A New Wonder Drug? Just Wait

Hyped but then discredited medications sow distrust in the clinical trials process

SUNDAY, Aug. 20, 2006 (HealthDay News) -- In the 1990s, millions of older women struggling with menopausal symptoms and worried about their bone health turned to hormone replacement therapy (HRT) after trials suggested it might help with both.

About the same time, a new generation of prescription analgesics called cox-2 inhibitors racked up sales in the billions, after trials showed they safely eased pain.

By 2005, the early promise of both of these blockbuster treatments was in tatters.

Evidence emerged that HRT boosted women's risk for cancer and stroke, and long-term use of cox-2s was found to raise cardiovascular dangers.

Based on these later findings, women largely abandoned long-term use of HRT, and the U.S. Food and Drug Administration pulled all but one of the cox-2s, Celebrex, from drugstore shelves.

Consumers have also had to put up with scientific flip-flops on everything from the value of vitamin E and dietary fiber, to the safety of aluminum cookware. So, it's no wonder many now feel mistrustful and confused when it comes to medical research.

A lot of that mistrust is justified, experts say.

"There's a lot of hyping of [study] results -- some of it comes from industry, where they often present results using relative terms, magnifying the benefit and minimizing the harm," said Dr. Lisa Schwartz, co-director of the VA Outcomes Group and an associate professor of medicine at Dartmouth Medical School. "So, there are all these other interests that are promoting drugs to be portrayed in a very favorable light."

One big problem: Many highly hyped trial results are presented at medical meetings. In that setting, researchers often offer up incomplete, "interim" data sets. Findings presented at meetings are also spared the scrutiny of peer review -- a prerequisite to publication in medical journals.

Nevertheless, eager researchers and an enthusiastic media can quickly get doctors and patients excited over results presented at meetings -- prompting them to try out a new "wonder drug" before all the information is in.

In fact, a recent study in the Journal of the National Cancer Institute found that use of the breast cancer drug Taxol soared five-fold after interim data on its efficacy was presented at a 1998 meeting.

Luckily for patients, Taxol lived up to its early promise in fighting tough-to-treat tumors. But that's not always the case, experts say.

"Our message is for the physicians in the community to be aware of the potential risks of adopting therapies too early," said the author of the JNCI study, Dr. Sharon Giordano, a professor of medicine in the department of breast medical oncology at the University of Texas M.D. Anderson Cancer Center, in Houston.

Another expert agreed.

"I don't think there's anything unique to any of these clinical trials -- clinical trials in general suffer from people not doing them appropriately and the pressure to cut corners," said Adil Shamoo, a professor of bioethics at the University of Maryland, Baltimore, and editor-in-chief of the journal Accountability in Research.

He said regulation isn't the only answer to this problem.

"We could regulate everybody to death, and society would stand still -- there'd be no progress," Shamoo said. "We obviously don't want to stop research, of course. But society has to find that fine balance -- how much should we regulate in order to reduce, to a reasonable level, these aberrations?"

Shamoo stressed that clinical trials -- which usually include study populations of only a few thousand -- will never be able to capture all the risks that can pop up when millions of people take a marketed drug.

On the other hand, he said, better trial oversight -- including random, independent "data audits" to keep researchers on the straight-and-narrow -- might not hurt.

Improved training of researchers would also help, said Shamoo, who teaches one of the few U.S. medical school courses devoted to the safety and ethics of clinical trials.

"There's currently no training and education for researchers [in medical schools], believe it or not," he noted. "I'd advocate that all researchers and graduate students undergo a minimum of 30 hours of training in research."

In the meantime, he said, the average consumer needs to listen to the latest report of a "major new treatment advance" with a healthy dose of skepticism.

"For me, unless I have no other choice, I'll never take a drug that has only one clinical trial behind it," Shamoo said. "I'll wait until two or three are done and show similar effects."

He pointed out that his own doctor prescribed him cox-2 pain relievers years ago to help ease exercise-related discomfort.

"I filled the prescription, just in case I got desperate, but I never used them," he said. "Why? Because I read the package insert, which told me how few clinical trials there were on the drug. So, when I needed pain relief, I took two or three ibuprofen, instead."

More information

For more on clinical trials, visit the U.S. National Institutes of Health.

SOURCES: Lisa Schwartz, M.D., co-director, VA Outcomes Group, Department of Veteran's Affairs, White River Junction, Vt., and associate professor, medicine, Dartmouth College, Hanover, N.H.; Sharon Giordano, M.D., M.P.., assistant professor, medicine, department of breast oncology, University of Texas M.D. Anderson Cancer Center, Houston; Adil Shamoo, Ph.D., professor, biochemistry and bioethics, University of Maryland, Baltimore, and editor-in-chief, Accountability in Research, March 15, 2006, Journal of the National Cancer Institute
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