source: Reuters/Yahoo! News 10/25/11

FDA Says Medtronic Heart Device has Safety Issues

U.S. reviewers said a Medtronic Inc. device was effective for treating a common heart rhythm disorder, but raised concerns about its safety. Medtronic's Cardiac Ablation System is designed to treat persistent atrial fibrillation, a major cause of strokes. In documents released October 25, U.S. Food and Drug Administration (FDA) staff expressed concerns about the high rate of stroke found in patients treated with the device. Medtronic is seeking approval for the device as a treatment for persistent atrial fibrillation. In a clinical trial, five people out of 176 had a stroke within a month of getting the device, and 38 people had at least one serious problem, such as low blood pressure or swelling of the covering around the heart, FDA reviewers said. The device helped some people in clinical trials; 55.8 percent of patients had regular heartbeats within six months of using the device, compared to 26.4 percent of patients who used the typical treatment of antiarrhythmic drugs to treat atrial fibrillation. Ablation is a nonsurgical technique used to neutralize the cells within the heart muscle that are responsible for starting or maintaining abnormally fast, and sometimes lethal rhythms. Source: Reuters/Yahoo! News 10/25/11

Editorial: Questioning Prostate Cancer Tests

American men now have an authoritative warning that getting a blood test for prostate cancer may push them toward needless tests and treatments that would do them more harm than good. The United States Preventive Services Task Force, after an exhaustive review of the scientific evidence, has suggested that healthy men should no longer receive the PSA blood test to screen for a protein that may indicate cancer. The task force of 16 independent experts advises the Department of Health and Human Services on which screening tests have clinical benefits and which do not. It gave the PSA test its lowest rating, a Grade D, because the harm caused by having it outweighs the benefits. ...Critics, including urologists, who diagnose and treat prostate cancer, charge that the task force’s recommendations are misguided and will hurt patients. They have already been held up for two years lest they ignite charges of government rationing. That’s absurd. The recommendations are intended as guidance to help men and their doctors decide whether to use the test and how to react if it is positive. This is information patients need to know. Source: New York Times 10/11/11

Targacept Depression Drug: High Risk, High Reward

Targacept Inc., a small drug maker that began life inside the bowels of R.J. Reynolds Tobacco Co., has for years been using its understanding of nicotine to develop experimental treatments for depression, schizophrenia, and, yes, smoking cessation. Now it is poised to release data from a crucial trial of its depression drug, TC-5214, being developed with AstraZeneca. TC-5214 is thought to work by modulating neuronal nicotinic receptors in the brain. Overstimulation of these receptors, or proteins, is thought to be associated with depression. A mid-stage trial showed that when TC-5214 was given to patients who did not respond well to citalopram, an antidepressant sold under the name Celexa, patients experienced a significantly greater lessening of their depression than those who took citalopram plus a placebo. The key now is whether those results can be replicated in a bigger, Phase III trial. Targacept will announce the results of the first such trial by the end of the year. Source: Reuters/Yahoo! News 10/3/11

FDA Guidance on Biosimilars May be Imminent

The U.S. Food and Drug Administration (FDA) appears to be on the verge of issuing long-awaiting guidelines for the development of generic versions of complex biotechnology medicines. The FDA still plans to release the guidance by the end of the year, but the agency's top drug official, Janet Woodcock, has indicated it could come "as early as the next few weeks, maybe even days," Janice Soreth, deputy director of the agency's Europe office in London, said September 23. The agency has reached a "tentative agreement" with industry representatives over user fees companies would pay to support the biosimilar approval process, according to minutes of a meeting between FDA and the industry posted on FDA's website. Agreement on a pathway for producing cheaper versions of biotech drugs has been far more difficult than for traditional pills and capsules because their complex manufacturing process does not lend itself to production of exact copies. Making biotech copies is also expected to be more costly, as manufacturers must conduct extra clinical trials to show the new version is as good as the old one. Source: Reuters/Yahoo! News 9/23/11

China and India Making Inroads in Biotech Drugs

Chinese and Indian drug makers have taken over much of the global trade in medicines and now manufacture more than 80 percent of the active ingredients in drugs sold worldwide. However, they had never been able to copy the complex and expensive biotech medicines increasingly used to treat cancer, diabetes, and other diseases in rich nations like the United States—until now. These generic drug companies say they are on the verge of selling cheaper copies of such huge sellers as Herceptin for breast cancer, Avastin for colon cancer, Rituxan for non-Hodgkin’s lymphoma, and Enbrel for rheumatoid arthritis. Their entry into the market in the next year—made possible by hundreds of millions of dollars invested in biotechnology plants—could not only transform the care of patients in much of the world, but also ignite a counterattack by major pharmaceutical companies and diplomats from richer countries. The new biotech copycats are likely to stir sharp debate among advocates for the poor. Already, some contend that the billions spent to treat AIDS have crowded out cheap and simple solutions to other afflictions of poverty, like childhood diarrhea. In retrospect, the battle 10 years ago over AIDS medicines was a small skirmish compared to the one likely to erupt over cancer, diabetes, and heart medicines. Source: New York Times 9/18/11

Malaria Vaccine Shows Promise

An experimental malaria vaccine tested on children in Burkina Faso has shown "a high level of efficacy" in protecting against the disease, a study published in the United States said in September. The study was initially planned to examine the safety and immune response of the vaccine, known by the name MSP3. "However, as malaria attacks were documented as part of the safety follow-up, the investigators decided to explore the protective effect of the vaccine," said report appearing in the September 15 New England Journal of Medicine. In the study, 45 children aged 12 to 24 months were randomized into three groups receiving doses of either 15 or 30 micrograms of the experimental malaria vaccine, or the control vaccine against Hepatitis B. "Comparing the groups, they found a striking difference," the study said. It found children who received the new vaccine at either dose had incidence rates three to four times lower than children who did not, "yielding efficacy rates of 64 and 77 percent protection against clinical malaria," the journal article said. The only malaria vaccine in advanced clinical trials is the RTS,S vaccine being developed by British drugmaker GlaxoSmithKline, which could become available in 2015. A 2008 study showed the RTS,S vaccine with an efficacy of 53 percent for young children and 65 percent for infants. Source: Agence France Presse/Yahoo! News 9/14/11 

Many Think New is Better, But Older Pills Often Safer

Many consumers mistakenly believe new prescription drugs are always safer than those with long track records, and that only extremely effective drugs without major side effects win government approval, according to a new study. A national survey of nearly 3,000 adults finds that about four in 10 wrongly believe the U.S. Food and Drug Administration (FDA) approves only "extremely effective" drugs. One in four mistakenly believes the FDA allows only drugs that don't have serious side effects. That means consumers "may not get the benefit from drugs they think they're getting, or they may expose themselves to more harm than they think," said study coauthor Dr. Steven Woloshin of the Dartmouth Institute for Health Policy and Clinical Practice and the VA Outcomes Group. In truth, the FDA approves a new drug when its benefits outweigh any known risks. FDA approval doesn't mean the drug's benefits are large compared to drugs already on the market, and risks for some drugs appear only after they've been used by millions of people and long after FDA approval. The new survey, appearing in the September 12 Archives of Internal Medicine, revealed a partial solution to consumer confusion: Simply worded cautions can make a difference in which drugs people choose. Source: Associated Press/Yahoo! News 9/12/11

Drug Firm Payments to Doctors Declining

Total payments to doctors for promoting pharmaceutical companies’ products to their colleagues appear to be falling in Massachusetts, suggesting that new restrictions designed to distance doctors from industry are leading some to abandon the lucrative speaking circuit. Eli Lilly and Co., one of the nation’s largest drug makers, paid Massachusetts healthcare providers $866,919 in 2010 for speaking about their drugs, a 46 percent drop from 2009, according to an analysis by The Boston Globe and ProPublica, a nonprofit online investigative journalism organization. Payments from GlaxoSmithKline fell at least 29 percent to $884,850, and probably more because the company’s 2009 data did not include the first quarter. The data also show that many Harvard-affiliated doctors have dropped out of company speakers bureaus, a sideline that has allowed many physicians to earn tens of thousands of dollars. In 2009 and the first half of 2010, doctors and researchers affiliated with Harvard Medical School--a brand prized by pharmaceutical companies as a powerful tool in promoting drugs--collected a large portion of the speaking fees paid by drug companies, according to a similar analysis the news organizations conducted a year ago. However, new data for 2010 and the first quarter of 2011 reveal that many Harvard doctors have stopped giving promotional talks as new limits have been phased in. Source: Boston Globe 9/8/11

Stroke Patients Who Got Stents Did Far Worse

A new nationwide clinical trial on the use of brain stents in stroke patients showed the opposite of what researchers were expecting: Patients who received the stents after a stroke did far worse after 30 days than their counterparts who got medical therapies alone. Early data showed that stented patients who also received medical therapy were more than twice as likely to have a second stroke or die than those treated with medical therapy alone--aggressive control of blood pressure, cholesterol, and diabetes; antiplatelet drugs like aspirin and clopidogrel; smoking cessation; and a healthy lifestyle. The difference was so pronounced that the trials stopped enrolling new subjects five months ago, after nearly 36 months' duration, and the National Institutes of Health (NIH) issued a clinical alert. The randomized study, published September 7 in the online edition of the New England Journal of Medicine, is sponsored by the NIH. "This trial shows yet again that any kind of new therapy should be validated," said Tudor G. Jovin, director of the Stroke Institute at the University of Pittsburgh Medical Center and a principal investigator of the center's study site. "This doesn't mean we write off stenting. We just have to find better stents and select patients better than we have in this trial." One possible explanation: Patients with recent strokes may have unstable plaque in their arteries that the stent could have dislodged. The study device--the Gateway-Wingspan intracranial angioplasty and stenting system--is the only system approved by the Food and Drug Administration for certain high-risk stroke patients. Source: Philly.com/Pittsburgh Post-Gazette 9/7/11 See also Study Halted as a Stent Fails to Stop Strokes and Damage From Brain Stents

AstraZeneca Pushes Research Makeover

The pharmaceutical giant AstraZeneca PLC is reshaping its research and development operations with a growing presence in the Boston area. The goal is to ensure that more of the Anglo-Swedish company’s medicines make it to market, said Martin Mackay, who was recruited from a rival, Pfizer Inc., last summer to be AstraZeneca’s president of research and development. Toward that end, Mackay said he has refocused research, conducting what he calls a "root-and-branch analysis." That has served to reduce the number of disease areas AstraZeneca scientists target in fields ranging from neuroscience and cardiovascular diseases to inflammation and respiratory disorders. Too often, he said, the company used to measure research progress more by the number of compounds entering development, rather than by how many drugs won approval to be sold. "Many of our competitors have diversified," said Mackay, a native of Scotland. "We’ve said very clearly that we want to be a pure play pharmaceutical company across the globe. We are focusing on programs that have the best chance of becoming medicines." Boston Globe 9/6/11

Bayer, J&J Stroke Drug Not Ready for Approval

Drug regulators said an experimental stroke preventer from Bayer and Johnson & Johnson (J&J) is not ready for approval and raised questions about its effectiveness. "There is a lack of substantial evidence that rivaroxaban will have its desired effect when used as recommended in labeling," said Food and Drug Administration (FDA) staff in documents released September 6. The FDA staff recommended the agency issue a so-called complete response letter for the drug, also known by its proposed trade name Xarelto, which means it cannot be approved without more data from the companies. Even if approved, the agency staff said the drug should be used as a third option behind other treatments, unless the companies submit more data on effectiveness and safety. Xarelto is one of several promising entrants angling to replace risky clot preventer warfarin for people with dangerously irregular heart rhythms, called atrial fibrillation. Source: Reuters/Yahoo! News 9/6/11

Delivering Viruses to Try Killing Tumors

IRA FLATOW (Science Friday host): [R]esearchers reporting in the journal Nature say...[t]hey've engineered a virus that they can give intravenously, and the virus seeks out...cancer tumors wherever they are in the body. Joining me now to talk more about it is John C. Bell, senior scientist at the Ottawa Hospital Research Institute and a professor of medicine at the University of Ottawa in Ottawa, Canada. ...[Researchers have, in other studies], injected the viruses right into the tumor. But what is new about what your study is...?
Dr. JOHN C. BELL: ...[W]e knew what we really want to do is develop a vector that could be used intravenously, allow it to spread throughout the patient and find tumors wherever they're hidden. And [in] this study in Nature we just reported, we've been successful in doing that. We've treated patients by direct IV infusions of billions of virus particles. The viruses spread throughout the body, and when they land at a tumor site, they're able to productively infect it.
FLATOW: And are they able to kill it?
BELL: We certainly saw in some patients, at the high doses, some tumor regression. But this study was really what I would call a proof-of-concept study. We're checking to see if this approach was safe, which it turned out to be very safe, and whether or not we could actually deliver virus to various tumor sites within the body. And also they were only single doses. So we really weren't hoping to get a lot of therapeutic benefit from it, but even in that case, we did see some patients having tumors shrink a bit.
FLATOW: Do you continue to give these patients the viruses, seeing that it works in some of them?
BELL: You know, we would love to do that, but the way the studies are designed in the first place...we're only able to give a single dose to these patients. We won't be able to follow up with them. So now we're going to start other new studies in which we'll be able to give multiple doses of the virus intravenously and follow those patients to see if we have a therapeutic benefit.
FLATOW: Do you have trouble finding patients for these studies?
BELL: You know, that unfortunately is not a problem. I work at a cancer center, and really, every day you can see the toll this disease is taken, and many people are sent home and told we have no other treatments for them. And so then these very brave people like to volunteer for clinical trials and experimental therapeutics, and we've had no problem whatsoever filling up our trials. ...[Along] with this company Jennerex we're working with, we've...found that liver cancer seems to be a good target. And we're now initiating trials, larger trials, in liver cancer specifically, to see if this virus can attack and reduce those tumors and prolong patients' lives.
Source: Science Friday/National Public Radio 9/2/11

Europe Wrestles with Outdated Clinical Research Rules

The European pharmaceutical sector is currently struggling to articulate a clear plan for escaping the shackles of outdated rules on clinical research. “Divergences in requirements and processes, which appeared across countries and continue changing across time, have considerably complicated the registration of clinical trials in the European Union,” according to Celgene, in a recent submission to the European Union authorities. Bristol-Myers Squibb says new rules should be introduced to “drive consistency” across Europe, notably by “eliminating country-specific required documents.” Any proposal “will only work if countries stop requiring additional pieces to the clinical trial application dossier.” The European Union has received 143 responses to a recent consultation on how it should update its clinical trials rules--with hospitals, investigators, and noncommercial or academic sponsors the loudest in raising their voices. The pharmaceutical industry big and small, including contract research organizations, were well represented too--but so were national health authorities, ethics committees, and patient organizations; and in true European style, their views do not always correspond. Source: PharmExecBlog 8/31/11

Paul Meier, 1924-2011: Applied Statistics to Medical Research

Paul Meier influenced the field of statistics in two major ways: as a proponent of a method that helped eliminate bias in determining the effectiveness of treatments in clinical trials, and by introducing a system used to estimate survival rates for patients undergoing different treatments in trials. A longtime professor at the University of Chicago, Meier, 87, died August 7 at his home in New York. He suffered a major stroke more than 10 years ago and had been beset by a series of strokes more recently. Meier's work in methods for clinical trials led to a system that randomized which patients received an experimental treatment, as opposed to a more traditional treatment. In 1958, Meier coauthored a paper that introduced the Kaplan-Meier estimator. The method incorporates data from patients in clinical trials who have been followed until death, as well as others who survived. Meier and Edward L. Kaplan had each submitted similar papers to the same publication, the Journal of the American Statistical Association. The editor then convinced them to combine their work into one paper, which has been cited about 34,000 times since its publication. Meier also helped found the Society for Clinical Trials, and from 1986 to 1987 was its president. He was also an advisor to the U.S. Food and Drug Administration. Source: Chicago Tribune 8/18/11 See also New York Times

HHS Cutting Red Tape to Speed Clinical Trials

Wide-reaching changes announced by the U.S. Department of Health and Human Services (HHS) would speed up the process of approving and monitoring federally funded clinical trials. The plans, which represent the first substantive revisions to the country's human research subjects regulations since they were adopted three decades ago, could help ease the regulatory burden faced by the estimated 30,000 U.S. physicians who act as clinical investigators. The rules, adopted after abuses in Tuskegee, Ala., and elsewhere came to light, initially took effect when most trials were conducted at a single site. Today, the vast majority are conducted at many different locations, yet federal rules require that each site's institutional review board (IRB) scrutinize and approve the research protocol and informed consent procedure. Critics have long argued that the process is redundant, does little to protect trial participants, and adds to the cost of conducting badly needed clinical research. The HHS proposal, announced in July, would mandate that all domestic sites in a multisite trial name a single review board as the IRB of record, legally responsible for complying with federal regulations and ensuring that trial participants are properly informed of the risks and protected from dangerous protocols. Local IRBs still could review protocols and consent forms and suggest changes, but they would have more of an advisory role. While seeking to lighten the regulatory burden, HHS plans to expand who is covered by federal rules. Currently, human research subjects regulations apply only to studies funded by one of 15 federal agencies or to Food and Drug Administration-regulated trials. Under the HHS proposal, the rules would apply to all studies—regardless of how they are funded—that are conducted at a U.S. institution that receives any federal funding for human subjects research. The HHS revisions also would limit the acceptable length of informed consent documents, prescribe how information is presented, allow certain types of information to be included in addenda, slash legal boilerplate terms, and make available standardized consent form templates. Such reforms are long overdue, experts said. Source: American Medical News 8/15/11

Comparative Effectiveness Research Tackles Medicine’s Unanswered Questions

Nobody familiar with American medical care in the 21st century should be surprised that a 73-year-old woman can be minutes away from getting a painful collapsed vertebra filled with liquid plastic and it’s impossible to say whether the procedure works, or how. American medical care is rife with such treatments, whose usefulness is uncertain not just to the doctors who deliver them but also to the patients who receive them. These days, however, many people are pinning their hopes on “comparative effectiveness research” as way to solve the dilemma of how best to treat this and hundreds of other common problems in day-to-day medicine. Comparative effectiveness research goes beyond the basic question—“Is this safe and effective?”—that must be answered before new a new drug or device goes on the market. Instead, this emerging field tries to determine where a drug, a procedure, a test, or a therapeutic strategy fits into the world of what’s already available and being used. Comparative effectiveness research studies now under way are funded by $1.1 billion in the Obama administration’s 2009 economic stimulus package. The purpose isn’t to declare hands-down winners (although that occasionally happens). It’s to provide practical guidance when there’s more than one reasonable option. The federal Agency for Healthcare Research and Quality this year is spending about $21 million on comparative effectiveness studies. This has never been a high priority for the country or its scientists. Only 1.5 percent of money spent on medical research goes to “outcomes research,” of which comparative effectiveness is a sub-category. About 13,000 new clinical studies start up each year; about 112,000 are running now. A meticulous search in 2008 revealed only 689 studies that fit the general description of “comparative effectiveness.” Many experts believe that’s not enough. Source: Washington Post 8/15/11

Bernadine Healy, NIH and Red Cross Leader, Dies at 67

Bernadine Healy, the accomplished and controversial first female director of the National Institutes of Health (NIH) and past president of the American Heart Association and the American Red Cross, died August 6 of a recurrence of brain cancer at her home in Gates Mills, Ohio. She was 67. Dr. Healy, a cardiologist, helped turn around a fractured, underfunded, and under-fire NIH in the early 1990s. Her most notable accomplishment there was launching the Women’s Health Initiative, a $500 million, 10-year study of diseases that affect women at midlife and beyond. The study revealed previously undiscovered dangers to women’s health, such as the risks of heart attacks and strokes in postmenopausal women. In the late 1990s, she was hired as a change agent at the notoriously change-resistant Red Cross. She was forced out of that job after two years, after a series of differences with longtime board members who objected to her assertive, sometimes steely style, and to their loss of control over day-to-day decision-making. Source: Washington Post 8/8/11

Report Questions "Offshoring" in U.S. Heart Studies

Major U.S.-sponsored clinical trials on heart disease often turn to other countries to recruit patients, and a new report questions whether that undermines the evidence they generate and the health of the American clinical trial system. Researchers found that of 24 U.S. taxpayer-funded clinical trials on heart disease in the past decade, 19 included patients from other countries. Across 11 of those studies, international patients accounted for nearly half of participants. That high international involvement raises concerns about whether the results of a trial that includes people from other nations--and possibly with varied demographics, lifestyle habits, and healthcare systems--can be reliably applied to the U.S. population. There is also the question of whether far-flung research sites can be expected to follow the ethics and standards of conduct required in studies funded by the U.S. National Heart, Lung, and Blood Institute. Often, "offshoring" occurs because the studies cannot get enough U.S. patients to participate. Source: Reuters/Fox News 8/3/11

IRB is Fooled by Phony Clinical Trial Sponsor

Earlier this year, the Food and Drug Administration (FDA) issued an alert that some fictitious applications were submitted to several institutional review boards (IRBs). The agency noted that the name and address of the clinical investigator listed on a required FDA form are the same as that used in a sting operation conducted by the U.S. Government Accountability Office two years ago. One of the IRBs tripped up in the new effort was Essex Institutional Review Board, a for-profit operator. Last March, FDA inspectors visited its Lebanon, N.J., facilities and found that not only had Essex reviewed and approved the fictitious clinical investigator to conduct a clinical trial protocol, but the IRB failed to protect the participants, according to a July 26 FDA Warning Letter (posted here). Source: Pharmalot 8/2/11

Study of Medical Device Rules is Attacked, Unseen

Allies of the medical device industry are waging an extraordinary campaign in Washington to discredit a report by one of the country’s pre-eminent scientific groups that examines possible new regulations on the industry. The scientific group, the Institute of Medicine, was scheduled to release a report on July 29 that could propose a tougher approval process for a wide range of devices like hip implants, hospital pumps, and external heart defibrillators. The report, commissioned by the Food and Drug Administration, comes after several well-publicized recalls in recent years of devices that have failed in thousands of patients, causing numerous injuries. However, a business group and others have taken the highly unusual step of making a pre-emptive strike, arguing that the report is biased. That attack began even before the study panel finished its review, and has intensified in recent weeks. Source: The New York Times 7/27/11

FDA Says Cetero Faked Documents, Manipulated Samples at Lab

Sponsors face having to redo lab work after the Food and Drug Administration (FDA) alleged that contract research organization Cetero falsified records and manipulated samples to meet acceptance criteria. Alleged misconduct at Cetero’s laboratory in Houston, Texas, has been the focus of internal and external investigations for more than two years. After receiving unsatisfactory responses to two 2010 inspections, the FDA has gone public with the investigation. A Cetero employee told the FDA that equilibration or “prep” runs were allegedly used to manipulate data by “fixing” runs to meet acceptance criteria prior to inclusion of data in the official study folder. Also, an internal investigation by Cetero found 1,900 cases in which samples were allegedly taken on weekends or holidays, but chemist arrival times were more than one hour after the extraction. Cetero said this was so employees received weekend work “financial incentives,” but the FDA disagrees. Source: Outsourcing-Pharma.com 7/28/11

Watchdog Group Wants "Unethical" Canakinumab Trials Halted

The watchdog group Public Citizen wants the U.S. Food and Drug Administration (FDA) to immediately suspend two clinical trials involving canakinumab (Ilaris, Novartis) in light of safety concerns that caused an FDA advisory panel to vote against approving the drug for acute flares of gouty arthritis. A spokesperson for Novartis counters that the company is confident in the safety profile of the drug "for appropriate patients." One of the clinical trials, sponsored by Novartis and aiming to enroll 7,200 subjects, is designed to determine whether canakinumab decreases the chances of major cardiovascular events in patients who have had a heart attack. The other study, sponsored by the National Institutes of Health, looks at the effects of the injectable immunosuppressant on the progression of type 1 diabetes in 66 subjects, as young as 6 years, with newly diagnosed disease. Both trials appear unwarranted and "unethical," given the apparent lack of preliminary data regarding the drug's potential benefits, according to a July 19 letter from Public Citizen to the FDA. Source: MedScape News 7/26/11

Japanese Herb for Hot Flashes Fails in U.S. Trial

An herbal remedy widely used in Japan to ease menopause symptoms failed to show the same benefits in a clinical trial of U.S. women, researchers report. The study looked at the effects of keishi-bukuryo-gan, a mix of cinnamon bark, peach pit, and several other botanicals that is also known as known as TU-025. Long used in traditional Japanese medicine, the herbal remedy is now regulated as a prescription drug in Japan, where gynecologists commonly recommend it for treating hot flashes and other symptoms of menopause. So researchers were interested in whether benefits would be seen in U.S. women as well. The authors randomly assigned 178 postmenopausal women to take either keishi-bukuryo-gan tablets or a placebo every day for three months. Tokyo-based Tsumura & Co. provided the herb and partially funded the study. In the end, the researchers found that while women on the herb saw improvements in hot flashes, sleep problems, and other symptoms, so did women on the placebo. And there was no clear advantage of the herbal product, the team reports in the journal Menopause. Source: Reuters/Yahoo! News 7/22/11

Transparency Rules a Tangle at Cancer Journals

Economic ties that could bias drug trials and patient care might remain hidden due to tangled disclosure rules at medical journals, a new study reveals. Researchers found that of 131 cancer journals, only 112 had policies requiring researchers to state conflicts of interest, such as drugmaker stock ownership or speaker fees. Furthermore, among journals that did have such policies, the rules were all over the map. "Journals can't even agree on what a conflict of interest means," said Dr. Aaron S. Kesselheim of the Harvard Medical School in Boston. "It is certainly confusing to authors and to readers." Scores of studies have shown that when researchers have a financial stake in their work, their reports are more likely to promote drugs and downplay side effects. Source: Reuters/Yahoo! News 7/22/11

Tarceva Battles Lung Cancer in Some

New research finds that the targeted cancer drug Tarceva nearly triples the amount of time lung cancer patients survive without a recurrence and has fewer side effects than standard chemotherapy. The authors of a study appearing in the July 21 online issue of The Lancet Oncology recommend using Tarceva (erlotinib) as a first-line treatment for patients with advanced non-small cell lung cancer who have the particular gene mutation this drug targets. Other experts agreed. The findings echo the results of previous randomized trials but, according to the authors, this is the first study to show that patients with the EGFR mutation who take Tarceva can live for more than a year without their cancer returning. The study was partially funded by F. Hoffmann-La Roche Ltd., maker of Tarceva. Right now, Tarceva is approved to use as a second-line treatment for lung cancer. Source: HealthDay/Yahoo! News 7/21/11

Unpublished Cancer Trials May Affect Clinical Practice

A "substantial" number of Phase III clinical trials of systemic cancer treatments with potential influence on clinical practice remain unpublished after 6.5 years or more, according to a new report from Canadian researchers. The investigators also found that "many" other trials are published after a delay of five years or more. Their paper was published online July 11 in the Journal of Clinical Oncology. The findings have potential ramifications in oncology practice, say the authors. "Nonpublication of clinical trials predominantly reporting negative results can lead to overestimation of treatment benefits and may adversely influence clinical practice," write the authors, led by Vincent C. Tam, MD, from the University of Toronto, in Ontario. "Nonpublication also breaks an implicit contract that investigators make with study participants," they write. Source: Medscape Medical News 7/13/11

FDA Proposes Targeted Drug Testing Guidelines

Targeted drugs or therapies up for regulatory approval would have to be reviewed simultaneously with the diagnostic devices they rely on, according to a proposed policy issued July 12. So-called targeted treatments or personalized medicines are tailored to a person's genetic makeup and are being increasingly developed by drug companies. The Food and Drug Administration (FDA) proposed rules saying these personalized treatments would gain approval only after their accompanying diagnostic devices also receive approval--unless the treatment is for a serious or life-threatening condition. Diagnostic tests, known as companion diagnostics, improve the effectiveness of targeted treatments by determining if a patient is a genetic fit with a therapy. The FDA is now seeking public comment on the proposal. Source: Reuters 7/12/11

How Bright Promise in Cancer Testing Fell Apart

Serious problems have become evident in a field in which the medical community has placed great hope: using patterns from large groups of genes or other molecules to improve the detection and treatment of cancer. Companies have been formed and products have been introduced that claim to use genetics in this way, but assertions have turned out to be unfounded. While researchers agree there is great promise in this science, it has yet to yield many reliable methods for diagnosing cancer or identifying the best treatment. Instead, as patients and their doctors try to make critical decisions about serious illnesses, they may be getting worthless information that is based on bad science. So far, the Food and Drug Administration (FDA) “has generally not enforced” its regulation of tests created by individual labs because, until recently, such tests were relatively simple and relied heavily on the expertise of a particular doctor, said Erica Jefferson, a spokeswoman for the agency. However, with labs now offering more complex tests on a large scale, the FDA is taking a new look at enforcement. Source: New York Times 7/7/11

Behind Potent Drug Names, a Complex Mix of Ingredients

They are the public face of products that can take many years and hundreds of millions of dollars to develop. Over time, they often become household names: Think Prozac or Viagra. However, drug names start out as alien words: random collections of syllables that can sometimes seem almost as mind-boggling as the complicated chemistry and biology that went into the development of the new medication. Looking behind the curtain at how drug names are born provides a window into a side of the drug industry that most people interact with but few understand, a process that is both art and science, and requires a delicate balance of regulatory, legal, and linguistic concerns. Names aren’t allowed to make an unsupportable claim about the product and can’t be similar to existing names. They they also have to survive a detailed linguistic analysis: Are the names pronounceable in other languages? Do they have negative or confusing cultural meanings? Source: Boston Globe 7/4/11

U.S. Panel Rejects Avastin for Breast Cancer Use

An expert panel urged the U.S. Food and Drug Administration (FDA) to strip the Roche-made drug Avastin of its label for use against breast cancer because it is neither safe nor effective. After a rare two-day appeal hearing by Genentech, a U.S. unit of the Swiss pharmaceutical giant, the panel voted 6-0 to uphold its earlier recommendation in December to stop the use of Avastin for breast cancer. The drug, also known as bevacizumab, carries risks such as severe high blood pressure and hemorrhage and does not prolong overall survival in women suffering from breast cancer, the panel said. A final decision by the FDA commissioner must be issued, but will not likely come before the end of July. The FDA does not have to follow the recommendations of the expert panel, but it usually does. Source: Agence France-Presse 6/29/11

Spine Experts Repudiate Medtronic Studies

In an extraordinary move, a group of spine specialists are publicly repudiating the research of other experts that has backed the widespread use of a Medtronic bone growth product. In a series of reports published in a medical journal on June 28, the specialists called the research misleading and biased. The repudiation, appearing in a full issue of The Spine Journal devoted to the topic, represents a watershed in the long-running debate over conflicts of interest for the sponsorship of scientific studies by makers of drugs and medical devices. It is extremely rare for researchers to publicly chastise colleagues, and editors of leading medical journals said they could not recall an instance in which a publication had dedicated an entire issue for such a singular purpose. Source: New York Times 6/28/11

Drug Research and Development Spending Fell in 2010, and Heading Lower

The global drug industry cut its research spending for the first time ever in 2010, after decades of relentless increases, and the pace of decline looks set to quicken this year. Overall expenditure on discovering and developing new medicines amounted to an estimated $68 billion last year, down nearly 3 percent on the $70 billion spent in both 2008 and 2009, according to Thomson Reuters data released on June 27. The fall reflects a growing disillusionment with poor returns on pharmaceutical research and development. Disappointing research productivity is arguably the biggest single factor behind the declining valuations of the sector over the past decade. Source: Reuters 6/27/11

Diabetes Vaccine Stumbles at Second Hurdle

An experimental vaccine to prevent progression of type 1 diabetes failed at the second step of the three-phase trial process, doctors said in a study reported online by The Lancet. The vaccine sought to protect insulin-producing beta cells in the pancreas from the body's immune system. Its formula is based on an enzyme called glutamic acid decarboxylase (GAD), which the over-sensitized immune system targets--and in doing so destroys the precious beta cells. The idea was that by vaccinating patients with GAD, this would teach the immune system's T cells to tolerate the enzyme. The trial was carried out on 145 patients aged three to 45 years living in the United States and Canada who had been diagnosed with type 1 diabetes within the previous three months. The volunteers were given either the vaccine; the vaccine plus a standard immune-system booster; or just the booster alone. Patients in all three groups experienced similar progression in the disease, with no difference among them in side effects. Source: Agence France-Presse 6/27/11

Senators Investigate Medtronic Spine Implant, Payments to Doctors

The U.S. Senate Finance Committee is investigating whether Medtronic and physicians with "substantial financial ties" to the company were aware of complications associated with the company's Infuse bone graft product and failed to report them in published clinical trials. In a letter requesting a host of documents from the medical device manufacturer, Senate Finance Committee Chairman Max Baucus (D-Mont.) and Senior Member Charles Grassley (R-Iowa) write that they are "extremely troubled by press reports" suggesting that in its own funded clinical trials, Medtronic and its paid consultant physicians may have "unreported or under-reported" potential risks associated with the use of Infuse, a recombinant human bone morphogenetic protein 2 (rhBMP-2) treatment used in spinal fusion surgery. The product is used to stimulate bone growth and, by Medtronic's estimates, has been used in more than 500,000 patients. Despite its widespread use, Infuse has come under scrutiny for alleged off-label use and accusations of falsified research funded by Medtronic. Several independent studies have identified risks ranging from sterility in men to "life-threatening complications," such as neurological impairment and swelling in the neck and throat. Source: Outpatient Surgery Magazine 6/26/11

Biotech Firms Want Drug Approval Hastened

Biotechnology industry leaders want to speed up the process of bringing drugs to market by broadening the mission of the Food and Drug Administration (FDA) to include innovation as well as product safety. A raft of industry proposals to overhaul the FDA and create fresh incentives to help fund drug development will be unveiled in Washington, D.C. next week at the 2011 international convention of the Biotechnology Industry Organization, known as BIO. “We want to change the formal legal mission statement of the FDA," BIO president James C. Greenwood said. “The FDA understands that if they approve a product that’s not safe or effective, that’s a failure, and we agree with that. But it’s not deemed a failure if people die because they took too long to approve a product." FDA spokeswoman Karen Riley said part of the reason it takes so long to get drugs on the market is the length of companies’ clinical trials. Source: Boston Globe 6/22/11

Merck KGaA Pulls Plug On Cladribine After FDA Feedback

Merck KGaA said U.S. Food and Drug Administration (FDA) concerns about the risks of its cladribine pill will put an end to any development or marketing plans for the multiple sclerosis (MS) treatment. Merck also said it planned to withdraw cladribine from markets in Australia and Russia, where it has been approved and is available under the name Movectro. "Merck believes that data from ongoing clinical trials are very unlikely to address the FDA's requirements," it said, adding new trials would not justify the costs. In March, the FDA asked Merck to either provide additional analyses of study results it had submitted, or to carry out new trials. Due to some cases of cancer that emerged during a trial, the drug had been rejected in September by regulators in the European Union, which would have been its largest market. Cladribine had initially been regarded as a formidable contender in the race with Novartis to bring the first oral treatment against MS to a major market. Source: Reuters/Yahoo! News 6/22/11

FDA Panel Unanimously Backs Regeneron Eye Drug

A panel of federal health experts has voted unanimously in favor of a new eye drug from Regeneron, bringing the company one step closer to competing against Lucentis, a blockbuster Roche drug that currently dominates the market. A Food and Drug Administration (FDA) advisory panel voted 10-0 that Regeneron's drug is a safe and effective treatment for a condition that can lead to blindness in seniors. More than 200,000 new cases of the condition, called wet macular degeneration, are diagnosed each year. The FDA is scheduled to make its final decision on approval by August 20. If approved, Regeneron and its partner Bayer Pharmaceuticals will co-market the drug under the brand name Eylea. Regeneron may face its toughest competition from another Roche drug called Avastin, a cancer drug that doctors have used for many years to treat the eye disease even though it is not approved for that purpose. Source: Associated Press/Yahoo! News 6/17/11

Biotechs Spending Less On Drug Discovery

U.S. biotechnology companies raised 15 percent more in investment funding last year than in 2009, but “innovation capital"—the amount actually spent on drug discovery—declined by 20 percent, according to a new Ernst & Young report. That’s because nearly half of biotechnology investment money last year was raised through debt financing deals by larger mature companies, which used it to buy back their own shares, boosting earnings and share prices. Meanwhile, the report said, start-ups and unprofitable smaller companies found it increasingly difficult to raise cash. The findings suggest that the industry’s investor-backed business model has come under strain. One sign cited by the report: The number of drugs approved annually by federal regulators fell to 21 between 2005 and 2010, down from an average of 36 approved between 1996 and 2004. Source: Boston Globe 6/14/11

Pfizer Joins Academics in Bid for Progress

Pharmaceutical giant Pfizer Inc. announced an unusual partnership with leading Boston-area hospitals, medical schools, and universities in June, in an attempt to address a major problem in medicine: the years-long gap between basic science discoveries and the testing of drugs stemming from those advances. The company will invest $100 million over five years and establish a research space in the heart of the Longwood Medical Area, where Pfizer scientists will work in teams with academic researchers, jointly planning and pursuing projects. The new Center for Therapeutic Innovation, which will create about 50 jobs, is part of a global Pfizer initiative to foster new kinds of collaboration with academia to accelerate drug development, a program that will be headquartered in Boston. Source: Boston Globe 6/9/11

Drugs for Rare Cancers Approved After Subpar Tests

Drugs for rare cancers are allowed to hit the U.S. market based on low-quality clinical tests that raise concerns about both safety and efficacy, researchers said in June. Known as orphan drugs, such medications are used to treat diseases that affect fewer than 200,000 Americans and so generate only limited revenue for manufacturers. Under the Orphan Drug Act of 1983, the government has been incentivizing their development heavily by offering market rights, research grants, and tax credits to manufacturers. However, recent scandals have sparked concerns about orphan drugs. In the current study, researchers tapped into Food and Drug Administration data from 2004 to 2010 to get a sense of the evidence behind new cancer medications, which account for a large proportion of orphan drugs. They found 15 orphan drugs and 12 nonorphan drugs, and the quality of the clinical testing varied markedly between the two groups. The trials of orphan drugs enrolled only 96 patients on average, for example, compared to 290 in the other trials. They were also less likely to assign patients randomly to different treatments. Dr. Aaron S. Kesselheim, who worked on the study, said his findings didn't mean that orphan drugs are necessarily unsafe or shouldn't have been approved--it might not be realistic to do large trials for very rare diseases, for instance. However, amending the law might be necessary to achieve more credible data and save ourselves unpleasant surprises, he told Reuters Health. Source: Reuters/Yahoo! News 6/6/11

NIH Clinical Trials Look for Causes and Cures for Disease

Scientists at the National Institutes of Health (NIH) know Samantha Seinfeld’s body down to its cellular makeup. Every few months, she shows up at NIH’s leafy Bethesda, Md., campus to get a CT scan and be injected with an experimental vaccine...in the hopes of staving off a recurrence of stage IV breast cancer. For three years, Seinfeld has been a participant in one of NIH’s clinical trials. This one is testing whether a vaccine called PANVAC and its follow-up boosters will keep breast cancer, which first showed up in Seinfeld’s breastbone five years ago, from coming back. It’s risky, uncertain, and exhausting at times—and not necessarily foolproof. Despite all that, Seinfeld is extremely grateful. “Clinical trials are not for everyone,” she said. “My faith has been in science. I find it more comforting to look at numbers, statistics, and research. Cancer is not a death sentence in this day and age. So much is being done in research.” NIH is conducting nearly 1,500 trials at its Clinical Center and is budgeted to spend about $10 billion on clinical research this year, with about $3 billion of that specifically for trials. The center is the largest hospital in the world dedicated to these human studies, and many of the diseases being studied are too rare, or the treatment is too much of a gamble, for pharmaceutical companies, biotech firms, or universities to pursue. About 400,000 patients have participated in NIH-sponsored clinical trials, and they come for a variety of reasons: a last-ditch effort at a cure, a rare disease no one else understands or can treat further, a new approach to a disease, or a desire to further medical knowledge. Source: Washington Post 5/30/11

Op-Ed: Drugs and Profits

Writing in the New York Times, oncologist Frederick C. Tucker Jr. points out that last year the Food and Drug Administration (FDA) rescinded approval of the drug Avastin for treating breast cancer patients, prompting a firestorm of criticism. The decision was denounced by some politicians as healthcare rationing, and by breast cancer patients who feared that they would be deprived of a drug that they felt had helped them immensely. But these criticisms ignore the facts: Avastin was rejected simply because it didn’t work as it was supposed to, and the FDA should resist the aggressive campaign by Genentech, the drug’s maker, to get that ruling reconsidered at a hearing in late June. Treating a breast cancer patient with Avastin costs about $90,000 a year, and Genentech could lose $500 million to $1 billion a year in revenue if the FDA upholds the ban. Avastin will not disappear because of the FDA decision. It remains available for treating other cancers, and research to find its appropriate role in breast cancer treatment continues. In the meantime, the FDA, which is expected to make its decision in September, needs to resist Genentech’s attempt to have it ignore scientific evidence. Serious progress in the treatment of cancer will not be the result of polemics, lobbying, or marketing. Genentech’s money and efforts would be better spent on research for more meaningful treatments for breast cancer. Source: New York Times 5/25/11

Vertex Hepatitis C Drug Wins Approval

Vertex Pharmaceuticals Inc.'s highly anticipated hepatitis C drug won U.S. approval on May 23, heralding a new era of treatment for the liver-destroying condition and setting up a head-to-head marketing battle with a rival medicine from Merck & Co. Incivek, a pill also known as telaprevir, is poised to help transform treatment of hepatitis C by nearly doubling the chances of curing the serious liver disease. A 12-week regimen of Incivek works in combination with current standard drugs pegylated interferon and ribavirin. The older treatments are then continued for as few as another 12 weeks. In clinical studies, as many as 79 percent of patients taking Incivek experienced a sustained virologic response--which is tantamount to a cure. On May 13, the Food and Drug Administration approved Merck's Victrelis for hepatitis C, which works the same way as Incivek and is also taken with the older medicines. Analysts expect Incivek to eventually control more of the market because it has shown a higher cure rate. Source: Reuters/Yahoo! News 5/23/11

Merck’s Hepatitis Drug Gets Final FDA Approval

The Food and Drug Administration (FDA) approved a highly anticipated hepatitis C drug from Merck & Co. in May that is the first new treatment for the virus in 20 years. The first-of-its-kind pill, Victrelis, has been shown to cure more patients in less time than the drugs now used. The current two-drug treatment for the virus cures only about 40 percent of people and causes side effects such as nausea, fatigue, and vomiting. The FDA said it approved the new drug based on two trials in which more than 65 percent of patients were cured when combining Victrelis with the two older drugs. Some patients were also able to eliminate the virus in seven months on the drug, half the time needed with the current treatments alone. Source: Boston Globe 5/14/11

Experts Debate Destroying Last Smallpox Viruses

Smallpox, one of the world's deadliest diseases, eradicated three decades ago, is kept alive under tight security today in just two places—the United States and Russia. Many other countries say the world would be safer if those stockpiles of the virus were destroyed. In May, for the fifth time, at a World Health Organization meeting, they pushed again for the virus' destruction; and again, it seems likely their efforts will be futile. U.S. and Russian government officials say it is essential they keep some smallpox alive in case a future biological threat demands more tests with the virus. They also say the virus samples are still needed to develop experimental vaccines and drugs. Smallpox is one of the most lethal diseases in history. For centuries, it killed about one-third of the people it infected. The last known case was in Britain in 1978. Source: Associated Press/Yahoo! News 5/13/11 See also UN Puts Off Destroying Last Smallpox Viruses 

Earlier HIV Therapy Protects Against Virus Spread

Treating HIV right away, before patients are too sick, dramatically lowers their chances of spreading the AIDS virus to a sexual partner, says a major international study that may convince more doctors to offer medication sooner. The nine-nation study offers convincing evidence of what scientists have long believed—that HIV medicines don't just benefit the patient, but may act as a preventive by making those people less infectious. Earlier treatment in the study meant patients were 96 percent less likely to spread the virus to their uninfected partners, according to preliminary results announced in May by the National Institutes of Health (NIH), which oversaw the research. Those findings were striking enough that the NIH said it was stopping the study four years ahead of schedule to get the word out. Source: Associated Press/Yahoo! News 5/12/11

Big Pharma's Global Guinea Pigs

April's annual meeting of the American College of Cardiology in New Orleans, La., was the usual frenetic mix of world-class science, networking, and lobbying by the $850 billion-a-year pharmaceutical industry, seeking to promote its wares to key opinion leaders. The commercial razzmatazz of the convention center's giant trade stands, advertising big brand drugs and medical devices, painted a bright picture for medical science. Under the surface, however, a growing number of doctors are worried about the tectonic changes in drug research. They resent the export of clinical trial work, which they blame not only on industry's endless pursuit of lower costs, but also on the increasing red tape surrounding trial procedures at home. Source: Reuters 5/6/11

Consumer Group Says Key Data Left Out of Alzheimer's Study

A U.S. consumer group alleged on May 10 that researchers with ties to Eli Lilly and Co. withheld important information from a medical journal in their study of an imaging drug for Alzheimer's disease. In a letter published in the Journal of the American Medical Association, Public Citizen criticized a January 19 study that assessed the effectiveness of brain scans using Lilly's Amyvid, an experimental dye to detect brain abnormalities. The letter said the study authors excluded data on how accurately the scans were interpreted from one physician to the next. Lilly strongly disputes the claims, calling them "inaccurate." "We worked with the [Food and Drug Administration (FDA)] to identify and specify all endpoints for the Amyvid Phase III study," the company said in a statement. Lilly said all of the endpoints in the study were met, and they were "disclosed appropriately. In March, the FDA declined to approve Amyvid, citing the need to ensure the scans can be accurately read. An unreliable test could lead some people to believe they had Alzheimer's when they did not, and it could give others the false assurance that the did not have the disease, when they might, Public Citizen said in an e-mail. Source: Reuters/Yahoo! News 5/10/11

Drug Makers See Gains in Broadening Their Reach

After years of focusing on common diseases afflicting mostly middle-class and affluent patients, drug companies are devoting more resources to rare disorders, illnesses that are prevalent in the developing world, and medical conditions that affect minority populations in rich countries. The trend, which is slowly gaining momentum, is being driven by several factors, including the emergence of "personalized" medicines based on an individual’s genetic makeup, and the success of companies that already specialize in making drugs to treat rare diseases. Even companies that concentrate on more common diseases are coming under pressure to test their drugs on different segments of the population. Source: Boston Globe 5/4/11

The Push is On for More Drugs for Deadly Rare Diseases

Many observers would say that getting help to fight a rare disease shouldn't be as hard as it is, but it's a huge challenge to generate drug company interest in the expensive testing of medicines for diseases so rare that the market is only a few hundred or few thousand people a year. There are treatments for just 200 of the roughly 7,000 rare diseases, or illnesses that affect fewer than 200,000 people (and often far fewer). However, if you add those diseases together, more than 20 million Americans have one. Now a movement is beginning to spur more rare disease treatments: The National Institutes of Health this fall will open a center to speed genetic discoveries into usable therapies, doing some of the riskiest early-stage research in hopes companies then will step in. A new International Rare Diseases Research Consortium is pushing for at least 200 more treatments by 2020, in part by pooling the work of far-flung scientists and families. Rather than starting from scratch, the Food and Drug Administration (FDA) is pointing the way for manufacturers to "repurpose" old drugs for new use against rare diseases, publishing a list of those deemed particularly promising. Also, bipartisan legislation recently introduced in the Senate, called the Creating Hope Act, would offer drug makers another financial incentive—a voucher promising fast FDA evaluation of their next blockbuster drug in return for developing a therapy for a rare or neglected disease that disproportionately affects children. Source: Associated Press/Yahoo! News 4/26/11

FDA Says Merck Drug Successfully Fights Hepatitis

Federal health officials say a highly anticipated drug to treat hepatitis C made by Merck appears to cure more patients in less time than established drugs that have been used for 20 years. However, the agency has questions about how the drug should be combined with older medicines for the maximum effect. The Food and Drug Administration (FDA) posted its review of Merck & Co. Inc.'s boceprevir ahead of a public meeting April 27 to consider whether to approve the medication. The meeting brings to a head more than 15 years of research to find a better therapy for a virus that infects about 3.2 million people in the U.S. On April 28, Vertex Pharmaceuticals Inc. will bring a similar drug called telaprevir before the FDA's panel. Both drugs are considered breakthrough medications with the potential to rack up more than a billion dollars in annual sales. The new drugs would be prescribed as part of a cocktail with the older drugs to help lower viral levels. Source: Associated Press/Yahoo! News 4/25/11

FDA Clears First-of-a-Kind Device for Brain Cancer

Device maker Novocure said in April that the Food and Drug Administration (FDA) approved its first-of-a-kind treatment that fights cancerous brain tumors using electrical energy fields. The FDA approved the device for patients with aggressive brain tumors that have returned after treatment with chemotherapy and other interventions. Patients with recurring brain cancer usually live only a few months. Studies showed that people using the device lived about as long as those taking chemotherapy, roughly six months. However, patients using the device had significantly fewer side effects. For decades, doctors have treated cancer with three methods: drugs, radiation, or surgery. Novocure's NovoTTF device represents a fourth approach. The portable device uses electric fields to disrupt the division of cancer cells that allows tumors to grow and spread. The electric fields have little effect on healthy cells because they divide at a much slower rate, if at all, compared to cancer cells. Source: Associated Press/Yahoo! News 4/15/11

Biotechs’ Autonomy Threatened

Major biotechnology companies, long a staple of the Boston area’s economy, are finding it harder to remain independent. Speaking a week after the sale of Genzyme Corp. left the state’s top biotech firm in the hands of a French buyer, executives at the BD Biotech Conference in Boston said the pressure to merge is being driven by an unforgiving regulatory environment and the failure of the world’s largest pharmaceutical companies to develop new drugs. Not all biotechs shun buyouts. Smaller, underfunded companies welcome suitors, and many venture-backed start-ups are presumed to be for sale at a time when the market for going public remains weak. However, the larger publicly traded biotechnology companies that grew out of area labs and now have hundreds of employees locally also are the focus of attention from would-be buyers, even if they want to remain stand-alones. Source: Boston Globe 4/13/11

Experimental Weight-Loss Drug Seems to Work

Obese patients taking a high dose of an investigational weight-loss pill called Qnexa lost an average of 22 pounds over a year, while also lowering their cholesterol and blood pressure numbers, a new study has found. Qnexa is a combination of two medications: phentermine, the most widely used weight-loss drug in the United States, currently available under a variety of brand names as well as a generic; and topiramate (Topamax), best known as a drug used to ease epilepsy and migraine. Qnexa was recently turned down as a weight-loss aid by the U.S. Food and Drug Administration, because there was not enough data on the risk of birth defects and heart problems related to the drug. Topiramate has been linked to an increased risk for cleft lip/palate in babies born to women who took the drug. The results of the new study, which was funded by Vivus, Qnexa's maker, suggest that "the combination of topiramate and phentermine when administered with some lifestyle counseling could be a valuable treatment for obesity," said lead researcher Dr. Kishore Gadde, an assistant professor of psychiatry and director of the obesity clinical trials program at Duke University Medical Center. Source: HealthDay/Yahoo! News 4/10/11

Progesterone Gel Lowers Risk of Preterm Birth for Some

Daily use of a vaginal gel containing the hormone progesterone in the second half of pregnancy substantially reduces the risk of premature delivery in a specific group of women, according to a new study. Women with a short cervix—the neck-like structure connecting the base of the womb to the vagina—have a 50 percent chance of delivering a baby at least two months early. Between 2 and 3 percent of pregnant women fall into that category, and they are the ones who might benefit from a progesterone gel. “I believe this will change practice. I believe screening for a short cervix and treating it will become routine in pregnancy,” said Roberto Romero, a researcher at the National Institutes of Health’s National Institute of Child Health and Human Development, who led the clinical experiment of 32,000 pregnant women in the United States and nine other countries. Source: Washington Post 4/7/11

FDA Approves AstraZeneca's Thyroid Cancer Drug

The U.S. health regulator has approved AstraZeneca Plc's medullary thyroid cancer drug, vandetanib, the first treatment to be approved for the rare form of cancer. The drug had faced a three-month delay after U.S. regulators, in January, extended the time needed to complete a review. The decision, which was supposed to come by January 7, was pushed to April 7 after the company submitted a risk evaluation and mitigation strategy to the regulators. The drug has been approved to treat patients with nonoperable advanced medullary thyroid cancer, which accounts for 3 to 5 percent of thyroid cancers. Source: Reuters/Yahoo! News 4/6/11

Supreme Court Sides With Investors in Zicam Case

Ruling in March on a case that had drawn the strong interest of the business community, the Supreme Court made it easier for stockholders to sue companies. The court ruled unanimously that a group of investors may proceed with its lawsuit against the makers of the cold remedy Zicam, which is no longer sold, saying the manufacturer should have disclosed that some who used the nasal spray lost their sense of smell. Investors allege that Matrixx Initiatives had been warned about adverse reactions to its most popular drug but had issued statements saying such allegations were “completely unfounded and misleading.” Matrixx said it had no reason to disclose incidents in which users reported a loss of smell because the number was not statistically significant. It asked the court to set a rule that would protect companies from having to disclose such information. But Justice Sonia Sotomayor, writing for the court, said such allegations were part of the mix of information stockholders look at when making investments. The unanimous decision did not provide detailed guidance on what a company must disclose and when. Sotomayor said that the ruling “does not mean that pharmaceutical manufacturers must disclose all reports of adverse events.” Source: Washington Post 3/22/11 For more information, click here.

Patients Take On Expanded Role

Patient activists are stepping up their role in healthcare and drug development, funding research, helping companies set priorities, and banding together over the Internet to share their experiences with everything from doctors to therapeutics. The emerging "participatory medicine" trend, which will have a profound impact on healthcare providers and the biomedical industry, was a focus of the opening sessions at the Massachusetts Biotechnology Council's annual meeting in March. Among the factors empowering patients are the move toward personalized medicine, customizing healthcare based on genetic variability; a growing emphasis on "wellness" in an aging population through disease prevention; and the rise of social media enabling patients and doctors to form online communities. Beyond sharing medical insights, patient advocacy groups are claiming a larger role in underwriting drug research and development, while working with biotechnology and pharmaceutical companies on setting research priorities. Source: Boston Globe 3/22/11

Melanoma Drug Extends Survival in Study

Bristol-Myers Squibb Co.'s eagerly anticipated experimental drug ipilimumab extended survival of previously untreated patients with advanced melanoma in a late-stage study, the company said. Details of how much longer patients who were suffering from the deadly skin cancer lived after taking the drug will be unveiled at a major medical meeting in June. Extending overall survival—the primary goal of the study—is considered the gold standard for cancer drug trials. U.S. health regulators were expected to approve ipilimumab in March based on results of a different study of patients who had received prior treatment for advanced melanoma. In that study, the drug extended survival by an average of four months, which was seen as a major advance for a disease littered with drug failures and for which there are really no effective treatment options. The Food and Drug Administration in November delayed its approval decision to give it more time to review data on the medicine, setting a new action date of March 26. Source: Reuters/Yahoo! News 3/21/11

Editorial: To Improve Drug Safety, FDA Should Check its Mixed-Up Files

Following a number of high-profile drug safety scandals in recent years, the pharmaceutical industry and the Food and Drug Administration (FDA) are spending a great deal of energy trying to uncover, as early as possible, rare side effects that make a drug too risky. Sometimes, the agency asks companies to go back to the drawing board to redesign their studies or expand clinical trials to answer specific concerns about safety, but in some cases, the expanded data already exists in the FDA bureaucracy. There is a vast amount of clinical information sitting in the agency’s archives that could be analyzed and mined to flag worrisome side effects. Multiple years’ worth of clinical trial data from studies of diabetes, high blood pressure, heart disease, Alzheimer’s disease, multiple sclerosis, cancer, and many other conditions could be used to answer important questions about drug safety and efficacy; however, most of those data are unsearchable, existing in the form of paper submissions or unwieldy electronic files that can’t be downloaded and analyzed. As recently as 2007, only 37 percent of drug applications were submitted to the FDA in electronic format. The figure has improved—it was around 70 percent last year—but that means 30 percent of data still comes in as binders full of paper. In the era of the iPad, this is unfathomable. The FDA’s first priority should be to develop a standardized database into which data from past and present clinical trials can be deposited for further analysis. To make such databases useful, it is also imperative that industry transition to a standardized reporting format for all electronic data submissions. Source: Boston Globe 3/20/11

Why Do Clinical Trials Exclude Depressed People?

When the U.S. Food and Drug Administration (FDA) announced in 2009 that Pfizer Inc.'s smoking-cessation drug Chantix would need to carry a restrictive "black box" warning label, the move didn't really surprise the market. The FDA's response came after hundreds of reports of erratic behavior and several suicides. Now, Pfizer faces a civil lawsuit involving at least 1,200 patient complaints. In retrospect, experts say all of this could have been avoided—or at least predicted—had the company's clinical trials been designed differently. Pfizer tested Chantix on thousands of smokers in order to get FDA approval, but the clinical trials excluded people with depression. Doctors have long known that depression often coincides with other disorders, such as addiction and heart disease. Yet clinical trials for these related conditions routinely exclude patients with depression. This strategy may benefit drug companies during the early testing stages, but it can backfire once the treatment is released into the real world. Clinical trials are supposed to mirror the potential patient population as much as possible, but drug companies frequently screen out participants with a history of psychiatric illness, citing safety concerns and fears that their disorder will interfere with the study protocol or cloud the results. Source: Reuters/Yahoo! News 3/18/11

U.S. Clears New Lupus Drug

The first new treatment for lupus in a half-century won U.S. approval on March 9, a milestone for patients with the disabling disease and a potential blockbuster for its tiny biotech maker. Health officials cleared Benlysta, discovered by Human Genome Sciences Inc, to combat the disease that causes the immune system to attack joints and organs and has proved tough to study and treat. For doctors and patients, Benlysta is a welcome advance after decades with few good options and a string of research failures. The Food and Drug Administration (FDA) approved Benlysta for certain patients with active lupus who are receiving standard therapy. It said black patients "did not appear to respond to treatment" with Benlysta in clinical trials, but added that studies "lacked sufficient numbers to establish a definite conclusion." The FDA has required the company to run a postapproval study in blacks. Source: Reuters/Yahoo! News 3/9/11

Influential Research Misses Financial Conflicts

Scientists who review large sets of drug trials for medical journals often ignore financial conflicts that might warp the evidence, according to a new study. That's more than just an academic problem, experts say, because the reviews are considered just about the strongest evidence that medical science can muster. According to findings published in the Journal of the American Medical Association (JAMA), from a total of 29 reviews, or "meta-analyses," of earlier drug trials—culled from top journals like JAMA and The Lancet—only two reported who had funded the original trials included in the review, and none of the reviews mentioned whether the authors reporting on those trials had been paid by drug makers. The new study's authors found that more than two-thirds of the original drug trials that ended up being included in the 29 reviews were funded by pharmaceutical companies, and that only 318 of the 509 trials reviewed gave the conflict-of-interest information in the first place. Source: Reuters/Yahoo! News 3/8/11

Patent Woes Threaten Drug Firms

This year alone, because of patent expirations, the drug industry will lose control over more than 10 megamedicines whose combined annual sales have neared $50 billion. This is a sobering reversal for an industry that just a few years ago was the world’s most profitable business sector, but is now under pressure to reinvent itself and shed its dependence on blockbuster drugs. It casts a spotlight on the problems drug companies now face: a drought of big drug breakthroughs and research discoveries; pressure from insurers and the government to hold down prices; regulatory vigilance and government investigations; and thousands of layoffs in research and development. While industrywide research and development spending has nearly doubled to $45 billion a year over the last decade, the Food and Drug Administration has approved fewer and fewer new drugs. Consumers should see a financial benefit as less expensive generics replace the pricy elite drugs, but may suffer in the long term if companies reduce research and do not produce new drugs that meet the public’s needs. Source: New York Times 3/7/11

They Got a (Sugar) Pill for That

Dianne Sanborn admits that "it didn't make any sense" to take a sugar pill to relieve the severe constipation, abdominal pain, and bloating—attributed to irritable bowel syndrome—that she'd suffered from since college, but eager to try anything, the 64-year-old entered a clinical trial last spring at Beth Israel Deaconess Medical Center, where she was told to take placebo caplets twice a day for three weeks. "Before I took each pill, I had to tell myself that it was going to make me feel better, but I was very skeptical. I certainly knew placebos could work, but only if you didn't know it was a placebo." When her symptoms dissipated three days after she started taking the placebos, however, she became a believer. So did many of the other 36 volunteers who were randomly assigned placebos along with any of their usual treatments in the December study published in the journal PLoS ONE. Nearly 60 percent of them reported an improvement in their irritable bowel symptoms compared to 35 percent of the 43 volunteers who weren't given placebos, but were just told to continue their standard therapy. Other research had already demonstrated that sham treatments could dull pain, alleviate depression, and even quell Parkinson's tremors, but this study was the first to show that placebos have power even when patients know they're taking them. Now placebo researchers are gearing up to figure out ways to move side-effect-free sugar pills into mainstream clinical practice—that's the goal of a placebo-studies program that Harvard Medical School is launching in July with 30 faculty members. Source: Boston Globe 3/7/11

When Optimism is Unrealistic

For almost four decades, researchers and patient advocates have debated the ethics of informed consent in early-phase clinical trials, studies that test only toxicity and dosing and offer little, if any, therapeutic benefit to those enrolled. A major part of the debate has focused on the motivations of patients who participate. Some research on patient motivations has had disturbing ethical implications, indicating that patients may never fully understand the purpose of trials, despite explanations by the researchers. Others have been more reassuring, noting that patients are driven by a sense of altruism and a desire to help others who may one day suffer from the same disease. More recently, a few studies have offered what appears to be the happiest of hypotheses. Patients may simply be optimistic and have strong needs to express hope. ...[However, it] turns out that when it comes to being hopeful, not all optimism is created equal. [E]thicists [who] surveyed 70 patients enrolled in several early-phase cancer trials and asked them about their expectations and understanding of their respective trials....[found that a] solid majority understood that the trials’ purpose was to advance research, not to treat them. But despite clearly understanding the purpose, and limits, of early-phase trials, the patients were also blinded by what researchers called an “unrealistic optimism,” or an optimistic bias, when it came to applying that knowledge to their own particular situations. A majority of patients assumed that the experimental drugs would control their cancer and that they would experience benefits but not complications. In essence, they believed they would fare better than the average patient enrolled in the same trial. Source: New York Times/Doctor and Patient blog 3/3/11

FDA Cracks Down On Unapproved Cold, Cough, Allergy Drugs

Federal health officials announced in early March that they were cracking down on certain prescription cold, cough, and allergy products that had never been approved by regulators. The Food and Drug Administration (FDA) said the products had not been evaluated to make sure they were safe and effective and so about 500 products will be removed from the market. Some of the products are labeled for use for young children. Some are supposed to be time-released, which can be difficult to produce. Companies making the affected products must stop manufacturing them within 90 days and stop shipping the products within 180 days, the agency said. Source: Washington Post/The Checkup 3/2/11

Past Medical Testing On Humans Revealed

Shocking as it may seem, U.S. government doctors once thought it was fine to experiment on disabled people and prison inmates. Such experiments included giving hepatitis to mental patients in Connecticut, squirting a pandemic flu virus up the noses of prisoners in Maryland, and injecting cancer cells into chronically ill people at a New York hospital. Much of this horrific history is 40 to 80 years old, but it is the backdrop for a meeting in Washington this week by a presidential bioethics commission. The meeting was triggered by the government's apology last fall for federal doctors infecting prisoners and mental patients in Guatemala with syphilis 65 years ago. U.S. officials also acknowledged there had been dozens of similar experiments in the United States--studies that often involved making healthy people sick. An exhaustive review by The Associated Press of medical journal reports and decades-old press clippings found more than 40 such studies. At best, these were a search for lifesaving treatments; at worst, some amounted to curiosity-satisfying experiments that hurt people but provided no useful results. Source: Associated Press/Yahoo! News 2/27/11 See also Panel Told No Guarantee Against Unethical Research

FDA Wants More Data On Pfizer-Protalix Drug

U.S. health regulators have asked for additional data to support Pfizer Inc.'s and Protalix Biotherapeutics Inc.'s new drug application for taliglucerase alfa, a treatment for Gaucher disease, a rare genetic disorder. The U.S. Food and Drug Administration (FDA) issued a complete response letter asking for additional data from the companies' switchover trial and long-term extension trial. The main questions raised by the agency relate to clinical, chemistry, and manufacturing controls, Protalix said in a news release. Protalix said FDA did not identify issues in its audit of clinical sites and did not ask for more clinical studies. The company said it will request a meeting as soon as possible to clarify the path to regulatory approval. Source: Reuters/Yahoo! News 2/25/11

Japan Digs Site Linked to World War II Human Experiments

Japan began excavations in February at a former army medical school to search for human remains linked to a notorious World War II program that allegedly conducted biological warfare in China and live experiments on foreign prisoners of war. There is no certainty the excavation will unearth anything, but it is a sign that the government is open to the possibility of facing its long-kept wartime secrets, including the experiments conducted by the military's shadowy Unit 731. Its activities have never been officially acknowledged by the government even though historians and participants have documented them. Source: Associated Press/Yahoo! News 2/21/11

Biotech Industry Says Cuts Will Stifle

The Massachusetts biotech industry says an obscure provision in President Obama’s budget could deal it a "fatal blow" and cost the state untold numbers of jobs; a Bay State hospital says other proposed cuts would devastate its efforts to train pediatricians; and an array of research institutions fear what is coming next from a Republican budget proposal. Now, in response to what some of them consider a misguided assault by Washington, some employers and industry groups from the Bay State are adding their voices to persuade Congress to curtail or eliminate the cuts. Much is at stake, they say, noting that the medical research, biotech, and pharmaceutical industries account for more than 46,000 jobs and $4.1 billion in salaries for Massachusetts residents. Source: Boston Globe 2/19/11

Biogen Reports More Tysabri PML Cases, Deaths

Ten more patients taking Biogen Idec's multiple sclerosis (MS) drug Tysabri have developed the serious brain infection known as PML and there were reports of four more deaths, according to a monthly update by the U.S. biotechnology company. The new cases of potentially fatal progressive multifocal leukoencephalopathy, or PML, were detected between January 7 and February 2, bringing the total number of confirmed cases to 95 and deaths to 20 since the drug was reintroduced into the market in July of 2007, Biogen said. The number of new PML cases per month has been climbing. Four new cases were reported in November and six in December. Tysabri was briefly pulled from the U.S. market over concerns about the brain infection, but was considered to be so effective compared with other available treatments that MS patients considered the risk worth taking and clamored for its return. Source: Reuters 2/18/11

Drug May Slow Growth of Early Prostate Cancer

A new study suggests a way to help men with early, low-risk prostate cancer avoid being overtreated for a disease that in most cases will never threaten their lives. It found that the GlaxoSmithKline drug Avodart can slow the growth of these tumors in men who opt to be monitored instead of having treatment right away. This is the first time that a drug for treating enlarged prostates also has been shown to help treat prostate cancer in a rigorous study. It may persuade more men to choose active surveillance, or "watchful waiting," instead of rushing to have treatments that can leave them with urinary or sexual problems, doctors say. However, the results also show that most of these men do very well with no treatment at all. Source: Associated Press/Yahoo! News 2/16/11

Sanofi-Aventis to Buy Genzyme for More Than $20 Billion

French drug maker Sanofi-Aventis SA agreed to buy Genzyme Corp. with a sweetened $20.1 billion cash offer, plus payments tied to the success of the U.S. biotech group's drugs, the companies said on February 16. The acquisition--which comes nine months after Sanofi-Aventis CEO Chris Viehbacher first put the idea to Genzyme's Henri Termeer--is expected to boost Sanofi-Aventis' earnings from the first year after completion by giving it a new platform in rare diseases. The deal's announcement marks the second-biggest acquisition in biotech history and will help Sanofi-Aventis offset declining revenue from drugs that have lost, or are set to lose, patent protection. Source: Reuters/Yahoo! News 2/16/11

FDA Aims to Accelerate Medical Device Reviews

U.S. federal health officials are proposing a plan that would speed up the approval of innovative medical devices that have the potential to dramatically improve patients' lives. The so-called Innovation Pathway, announced in February by the Food and Drug Administration (FDA), would aim to review first-of-a-kind devices in five months, which is half the time currently spent reviewing most new devices. The FDA has had an accelerated approval program for drugs since 1992, but never one for devices. The initiative comes amid complaints from medical device manufacturers that U.S. review times lag behind other countries. Source: Associated Press/Yahoo! News 2/8/11

FDA Declines to Approve Diet Drug

The U.S. government has unexpectedly rejected what appeared to be the most promising candidate among a class of new diet drugs, wiping out hopes for a new medication to fight obesity anytime soon. Orexigen Therapeutics Inc. said the Food and Drug Administration (FDA) is concerned about the heart side effects of its drug Contrave and will require a new study, a costly undertaking that may prove too burdensome for the small drug maker. The FDA's ruling marks the third rejection of a weight loss drug in recent months, raising questions about whether any new drugs in the class can be made safe enough to win approval. The FDA has not approved a new diet pill in more than a decade. Source: Associated Press/Yahoo! News 2/1/11

Study Finds No Evidence Black Cohosh Damages Liver

Despite reports of liver damage in some women using black cohosh to ease menopause symptoms, clinical trials testing one major brand of this herb have so far found no evidence that it is to blame, according to a research review. Extracts of black cohosh are marketed as a "natural" form of hormone replacement therapy and are most commonly used to treat hot flashes and other symptoms of menopause. For the new study, reported in the journal Menopause, researchers combined the results of five previously published clinical trials of the black cohosh product Remifemin. Together, the studies involved more than 1,100 women who used either this product or a comparison substance--either an inactive placebo or a hormonal medication called tibolone--for three to six months. Overall, the researchers found, 88 women dropped out of the studies, but none did so because of abnormal liver enzymes, a potential sign of liver damage. Source: Reuters/Yahoo! News 1/28/11

Breast Cancer Drug Fails to Extend Survival

An experimental drug for a type of advanced breast cancer being developed by sanofi-aventis failed to extend life or slow disease progression in a late-stage clinical trial, the French drug maker said. The news was particularly disappointing as the drug, BSI-201, or iniparib, did extend survival by an average of almost five months over chemotherapy alone in an earlier mid-stage study of women with metastatic triple-negative breast cancer. However, an analysis of subjects in the 519-patient Phase III study who were undergoing their second or third treatment regimens for the disease did show an improvement in both overall survival and progression-free survival that was consistent with results from the earlier trial, the company said. Source: Reuters 1/27/11

Federal Research Center Will Help Develop Medicines

The Obama administration has become so concerned about the slowing pace of new drugs coming out of the pharmaceutical industry that officials have decided to start a billion-dollar government drug development center to help create medicines. The new effort comes as many large drug makers, unable to find enough new drugs, are paring back research. Promising discoveries in illnesses like depression and Parkinson’s that once would have led to clinical trials are instead going unexplored because companies have neither the will nor the resources to undertake the effort. The job of the new center, to be called the National Center for Advancing Translational Sciences, is akin to that of a home seller who spruces up properties to attract buyers in a down market. In this case, the center will do as much research as it needs to do so that it can attract drug company investment. Under the plan, more than $700 million in research projects already under way at various institutes and centers would be brought together at the new center. But officials hope that the prospect of finding new drugs will lure Congress into increasing the center’s financing well beyond $1 billion. Source: New York Times 1/22/11

Trial in a Vacuum: Study of Studies Shows Few Citations

Science, so the story goes, is a meticulously built edifice. Discoveries balance on ones that preceded them. Research is stimulated by studies that went on before. But what, then, can explain the findings by two investigators at Johns Hopkins University School of Medicine? The researchers, Karen A. Robinson and Dr. Steven N. Goodman, looked at how often published papers on clinical trials in medicine cite previous clinical trials addressing the same question. They report in the January 4 issue of Annals of Internal Medicine what Dr. Goodman describes as “a rather shocking result.” He summarizes: “No matter how many randomized clinical trials have been done on a particular topic, about half the clinical trials cite none or only one of them.” Source: New York Times 1/17/11

Can Johnson & Johnson Get its Act Together?

Many Johnson & Johnson products are in short supply at drugstores and supermarkets because the company's McNeil Consumer Healthcare unit last year recalled about 288 million items, including about 136 million bottles of liquid Tylenol, Motrin, Zyrtec, and Benadryl for infants and children. Johnson & Johnson has had to recall such a variety of products because of quality-control problems across product lines, in multiple factories and in several units last year. Some of its consumer products, for instance, may have contained bits of metal. Others came in bottles with a moldy smell. And some products have gone missing from stores with hardly an explanation. All of this has put the company and its manufacturing under the intense scrutiny of lawmakers and officials at the Food and Drug Administration. Source: New York Times 1/15/11

New Hope for Hepatitis C

There's new hope for an overlooked epidemic: Two powerful drugs are nearing the market that promise to help cure many more people of liver-attacking hepatitis C—even though most who have the simmering infection don't know it yet. Surprisingly, two-thirds of hepatitis C sufferers are thought to be baby boomers who've harbored since their younger, perhaps wilder, years a virus that can take two or three decades to do its damage. What could be a treatment revolution is spurring the government to consider if it's time to start screening aging baby boomers for hepatitis C, just like they get various cancer checks. Today's two-drug treatment for hepatitis C cures only about 40 percent of people with the most common variety of the virus, and causes some grueling side effects. Now major studies show that adding a new drug—either Vertex Pharmaceuticals' telaprevir or Merck & Co.'s boceprevir—can boost those cure rates as high as 75 percent; and they allow some people to cut treatment time in half, to six months, thus lessening how long they must deal with those side effects. If the Food and Drug Administration approves the drugs—a decision widely expected this summer—they would be the first that work by directly targeting the hepatitis C virus. Source: Associated Press/Yahoo! News 1/18/11

Knowledge of Trial Process Can Drive Away Volunteers

The more a potential trial volunteer learns about the clinical trial process, the less likely he or she is to agree to participate. That finding stunned Patrina Caldwell, lead author of a report on the topic in a recent issue of PLoS Medicine. For the study, Caldwell and colleagues from the University of Sydney, New South Wales, Australia, culled through reference lists and scrutinized recruitment strategies used in 37 studies in which 18,812 of at least 59,354 people approached agreed to participate in a clinical randomized trial. The recruitment strategies they examined were divided into four groups: novel trial designs, recruiter differences, incentives, and provision of trial information. However, Caldwell’s research also showed that understanding more about their health conditions and the impact on them—and perhaps the lack of information that currently exists about the subject—may have a motivating effect on people considering participation in a clinical trial. Source: Clinical Trials Today 1/10/11

At Novartis, a Winning Formula

When drug maker Novartis AG tapped a Massachusetts General Hospital cardiologist to run its global research operations in 2002, some company insiders chafed at his ambition to reinvent drug discovery in a setting nearly 4,000 miles from the company's headquarters in Basel, Switzerland. Mark C. Fishman, the Novartis research chief, got doctors involved in early-stage research, kept marketers at bay, and focused on medicines that might treat smaller numbers of patients, but also yield insight into ways to fight other diseases. Nearly nine years later, while other companies are scaling back drug development, Fishman, 58, is overseeing an expansion of Novartis research in Boston and around the world. As president of the Novartis Institutes for BioMedical Research, he has emphasized more science, struck fresh academic and commercial partnerships, and pioneered what he calls a "new grammar" of drug development in fields ranging from cancer and muscle growth to cardiovascular and infectious diseases. Fishman’s strategy appears to be working: Novartis won Food and Drug Administration approval for four new drugs last year, the company has nearly 50 drugs in late-stage clinical trials, and in recent years it has roughly doubled the number of drugs that have made it through early-stage trials. Source: Boston Globe 1/4/11

Doctor Behind Study Linking Vaccine to Autism Accused of "Deliberate Fraud"

An in-depth investigation just published in a prominent medical journal alleges that a decade-long effort to link childhood vaccinations with autism was really an elaborate hoax perpetuated by a British doctor who has since been banned from practicing medicine in that country. The doctor's original research, first published in 1998, turned many parents away from immunizing their children, which some experts now link to recent outbreaks of illnesses that had once been well under control. "The MMR [measles-mumps-rubella vaccine] scare was based not on bad science but on a deliberate fraud," Dr. Fiona Godlee, editor-in-chief of the BMJ, which published details of the new investigation on January 5, said in a statement. "Such clear evidence of falsification of data should now close the door on this damaging vaccine scare." Source: HealthDay/Yahoo! News 1/5/11

Limits on Test Drugs Add to Patients’ Ordeals

Patients' lives can be thrown into upheaval by decisions about who can receive experimental drugs. Desperate to regain their health, they might be willing to try almost any treatment, even if it is unproven. However, regulators and companies also must make sure a drug that is initially effective does not later prove to be dangerous. That requires years of clinical trials, and to make trials scientifically valid, some patients enrolled in them—no matter how ill—don’t receive the drug being tested. In certain cases, "compassionate use" of experimental drugs is permitted through expanded access programs, but researchers worry that if the health of patients taking one experimental drug worsens, they might leave the trial to get another treatment by signing up for an expanded access program. That would compromise the integrity of the first study and further bog down the approval process for that drug. Such scientific concerns rankle patients who only want to get a drug that might work where others have failed. Source: Boston Globe 1/5/11

Pain Drug Put on Clinical Hold by FDA

Regeneron Pharmaceuticals Inc. said in late December that U.S. regulators placed the company's experimental pain drug for osteoarthritis on clinical hold after a patient in another company's trial developed a serious bone disorder. Regeneron said it and its partner, Sanofi-Aventis SA, were informed by the U.S. Food and Drug Administration (FDA) that the drug, REGN475/SAR164877, was being placed on clinical hold after a patient in another company's trial developed avascular necrosis of a joint. The condition is caused when bone tissue dies due to lack of blood supply, eventually causing the bone to collapse. Regeneron's drug is designed to work by blocking a protein called nerve growth factor (NGF) that is associated with pain. An NGF-inhibitor under development by Pfizer Inc. was put on hold earlier in 2010 after some patients required joint replacement surgery. The FDA believes its safety concerns could affect the entire anti-NGF class. Source: Reuters/Yahoo! News 12/27/10

Experiments Test if Implant Can Block Sleep Apnea

Loud snoring may do more than irritate your spouse: It can signal sleep apnea, depriving you of enough zzzz's to trigger a car crash, even a heart attack. Now scientists are beginning to test if an implanted pacemaker-like device might help certain sufferers, keeping their airways open by zapping the tongue during sleep. By the end of January, Minneapolis-based Inspire Medical Systems plans to begin enrolling 100 apnea patients in a key study in the U.S. and Europe to see if so-called hypoglossal nerve stimulation really could work. Two competitors are developing similar implants: ImThera Medical of San Diego says it hopes to begin U.S. studies later next year, and Apnex Medical of St. Paul, Minn., has announced some small-scale testing. Source: Associated Press/Yahoo! News 12/27/10

Enlisting the Dying for Clues to Save Others

Research on a new breed of cancer drugs that work by blocking the particular genetic defect driving an individual tumor’s out-of-control growth relies on pinpointing which new genetic alteration is driving the cancer when it evades the blockade—as it nearly always does—and coming up with similarly tailored drugs that may be able to hold it off for longer. The crucial evidence resides in the tumor cells of patients who have relapsed, but the need to ask those who know their time is short to undergo another invasive procedure in the name of science is just one obstacle to what many oncologists see as the best chance to give future cancer patients a more permanent reprieve. A regulatory process that can take years to approve a drug for sale means that instead of thousands of patients to draw on, only the few hundred who receive the drug through clinical trials are available for such research. Despite the challenges, progress is emerging from a complex mix of academic ambition, collaboration and competition among scientists, and, especially, the willing participation of dying patients. Source: New York Times 12/26/10

Editorial: FDA's Avastin Strategy Shows Courage

The Food and Drug Administration (FDA) is preparing to withdraw its approval for using Avastin, a tumor-slowing drug, to treat advanced breast cancers that have spread to other parts of the body. It was a reasonable decision, based on scientific evidence. The drug has failed to extend lives in clinical trials, and it carries a small risk of devastating, sometimes fatal, side effects. We believe the FDA has shown courage in following the scientific evidence on this highly emotional issue. Even some advocacy groups for breast cancer patients have applauded the agency’s decision for making clear that the drug does not work very well. Genentech plans to request a hearing with the FDA to argue the case for retaining Avastin’s status as an approved breast cancer treatment. It should focus on proposing ways to identify the subset of women who can really benefit from Avastin. Source: New York Times 12/25/10

Intercell Kills Diarrhea Vaccine After Study

Austrian biotech company Intercell said it won't develop its travelers' diarrhea vaccine candidate because the drug failed in clinical trials. The company said that the patch-based vaccine candidate failed to meet efficacy endpoints to protect against enterotoxigenic E. coli mediated diarrheal infections in pivotal, randomized, and placebo-controlled efficacy studies. While it doesn't plan to develop the vaccine anymore, Intercell said the studies support the continued investigation of its patch technology for future vaccines. Source: Reuters/Yahoo! News 12/12/10

Citing Liver Damage, Pfizer Withdraws Thelin

Pfizer Inc. said it is pulling its blood pressure drug Thelin off the market and stopping all clinical trials because the drug can cause fatal liver damage. Thelin is sold in the European Union, Canada, and Australia as an oral treatment for severe pulmonary arterial hypertension, or high blood pressure in the pulmonary artery. Pfizer said two patients who were taking Thelin died during a clinical trial, and a review of data from clinical studies and postmarketing reports showed a new link to liver injury. Liver damage was a known side effect of Thelin and similar drugs, the company said, but the review uncovered a link to liver damage that was not tied to identifiable risk factors. It said the problem was unlikely to be detected by routine monitoring, and in some cases, the problems did not go away after patients stopped taking Thelin. Source: Associated Press/Yahoo! News 12/10/10

Bone Drug Zometa Flops in Breast Cancer Study

One of the most promising new approaches for fighting breast cancer took a stunning setback when a major study showed that a bone-building drug did not stop cancer from returning or extend life for most women fighting the disease. However, the drug Zometa did seem to help certain postmenopausal women. Its maker, Novartis AG, is considering further study, but will suspend plans to expand it beyond its current use as a treatment for patients whose cancer has spread to the bone. Hopes that osteoporosis drugs could also prevent cancer soared after a study two years ago found Zometa cut the risk of cancer recurrence by 30 percent in younger women forced into early menopause by hormone treatments they received. The excitement grew last year, when a large study found that women who were not cancer patients and were taking daily bisphosphonate pills to prevent bone problems were about one-third less likely to develop breast cancer. The new study was meant to be definitive. It tested Zometa in 3,360 women of all ages in seven countries who had breast cancer that had spread to lymph nodes. All received standard cancer treatments, and half also got periodic infusions of Zometa for five years. After five years of followup, about 400 women in each group had died or suffered a recurrence. However, among the roughly 1,100 women who were at least five years past menopause when the study began, Zometa cut the risk of recurrence by about 27 percent and improved survival odds by about 29 percent. Source: Associated Press/Yahoo! News 12/9/10

Consumers, Companies See Room to Improve at FDA

Companies and consumers both feel the U.S. Food and Drug Administration (FDA) does a good job, but high-profile drug recalls have damaged the average American's faith in the agency, according to a report published in late November. Executives at 50 medical companies think FDA has improved it relationships with them, but say the agency is not keeping up with advances in technology, the survey by PricewaterhouseCoopers LLP and Biocom, an association of 550 California life sciences companies, found. The report is available here. More than 50 percent of consumers said they think FDA does a good job, but 36 percent said they have lost confidence in the agency over the past two years as a result of high profile safety concerns and product recalls. Seventy percent of consumers disapprove of having companies help pay for FDA's work. Only 36 percent knew that industry helps pay for FDA, and just 68 percent fully understood the agency is paid for by taxes. Source: Reuters/Yahoo! News 11/30/10

FDA Reviews Two New Drugs to Reduce Prostate Cancer

Health regulators in late November said that two drugs from GlaxoSmithKline and Merck reduce the risk of prostate cancer in men, but scientists questioned the drugs' overall benefit, since the tumors they prevent are usually not life-threatening. The reviewers for the Food and Drug Administration (FDA) also complained that the companies only studied a small number of African-American men, who are at high risk for prostate cancer. GlaxoSmithKline has asked the FDA to approve its drug Avodart for a new use against prostate cancer, after research showed a 23 percent reduction in low-grade prostate tumors among men taking the drug. Merck achieved similar results with its drug Proscar, and is asking the FDA to approve labeling about its drug's benefits in reducing prostate cancer risks. Both drugs are already approved to treat enlarged prostate. Source: Associated Press/Yahoo! News 11/29/10

Making Pills Passé

Whether it’s a headache or a sore knee, a toothache or a strep throat, people are used to taking their medicine in pill form. However, to scientists, such drugs can be hard to swallow because the pill is a blunt tool: As it wends its way through the body, such medication may wreak side effects or fail to make it to its intended destination efficiently. Now, with implantable pumps, tiny particles of drug, and novel materials that can release medication at a controlled rate, researchers are experimenting with new channels to deliver drugs directly to areas of the body where they can be most effective. Source: Boston Globe 11/29/10

Drug Makers’ Payments Detailed

Massachusetts health officials have published online the most comprehensive state database in the country listing payments drug companies and medical device makers have made to doctors, nurses, pharmacists, hospitals, and other healthcare providers. The report lists $35.7 million in payments from hundreds of companies in the second half of 2009, for speaking, consulting, food, educational programs, marketing studies, and charitable donations. About half of that money, or $16.4 million, went to physicians. Companies do not have to disclose funding for research meant to answer a legitimate scientific question, but they do have to report payments for marketing studies that are designed to promote products. Source: Boston Globe 11/23/10

Treatments for Retinas Make Gains

Elderly people losing their vision from age-related macular degeneration might one day have a treatment option that requires fewer injections into the eye than the standard drug now used. In testing, an experimental drug being developed by Regeneron Pharmaceuticals, when injected every eight weeks, proved as effective as the standard treatment, Lucentis from Genentech, which was injected every four weeks. The findings are from two clinical trials. In a separate development, Advanced Cell Technology was expected to announce that it has won regulatory approval to test a therapy derived from human embryonic stem cells in people with Stargardt’s macular dystrophy, another retina disease. It is only the second trial of a therapy derived from human embryonic stem cells to be cleared by the Food and Drug Administration. Source: New York Times 11/22/10

Vertex’s New Drug Tackles Cystic Fibrosis at its Roots

A small clinical trial of an experimental drug made by Cambridge-based Vertex Pharmaceuticals has successfully targeted the root cause of cystic fibrosis, generating cautious excitement among doctors and patients. Despite the limitations of the study, designed to test the safety of the drug over a short time frame, a number of researchers said the new work represents the most significant advance against the disease since the genetic defect that causes it was discovered in 1989. The compound, VX-770, acts directly on a protein that ordinarily acts as a channel for transporting salt and water, but malfunctions in cystic fibrosis. Vertex is testing the compound in a larger study designed to more broadly assess its effectiveness. Source: Boston Globe 11/18/10

Next Big Thing? Big Cholesterol Drop with New Drug

An experimental Merck drug safely boosted good cholesterol to record highs while dropping bad cholesterol to unprecedented lows in a study that stunned researchers and renewed hopes for an entirely new way of lowering heart risks. The drug, anacetrapib, won't be on the market anytime soon. It needs more testing to see if its dramatic effects on cholesterol will translate into fewer heart attacks, strokes and deaths. Merck & Co. announced a 30,000-patient study to answer that question and it will take several years. Anacetrapib helps keep fat particles attached to HDL, which carries it in the bloodstream to the liver to be disposed of. The Merck-sponsored study tested it in 1,623 people already taking statins, and was too small to tell whether anacetrapib lowered deaths, heart attacks, or other heart problems, but the trend was in the right direction. Source: Associated Press/Yahoo! News 11/17/10

U.S. Panel Backs Dendreon's Provenge for Medicare

A U.S. advisory panel backed Dendreon Corp.'s Provenge prostate cancer therapy on November 17, telling the Medicare insurance program for the elderly that available data showed it could help patients. A majority of the outside advisers said there was enough evidence to show that the therapeutic vaccine helped patients live longer. The agency is weighing whether to pay for the product nationwide. Centers for Medicare and Medicaid Services officials will take the panel's advice into account in making a final ruling, expected next year. Several health insurers have already agreed to pay for the vaccine, which does not prevent cancer, but fights the tumors. Provenge was approved for the U.S. market in April to treat men with advanced prostate cancer after company data showed it help men live another 4.1 months on average. Source: Reuters/Yahoo! News 11/17/10

Pfizer Arthritis Drug Succeeds in Late-Stage Trial

A closely watched experimental drug for rheumatoid arthritis being developed by Pfizer Inc. significantly reduced symptoms and improved physical function, according to data from a late-stage clinical trial. The drug, tasocitinib, met two of three primary goals of the 611-patient Phase III study at two tested doses, compared to a placebo, the data show. On the third primary goal, tasocitinib demonstrated a numerically higher measure of disease remission at three months than placebo, but that measure did not reach statistical significance. "This is the first oral medication for rheumatoid arthritis that has had a successful Phase III study this century," Dr. Roy Fleischmann, the study's primary investigator, said in a telephone interview. "It's an oral pill taken every day, rather than an injection. It seems to work similar to biologics and it's certainly more convenient for patients." Source: Reuters/Yahoo! News 11/7/10 

Off-Protocol Therapy May Impact Clinical Trial Accrual

Most recent oncology randomized controlled trials evaluate drugs that are available "off-protocol therapy" in the United States, and this can adversely impact trial enrollment, according to a study published online in the Journal of Clinical Oncology. Erika P. Hamilton, MD, of Duke University in Durham, N.C., and colleagues searched the medical literature from 2006 to 2008 for reports on Phase III medical oncology clinical trials, and used U.S. Food and Drug Administration approval data to determine the availability of the therapies outside of clinical trials (off-protocol therapy). The researchers found that, among the 172 trial reports reviewed, 108 trials (63 percent) evaluated drugs that were available for off-protocol therapy at the start of the trial, while the investigational drugs became available for off-protocol therapy during an additional 19 trials (11 percent). For the trials at U.S. sites, time to accrual was longer (41 versus 22 months) and not as efficient (8.8 versus 22.7 patients per month) when off-protocol therapies were available. Furthermore, although 47 percent of experimental therapies proved superior for at least one major clinical outcome and 27 percent improved survival, 66 percent of trials reported at least one serious toxicity. Source: HealthDay News/Doctors Lounge 11/1/10 See also The Perils of Taking Experimental Cancer Drugs Reuters/Yahoo! News 10/26/10 

Omega-3 Pills Fail to Work in Alzheimer's Patients

Omega-3 pills promoted as boosting memory did not slow mental and physical decline in older patients with Alzheimer's disease, a big disappointment in a multimillion-dollar government-funded study. "We had high hopes that we'd see some efficacy but we did not," said Dr. Joseph Quinn, an author of the $10 million study and a researcher at Oregon Health and Science University. The results with pills containing DHA, an omega-3 fatty acid, highlight "the continued frustration over lack of effective interventions" for the memory-robbing disease, an editorial said, published with the study in the Journal of the American Medical Association. The new research involved nearly 300 men and women aged 76 on average with mild-to-moderate Alzheimer's disease. They were randomly assigned to take either DHA pills or dummy pills daily for 18 months. Results were similar in both groups; DHA provided no benefits in slowing Alzheimer's symptoms. The pills also didn't work even in a subgroup of participants with the mildest Alzheimer's symptoms. Source: Associated Press/Yahoo! News 11/2/10 

Wrapped in Data and Diplomas, It’s Still Snake Oil

Ben Goldacre, author of Bad Science: Quacks, Hacks, and Big Pharma Flacks, is exasperated. He’s not exactly angry—that would be much less fun to read—except in certain circumstances. He is irked, vexed, bugged, ticked off at the sometimes inadvertent (because of stupidity) but more often deliberate deceptions perpetrated in the name of science. And he wants you, the reader, to share his feelings. You’ll get a good grounding in the importance of evidence-based medicine (the dearth of which is a “gaping” hole in our culture). You’ll learn how to weigh the results of competing trials using a funnel plot, the value of meta-analysis, and the Cochrane Collaboration. He points out common methodological flaws: failure to blind the researchers to what is being tested and who is in a control group, misunderstanding randomization, ignoring the natural process of regression to the mean, the bias toward positive results in publication. “Studies show” is not good enough, he writes: “The plural of ‘anecdote’ is not data.” Source: New York Times 11/1/10 

FDA Rejects Diet Drug Qnexa

Federal health regulators have decided not to approve an experimental diet pill called Qnexa, which had been touted by many experts as the most promising weight-loss drug in more than a decade. The drug's maker, Vivus Inc., said in a late October statement that the Food and Drug Administration (FDA) declined to approve the drug in its present form. The agency asked for more study results and additional information on its possible health risks, including major cardiovascular events and risks for women of childbearing potential. The FDA did not ask for new clinical studies, but more may be required if the agency's concerns aren't addressed, Vivus said. The company plans to respond to the FDA in about six weeks. Source: Associated Press/Yahoo! News 10/29/10 

FDA Rejects Arena Pharma Diet Drug

Arena Pharmaceuticals Inc. on October 23 said the Food and Drug Administration (FDA) rejected the company's application for lorcaserin, one of three drugs seeking to become the first new FDA-approved prescription weight loss drug in more than a decade. The federal agency's rejection came after an FDA panel of experts on September 16 recommended against approving lorcaserin in a 9-5 vote. Panelists raised concerns about tumors seen in rats in early-stage testing, one of the factors that Arena Pharmaceuticals said the FDA had cited in a letter responding to the company's application. A group of Arena investors later launched a campaign arguing that the FDA panel's review had relied on faulty scientific data. Arena said on Saturday that the FDA determined "that it cannot approve the application in its present form." In addition to citing safety concerns about tumors, the FDA's letter said the agency found that lorcaserin's weight loss efficacy "in overweight and obese individuals without type 2 diabetes is marginal," Arena said. Arena said the letter stated that the FDA may require additional clinical studies if the company cannot provide further evidence to address the concern about tumors. Source: Associated Press/Washington Post 10/23/10

Merck Shingles Vaccine Effective in Adults in 50s

In a huge study aimed at broadening the use of Merck & Co's shingles vaccine, Zostavax reduced the incidence of the painful disease by 70 percent compared to a placebo in adults in their 50s, the company said. The 22,439-subject study monitored adults aged 50 to 59 for at least one year after receiving either the vaccine or a placebo, with 30 cases of shingles occurring in the Zostavax group compared to 99 cases in the placebo group. That translates into a reduction in incidence of shingles of 69.8 percent for Zostavax over placebo. Based on the robust results, Merck said it has applied to the U.S. Food and Drug Administration for an expanded approval of Zostavax to prevent shingles. The vaccine is currently approved to prevent shingles, also known as herpes zoster, only in people aged 60 and older. In that population, the vaccine has been shown to cut incidence of shingles by 51 percent. Source: Reuters/Yahoo! News 10/21/10

Cancer Trials Suspended for New Patients

Boston's Beth Israel Deaconess Medical Center temporarily closed all its cancer trials to new patients in October, after audits found that several researchers had not properly submitted patient data to the committee that oversees oncology research and to trial sponsors. "Some of our clinical trials were not running with the high standards that we expect," Randy Mason, vice president of research operations, wrote in an e-mail to hospital executives and physician leaders. He described the lapses as involving documentation and reporting compliance and said the hospital has not found any "issues of patient harm." Care for patients already enrolled in the hospital’s 285 oncology trials is continuing without disruption, he said. Between 100 and 125 of those trials had been accepting new patients, executives said. "Some of the serious adverse events" experienced by patients enrolled in one trial, such as their disease worsening, were submitted to oversight authorities "later than required," an official said. Source: Boston Globe 10/21/10

FDA Seeks More Data on Diabetes Drug

U.S. health regulators declined on October 19 to approve a diabetes drug being developed by Amylin Pharmaceuticals Inc.'s and Eli Lilly and Co., citing the need for further studies, including the drug's effect on heart rate. The companies said they aim to reply to the Food and Drug Administration (FDA) by the end of next year. They expect the next review of the drug, Bydureon, to have a six-month clock—meaning a final FDA decision could come by mid-2012. The FDA requested in a "complete response letter"—a thorough study of the impact of the drug's main component, exenatide, on heart muscle, Amylin and Lilly said in a statement. In addition, the agency has requested results from an ongoing study of the drug. Source: Reuters/Yahoo! News 10/19/10

Prescription for Prestige: Drug Firms’ Speaking Fees Flow to Harvard Doctors

The Harvard brand, unrivaled in education, is also prized by the pharmaceutical industry as a powerful tool in promoting drugs. Its allure is evident in a new analysis of all publicly reported industry payments to physicians. Doctors and researchers affiliated with Harvard Medical School collected 45 percent of the $6.3 million given to Massachusetts doctors in 2009 and 2010 by seven pharmaceutical companies that disclosed their payments for parts of those years. The money was mostly for talking to other physicians about the companies’ drugs and the diseases they treat, but also for consulting on research and marketing. The proportion of money going to Harvard doctors underscores why the medical school and its affiliated hospitals, concerned that certain speaking fees can compromise the independence of doctors, are clamping down on such payments. Many hospitals and medical schools continue to permit doctors to participate in company speakers bureaus, and even at medical centers that largely ban the practice, the analysis—by ProPublica, a nonprofit, online investigative reporting organization, and the Globe—found spotty enforcement. Source: Boston Globe 10/19/10 For a related editorial, click here.

When Drugs Cause Problems They are Supposed to Prevent

In the past month, the Food and Drug Administration (FDA) has concluded that, in some cases, two types of drugs that were supposed to be preventing serious medical problems were, in fact, causing them. One is bisphosphonates, which is widely used to prevent the fractures, especially of the hip and spine, that are common in people with osteoporosis. Those drugs, like Fosamax, Actonel, and Boniva, will now have to carry labels saying they can lead to rare fractures of the thigh bone, a surprising new discovery that came after another surprise—that they can cause a rare degeneration of the jawbone. The other is Avandia, which is widely prescribed for diabetics, whose disease puts them at risk for heart attacks and heart failure. Two-thirds of diabetics die of heart problems, and a main reason for taking drugs like Avandia is to protect them from that. However, now the FDA and drug regulators in Europe are restricting Avandia’s use because it appears to increase heart risks. Something new is happening, said Daniel Carpenter, a government professor at Harvard who is an expert on the drug agency. The population is aging, many have chronic diseases; and companies are going after giant markets, huge parts of the population, heavily advertising drugs that are to be taken for a lifetime. However, the way drugs are evaluated, with the emphasis on shorter-term studies before marketing, is not helping, Dr. Carpenter said. “Here is a wide-scale institutional failure,” he said. “We have placed far more resources and requirements upon premarket assessment of drugs than on postmarket.” The difficulty is in figuring out how to assess the safety of drugs that will be taken for decades, when the clinical trials last at most a few years. Source: New York Times 10/16/10

Geron Tests Stem Cell Treatment on Patient

Geron Corp. has begun testing an embryonic stem cell treatment on a patient with spinal cord injuries, marking the first time such a medical therapy has been used on a human in a government approved study. The company said it enrolled the first patient in the early-stage study, which will look at the safety of the treatment and how well the patient can tolerate it. "When we started working with human embryonic stem cells in 1999, many predicted that it would be a number of decades before a cell therapy would be approved for human clinical trials," Dr. Thomas B. Okarma, Geron's president and chief executive officer, said in a statement. While a milestone in the technology, the drug candidate is still a long way from being proven and reaching the market. The company has said it plans to enroll eight to 10 patients in the study at sites nationwide. The trial will take about two years, with each patient being studied for one year. The therapy will be injected into the patients' spines one to two weeks after they suffer an injury between their third and 10th thoracic vertebrae, or roughly the middle to upper back. Later trials would include patients with less severe spinal injuries and damage to other parts of the spine. Source: Associated Press/Yahoo! News 10/11/10 

Sanofi-Aventis to Reduce U.S. Workforce by 1,700

Sanofi-Aventis, the world's fourth-biggest drug maker, said on October 8 it is eliminating 1,700 jobs in its U.S. pharmaceutical business in a restructuring triggered by growing generic competition and other factors. The layoffs at Sanofi come as the entire pharmaceutical industry struggles to compete with increasing, lower-cost generic drug options. The layoffs amount to about 25 percent of the workers in the company's U.S. pharmaceutical business, and will primarily hit sales representatives around the country and administrative staff at Sanofi's American headquarters in Bridgewater, N.J. About 1,400 sales staff will be laid off, as well as about 300 staff in various administrative jobs. Source: Associated Press/CNBC.com 10/8/10 

Side Effects May Include Lawsuits

For decades, antipsychotic drugs were a niche product. Today, they’re the top-selling class of pharmaceuticals in America, generating annual revenue of about $14.6 billion and surpassing sales of even blockbusters like heart-protective statins. While the effectiveness of antipsychotic drugs in some patients remains a matter of great debate, how these drugs became so ubiquitous and profitable is not. Big Pharma got behind them in the 1990s, when they were still seen as treatments for the most serious mental illnesses, like hallucinatory schizophrenia, and recast them for much broader uses, according to previously confidential industry documents that have been produced in a variety of court cases. Recent government warnings say the drugs may be fatal to some older patients and have unknown effects on children. The new generation of antipsychotics has also become the single biggest target of the False Claims Act, a federal law once largely aimed at fraud among military contractors. Every major company selling the drugs—Bristol-Myers Squibb, Eli Lilly, Pfizer, AstraZeneca, and Johnson & Johnson—has either settled recent government cases for hundreds of millions of dollars or is currently under investigation for possible healthcare fraud. Source: New York Times 10/2/10 

Wellesley Professor Unearths a Horror: Syphilis Experiments in Guatemala

Picking through musty files in a Pennsylvania archive, a Wellesley College professor made a heart-stopping discovery: U.S. government scientists in the 1940s deliberately infected hundreds of Guatemalans with syphilis and gonorrhea in experiments conducted without the subjects’ permission. Medical historian Susan M. Reverby happened upon the documents four or five years ago while researching the infamous Tuskegee syphilis study and later shared her findings with U.S. government officials. The unethical research was not publicly disclosed until October 1, when President Obama and two Cabinet secretaries apologized to Guatemala’s government and people and pledged to never repeat the mistakes of the past—an era when it was not uncommon for doctors to experiment on patients without their consent. Even so, Reverby found in the files a story of almost singular exploitation and deception, conducted in a foreign land because, the nation’s surgeon general at the time acknowledged, it could not have been done in the United States. The U.S. government has asked the Institute of Medicine to conduct a review of the experiments. Separately, a presidential bioethics commission will charter an international panel of specialists to explore the current state of medical research on humans around the world and ensure that the atrocities of the past cannot recur. Source: Boston Globe 10/2/10 See also MSNBC.com, New York Times, National Public Radio

Novartis Gains FDA Approval for New MS Drug

Federal health regulators have approved the first pill to treat the underlying causes of multiple sclerosis (MS), a debilitating nervous system disorder that has traditionally been treated with injectable drugs. The Food and Drug Administration (FDA) approved Swiss drug maker Novartis' treatment Gilenya to reduce relapses in patients with MS, who experiences loss of balance, muscle spasms, and other movement problems. There is no cure for the disease, but steroids can reduce the duration and severity of symptoms in the short term, and seven treatments on the market have had success in reducing recurrence of symptoms. All involve daily or regular injections, which doctors say discourages some patients from keeping up with their treatment. Source: Associated Press/Yahoo! News 9/22/10

Editorial: "Off label," But Not Off Target?

Doctors rightly treasure the freedom to prescribe a drug for purposes beyond those sanctioned by the Food and Drug Administration. But something’s gone awry in the drug-approval system when so-called “off-label’’ uses begin to predominate—especially in cases where there’s little evidence of the drug’s efficacy. ...Among the most popular drugs, an estimated 20 percent of prescriptions are off-label. In oncology, the figure is thought to be much higher, at 50 to 75 percent of all prescriptions. ...These uses are often justified. It can take years to prove that a drug designed for disease A is also helpful for disease B, even after some medical evidence surfaces in favor of the new use. Patients with cancer and other life-threatening diseases may have little time to lose. But those benefits need to be measured against the health and economic consequences of prescription practices that are backed by little medical evidence. ...[O]nce off-label uses become standard practice, manufacturers have little reason to sponsor clinical studies testing whether their drugs actually work in those unproven settings. The federal stimulus law appropriated $1.1 billion to support research that compares treatments that have not been rigorously studied side-by-side. Given the prevalence of off-label prescription in some diseases...Washington must fund studies evaluating whether any of these off-label uses are warranted. The government—not to mention insurers and employers—could help patients and save millions of healthcare dollars not just by identifying ineffective uses, but also by validating those that do help. Source: Boston Globe 9/21/10

Spiriva as Good as Serevent in Asthma Study

Researchers say they've found a possible new treatment for adults with hard-to-control asthma. Their discovery, however, came at a price. Scientists of a U.S. government-funded asthma study had to spend nearly $1 million of taxpayers' money after British drug maker GlaxoSmithKline (GSK) declined to donate its asthma drug and look-alike dummy medicine for the study, which compared two other treatments. Editors of the New England Journal of Medicine, which published the study, chastised GSK, saying its actions made the research harder and more expensive to do. Drug companies aren't required to supply their medicines for study, but they often do. "In the end, the study results provided the truth"—the drug, Boehringer Ingelheim's Spiriva, was as good as GSK's Serevent, they wrote. The $5.3 million study was funded by the National Heart, Lung, and Blood Institute. A researcher involved in the study said it's harder to get drug companies to donate their medicine for research compared to a decade ago: "Now more drug companies are more likely to ponder whether a trial could help them in the marketplace" and decline to provide their products for studies. Source: Associated Press/Yahoo! News 9/19/10

New Drugs Stir Debate on Rules of Clinical Trials

Last year, when two cousins each learned that a lethal skin cancer called melanoma was spreading rapidly through his body, the young men found themselves with the shared chance of benefiting from a recent medical breakthrough. Only months before, a new drug had shown that it could safely slow the cancer’s progress in certain patients. Both cousins had the type of tumor almost sure to respond to it. And major cancer centers, including the University of California, Los Angeles, were enrolling patients for the last, crucial test that regulators required to consider approving it for sale. The tumors in the cousin who was diagnosed first stopped growing after two months of taking the pills. But when the other cousin was admitted to the trial, he was assigned by a computer lottery to what is known as the control arm. Instead of the pills, he was to get infusions of the chemotherapy drug that has been the notoriously ineffective recourse in treating melanoma for 30 years. Even if it became clear that the chemotherapy could not hold back the tumors advancing into his lungs, liver, and, most painfully, his spine, he would not be allowed to switch, lest it muddy the trial’s results. Controlled trials have for decades been considered essential for proving a drug’s value before it can go to market. But the continuing trial of the melanoma drug, PLX4032, has ignited an anguished debate among oncologists about whether a controlled trial that measures a drug’s impact on extending life is still the best method for evaluating hundreds of genetically targeted cancer drugs being developed. Critics of the trials argue that the new science behind the drugs has eclipsed the old rules—and ethics—of testing them. They say that in some cases, drugs under development may be so much more effective than their predecessors that putting half the potential beneficiaries into a control group, and delaying access to the drug to thousands of other patients, causes needless suffering. Source: New York Times 9/18/10

Antibiotics Caught in Stalemate Over Clinical Trials

Drug companies are abandoning the antibacterial business, citing high development costs, low return on investment, and, increasingly, a nearly decade-long stalemate with the Food and Drug Administration (FDA) over how to bring new antibiotics to market. At the core of the problem is a regulatory impasse over whether drug companies seeking FDA approval for antibiotics should be required to run much more stringent clinical trials. The FDA says yes, citing advances in the science of clinical trial design and a series of humiliations involving trials for drugs the agency had approved. But the pharmaceutical industry and some infectious-disease doctors say the proposed rules will make it so difficult and expensive to gain approval for new antibiotics that the few remaining companies will abandon the field altogether. Source: Chicago Tribune/Richmond Times-Dispatch

FDA Delays Decision on Breast Cancer Drug Avastin

Drug maker Roche said in mid-September that U.S. health regulators will take more time to review its drug Avastin for breast cancer, a use that has generated vigorous debate among cancer specialists and patients. The company said in a statement that the Food and Drug Administration (FDA) extended its review of the drug by 90 days, or until December 17. The FDA granted Avastin accelerated approval for breast cancer in 2008, based on a study suggesting it halted the progression of breast cancer for more than five months. That study paired Avastin with the chemotherapy drug paclitaxel. Roche submitted two additional studies last year designed to win full approval for the drug, combining Avastin with four other chemotherapies. However, those studies showed a delay in cancer progression of one to three months. Because of those disappointing results, the FDA is now considering whether to revoke the drug's approval for breast cancer. More than 6,000 people have signed a petition urging the agency to keep the drug approved for breast cancer patients who respond well. The drug has already been approved for a half dozen forms of cancer, including colon, lung, kidney, and brain cancer. The FDA's current review only applies to the drug's use in recurring breast cancer. Source: Associated Press/Washington Post 9/17/10

Editorial: Medical Journals Must Police Studies with Industry Backing

Peer-reviewed journals keep doctors informed about cutting-edge research, but their credibility increasingly depends on ferreting out conflicts of interest that might call authors’ findings into doubt. Despite loud calls from medical ethicists to clarify financial ties between industry and the doctors who publish in journals, a study that appeared recently in the Archives of Internal Medicine found that, of 32 physicians who received consulting fees of $1 million or more in 2007 from orthopedic medical device companies, 25 published articles in orthopedic-related journals during 2008 without disclosing their financial ties to industry. These potential conflicts of interest were no more likely to be disclosed in journals with seemingly stringent policies than in other journals. ...[J]ournals must question would-be authors more intensely—and can use existing data sources to verify some of the information they receive from researchers. Journals should spot-check physician payments on the websites that pharmaceutical and device companies have put up—either voluntarily or after settlements with the Justice Department over allegations of kickbacks to doctors. The International Committee of Medical Journal Editors has proposed tighter reporting standards...[and] developed a thorough common disclosure form that asks researchers to disclose grants, consulting fees, writing assistance, and other items that they or their institutions have received. Journals would be wise to adopt it, at least as a minimum requirement. Source: Boston Globe 9/17/10

Scientists Warn Research Slowdown Poses Global Threat

A slowdown in research aimed at the development of new and more effective antibiotics poses the threat of a return to a situation that existed in the world before the discovery of penicillin, scientists have warned. "We have a big resistance problem that has become a global health crisis," said Dr. Ursula Theuretzbacher of the Austrian Center for Anti-Infective Agents at the 50th annual meeting of the Interscience Conference on Antimicrobial Agents and Chemotherapy, which opened in Boston on September 12. Theuretzbacher said thousands of people were being affected and dying from multidrug resistance, which had become a big problem in both developing and developed countries. She said a look at antibiotics that are being developed brings experts to the conclusion "that the medical need is not really met by the pharmaceutical industry." Dr. Gary Noel of the pharmaceutical company Johnson & Johnson said the volume of scientific research concentration on new antibiotics has probably dropped by half over the past 10 years. Source: Agence France Presse/Yahoo! News 9/13/10

U.K. Charity to Test Experimental Cancer Drug

AstraZeneca has agreed a deal with the commercial arm of a British charity to take an experimental cancer drug into early clinical trials. The experimental compound, called AZD-3965, is one of a new class of cancer metabolism drugs and targets the monocarboxylate transporter 1 (MCT-1), which is essential in cell metabolism. The idea is that by blocking MCT-1 the drug could limit cancer cells' ability to generate energy, reducing their ability to survive, Cancer Research UK said. The drug will become the sixth anticancer drug in the charity's clinical development partnerships project--a deal allowing drug companies to retain the rights to a potential treatment while the charity carries out early tests. Source: Reuters/Yahoo! News 9/12/10

Genzyme to Slash 1,000 Positions

Genzyme Corp., the Massachusetts biotechnology company fighting a takeover attempt by a French drug giant, said it will eliminate 1,000 jobs, or about 10 percent of the workforce, over the next 15 months to save money. Genzyme is the state’s largest biotechnology firm, with a stock market value of more than $18 billion. It has carved out a lucrative niche in the drug industry by selling expensive treatments for rare genetic disorders. Genzyme has about 12,800 workers globally, but plans to sell three divisions it does not consider essential. The 10 percent reduction figure does not include employees in those divisions. Source: Boston Globe 9/11/10

Alliance to Push for Medical Innovation

Worried that pressure to control healthcare costs will lead to lower prices for drugs and medical devices, making it less attractive to invest in life sciences companies, a group of venture capitalists and entrepreneurs has launched a national alliance to promote policies and regulations that favor U.S. medical innovation. The group, called the Medical Innovation and Competitiveness Coalition, or MedIC, will push for tax breaks and speedier approval of new products. It will be affiliated with the National Venture Capital Association, a trade organization representing investors in high-tech and life sciences start-ups. One focus of the coalition will be broad-based reform of the Food and Drug Administration, which life sciences executives and investors complain has inhibited innovation. Source: Boston Globe 9/8/10