JAMA: Comparative effectiveness must dig deeper for true impact

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Although comparative-effectiveness research is currently a hot topic on Capitol Hill, an expert at the Stanford University School of Medicine is suggesting that policymakers take a step back and make sure that the plans for comparative-effectiveness research "go deep enough to make a difference."

"The discussion that has taken place has been quite superficial and hasn't covered the range of changes that are needed for this type of research to be meaningful," said Randall Stafford, MD, PhD, associate professor of medicine at the Stanford Prevention Research Center in Stanford, Calif.

The Obama Administration sees comparative-effectiveness research as a strategy for reforming the U.S. healthcare system. The research would help identify the treatment options that are the most effective for a given condition. Despite this potential, Stafford and collaborator Caleb Alexander, MD, assistant professor of medicine at the University of Chicago, said that several challenges must be met if comparative effectiveness research is to be useful in significantly improving the quality and affordability of healthcare.

Stafford and Alexander's commentary, which appeared in the June 17 issue of the Journal of the American Medical Association, outlines five ways to put more meat on the bones of the discussions surrounding comparative effectiveness research:

Generate the data more rapidly. The pain reliever Vioxx is the best-known example of a drug originally aimed at a narrow patient population that became widely prescribed before evidence of harm was discovered. Ultimately, Vioxx was pulled from the market, but not before millions were exposed to these harms without substantial benefits. To prevent similar mishaps, Alexander said that obtaining comparative-effectiveness information earlier in the life of a new drug or device is a priority.

Link the evidence to strategies proven to modify how physicians practice medicine. The authors said that simply making the data available to physicians and patients isn't enough. "Unfortunately, we still want to believe that information alone will change physician practice. Years of research, however, suggest there are more potent influences on physicians, including their local culture of practice," Stafford said.

Broaden the agenda beyond drugs and devices. "It can't just be a comparison of this drug versus that drug," Stafford said. "This misses important aspects of practice and ends up exempting high-cost procedures from scrutiny." Researchers should focus on comparisons that include lifestyle modifications, such as diet and exercise, as well as alternative therapies that patients often implement on their own. In addition, research is needed on the most effective ways of delivering care. For instance, some studies show better chronic disease outcomes with nurse case managers compared with physicians working alone.

Alter the regulatory environment. Stafford noted that placebo-controlled trials are the standard for drug approval by the FDA, but said that the threshold must be raised for comparative effectiveness to work. The authors suggested that if a new medication isn't tested head-to-head against similar drugs, its labeling could be changed to indicate that it has not been found to be superior to the similar drugs. "This requirement would provide useful information to patients and physicians, as well as give manufacturers an incentive to perform more drug versus drug clinical trials," according to Stafford and Alexander.

Consider the cost implications. This is controversial because many fear that it may lead to restrictions on higher-cost treatments, regardless of the treatment's effectiveness. Some proponents of comparative effectiveness research have suggested not including cost as a factor. But "what good is comparative effectiveness if it cannot be used to discern anything about value to clinicians, insurers, patients and society?" the authors asked.

"The drive for comparative effectiveness has tremendous appeal. Who could argue against the idea of generating knowledge about what works and what doesn't?" said Alexander.

But the authors noted that broader changes are needed in the healthcare system--including the FDA's process for approving new medications and devices--to yield the right kind of data for such comparisons, and to ensure that patients, physicians and medical organizations make the wisest possible use of their healthcare dollars.

"Unless we start spending our resources