When it comes to “comparative effectiveness” research intended to improve the economics and efficiency of healthcare, policymakers must examine not just drugs and devices, but also health-delivery methods and lifestyle practices, Stanford University researchers say. The report in the Journal of the American Medical Association suggests five strategies that policy wonks should pursue when looking at comparative effectiveness research, for which the recent economic stimulus package allocates $1.1 billion.  

 

“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,” says Randall Stafford, associate professor of medicine at the Stanford Prevention Research Center. As the researchers point out, the Obama administration sees comparative effectiveness research as a key strategy for reforming the nation's healthcare system. The research is designed to help identify the treatment options that are the most effective for a given condition. But some in the scientific community argue the research—if not done correctly—could stifle innovation, as certain expensive treatments would be shunned if they were deemed too costly.

 

Policymakers should expand their focus beyond drugs and devices to avoid missing important aspects of practice and high-cost procedures. The researchers say that reformers 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. They add that research is needed on the most effective ways of delivering care.

 

They researchers also need to generate data more rapidly in order that comparative-effectiveness information is made available earlier in the life of a new drug or device. They cite the example of the pain reliever Vioxx, which was pulled from the market, only after millions were exposed to these harms without substantial benefits, the researchers say.

 

Any comparative-effectiveness evidence should be linked to strategies proven to modify how physicians practice medicine, the researchers say. “Unfortunately, we still want to believe that information alone will change physician practice,” Stafford says. “Years of research, however, suggest there are more potent influences on physicians, including their local culture of practice.”

 

The researchers also suggest that the regulatory environment should be altered so that clinicians aren’t comparing a new drug against a placebo if their goal is to compare different clinical strategies. Placebo-controlled trials are the standard for drug approval by the U.S. Food and Drug Administration, the researchers note. If a new medication isn't tested head-to-head against similar drugs, the researchers say its labeling could be changed to say, for instance, “This drug has not been found to be superior to the other calcium-channel blockers in the treatment of hypertension.” Such a requirement would have two results, they say. It would provide useful information to patients and physicians. It would also give manufacturers an incentive to perform more clinical trials that compare one drug to another.

 

Finally, comparative-effectiveness studies must also look at cost implications, even though the practice is controversial, the researchers say. “What good is comparative effectiveness if it cannot be used to discern anything about value to clinicians, insurers, patients, and society?” they ask.

 

Researchers say becoming most sophisticated about comparative effectiveness is critical if it is going to work. Previous reform attempts, such as the drive to develop clinical guidelines in the 1990s for treating specific illnesses, failed because “our approach to implementing them was simplistic and not sophisticated enough,” they say. “Unless we get it right with comparative effectiveness, it's at risk of a similar fate."