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A Great Piece of Advice to Follow: In a world where doctors are over-worked, hospital are full and drug companies lobby doctors to prescribe their products, Ray Moynihan's philosophy is simple - be an advocate for yourself.

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BECOME A HEALTHY SKEPTIC

When we go to the doctor, we assume that the drugs he or she prescribes have been carefully tested to make sure they are both safe and effective. Most times they are. Yet sometimes the drugs cause more problems than they solve. Adverse drug reactions kill tens of thousands of people annually; one widely cited study published in the Journal of the American Medical Association (JAMA) in 1998 puts the number at more than 100,000. Recently a series of drug recalls have pulled back the curtain to show how the media, the public, and some doctors can misinterpret medical studies or take them out of context in ways that make medical treatments look safer and more effective than they actually are.

To a greater degree than ever before, powerful forces in the marketplace are impacting the quality, use, and safety of prescription medications. Drug manufacturers are spending more to promote their products while being subjected to tighter regulation and greater pressure for financial returns. The media are talking up each new “miracle cure” in headlines and television segments. Doctors have to navigate a tangle of administrative and medical concerns, one physician noting that “if you have a patient in your office, you can’t say, ‘Oh, I’m going to look at the drug company’s online database about Zyprexa.’ Most doctors don’t even know the databases exist. But even if they did, the next thing you know, three or four hours have gone by and you’ve missed all the patients waiting to see you.” Insurance companies and even the stock market play a role too. And consumers, increasingly subject to pharmaceutical advertising, are routinely urged to demand the best and the newest for their health. All together, this is a perfect storm for prescription drug problems.

How often do today’s medical “breakthroughs” become tomorrow’s discredited science? John P. A. Ioannidis, an epidemiologist at Tufts University School of Medicine in Boston and the University of Ioannina School of Medicine in Greece, studied the question. He examined the most-cited clinical studies published in the top three medical journals between 1990 and 2000 to see how well researchers’ initial claims held up against subsequent research. His findings, published in JAMA, show that the key claims of nearly one-third (14 out of 49) of the original research studies he examined were either false or exaggerated. Small study size, design flaws, publication bias (failure to publish negative results or duplication of positive results), drug-industry influence, and the play of chance were among the problems Ioannidis found that caused false or exaggerated claims.

Studies can be designed and interpreted in ways that make even ineffective drugs seem like lifesavers, says Curt Furberg, a well-known cardiovascular epidemiologist and former chief of the clinical trials branch at the National Heart, Lung, and Blood Institute in Bethesda, Maryland. Furberg, a tall, square-faced man with a Swedish accent, wants more objectivity in medical research. “We need more publicly funded studies,” he says, adding that manufacturer-sponsored research tends to minimize risks and exaggerate benefits.

A score of studies support his opinion. Among them is a 2003 analysis by Cary P. Gross, an associate professor of medicine at Yale School of Medicine, that was published in JAMA. In his survey, one study found that industry-sponsored research was positive 87 percent of the time compared with 65 percent positive for research that was not industry sponsored. According to Gross, the evidence was overwhelming that “industry sponsorship was likely to yield pro-industry results.” A 2006 analysis published in the American Journal of Psychiatry found that 90 percent of manufacturer-sponsored studies of antipsychotic drugs led to claims that the study drug was as good as, or superior to, every other drug in its class. Shannon Brownlee, an award-winning medical writer based in Washington, D.C., ascribed this to the “Lake Wobegon effect,” which renders every drug “above average.”

Furberg’s efforts to debunk overly enthusiastic interpretations of medical studies have led to occasional clashes with his colleagues. In 2004 the U.S. Food and Drug Administration (FDA) was preparing to hold hearings on the safety of painkillers known as COX-2 inhibitors, including Vioxx, which David Graham, an official in the FDA’s Office of Drug Safety, said may have caused an estimated 39,000 to 60,000 heart-attack deaths in just five years. At the time, Furberg was a member of the FDA Advisory Committee on Drug Safety and Risk Management. But after he told The New York Times that the COX-2 inhibitor Bextra also caused heart attacks, the agency made a surprising move: It removed Furberg from the advisory panel. Sandra Kweder, acting director of the Office of New Drugs, Center for Drug Evaluation and Research at the FDA, told a reporter that Furberg’s comments showed he could not be objective. Furberg now asks, “If bias was a concern, why did they allow 10 advisory members with ties to the manufacturers to be seated?” He was reinstated to the panel two days later and vindicated when the FDA announced that it had asked Pfizer to voluntarily withdraw Bextra from the market.

Part of the difficulty in detecting drug side effects, Furberg says, has to do with study size. Drugs go through a regimen of tests prior to receiving approval from the FDA. During the first two stages, called Phase I and II trials, an experimental drug is tested on just a few hundred volunteers to look for side effects. If no serious problems are detected, the drug is tested for efficacy in a Phase III trial. But efficacy trials often involve only several hundred to a few thousand patients. And while a study of 200 to 300 arthritis patients is large enough to show whether a new drug relieves pain, just one such study isn’t large enough to pick up less-common—but potentially deadly—side effects. Furberg says, “If only one in a thousand patients will die from a heart attack, an efficacy study of 200 or even 2,000 patients is simply too small to get a reliable answer about rare side effects.” Seemingly rare side effects can take tens of thousands of lives when millions of prescriptions are written.

Such limitations in a study’s design can escape detection even by top peer reviewers and medical editors. Marcia Angell, the former editor-in-chief of The New England Journal of Medicine (NEJM), says that most doctors are ill equipped to critically assess the conclusions of researchers. A trim woman with a warm smile, Angell leans forward in her seat at her home in Cambridge, Massachusetts, and says, “Let me tell you the dirty secret of medical journals: It is very hard to find enough articles to publish. With a rejection rate of 90 percent for original research, we were hard pressed to find 10 percent that were worth publishing. So you end up publishing weak studies because there is so much bad work out there.” Doctors, Angell says, are not skeptical enough about what they read in top journals. “They should say, ‘I don’t believe this; prove it to me.’”

The truth in any drug study can be camouflaged by how it is reported. “One way that’s been done—for many treatments is to use combination end points.” Here’s how it works: A single drug can be tested for a variety of outcomes; for example, a cholesterol-lowering drug can be tested for its effect on cholesterol level, blood pressure, and/or rates of heart failure, heart attack, or death. By combining two or more of these outcomes to create a single category, you can say it helped “A and B” even if it only helped A and not B.   David L. Brown, chief of the division of cardiovascular medicine at the State University of New York, Stony Brook School of Medicine, calls the use of combination end points a “brilliant marketing tool.” Brown says that while combination end points have a legitimate purpose when researchers are testing drugs for a rare or infrequent outcome, far too often researchers use the information in ways that “mislead both doctors and the general public.”

Another way to make drugs look better and safer than they are is to report or cite only successful studies while ignoring those with bad outcomes. The problem of cherry-picking studies is a very real one, especially for antidepressants, says Erick Turner, a former FDA reviewer, now an assistant professor of psychiatry at the Oregon Health & Science University. Turner recently published research in NEJM showing that “when studies of antidepressants were negative, they were reported as negative only 8 percent of the time—but when studies were positive, they were reported as positive 97 percent of the time.”

John Abramson, a clinical instructor at Harvard Medical School and author of Overdo$ed America: The Broken Promise of American Medicine, says he grew concerned when he learned that the authors of professional guidelines recommending an expanded use of statins had ties to the drugs’ manufacturers.

Jerome Hoffman of UCLA, saying it is a shame that in the face of so many medical and pharmacological advances there is such an exposure to risk. “It’s ironic that one of the unintended consequences of the publication of so many untenable claims, based on poorly done research and spin, is that it can obscure true advances when they do occur,” he says. Citing the number of great successes medical research has brought us—lifesaving drugs from penicillin to insulin, along with invaluable treatments and medical devices—he adds, “It’s one more reason why we have to be appropriately skeptical, unafraid to speak out about misleading claims, and insistent upon holding clinical research to the standards of science.”

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