Comparative Effectiveness Research Pros, Cons

Published June 1, 2009

Until this year, most Americans hadn’t heard of comparative effectiveness research.

But then came the giant federal government stimulus package, part of which established a Federal Coordinating Council for Comparative Effectiveness Research and earmarked $1.1 billion for comparing medical treatments.

And then came President Barack Obama’s budget, which lauded such research and promised it would “enhance medical decision-making.”

This might be true, but if it’s mishandled, comparative effectiveness research (CER) could lead to severe restrictions on the kinds of treatments available to Americans who need medical care. It’s up to Obama to make sure his CER initiatives are implemented wisely and not used to justify rationing.

History of Abuse

Used properly, comparative effectiveness research can help patients and their doctors make informed health care decisions, by providing valuable information about what therapies work best.

But this type of research has a sordid history of being abused by government agencies to lower costs at the expense of patients’ health. Most notable is Great Britain’s National Institute for Health and Clinical Excellence (NICE).

The agency was established in 1999 to appraise various medical treatments and make treatment recommendations to the National Health Service, Britain’s government-run health care provider.

Ever since, NICE has frequently judged new medicines to be “not worth the cost.” The National Health Service has subsequently taken many of these medicines off its formulary, denying them even to patients who need them just to stay alive.

What’s behind unjust rulings such as these is a methodological flaw inherent in CER: The research tends to take into account only the “average” patient. If a medicine isn’t found to be both cost- and clinically effective for an average patient, NICE advocates against its use. But of course not everybody is average.

Low-Income Patients at Risk

This practice could easily wind up being implemented here in America, especially in government-sponsored health care programs such as Medicare, Medicaid, and state children’s health insurance programs. And if the kinks in the comparative effectiveness research methodology aren’t worked out, the United States could find itself complicit in the same wrongful denial of care that is now a trademark of the British system.

This is why lawmakers should make certain that the U.S. approach to comparative effectiveness sets aside cost considerations and focuses solely on clinical data.

Twenty-First Century Methods

Another way to make sure comparative effectiveness research is used properly is to follow the U.S. Food and Drug Administration’s lead in creating a Critical Path Initiative for CER. FDA’s current Critical Path Initiative aims at using the latest scientific advancements to modernize the process through which treatments are turned from laboratory discoveries into useable medical technologies.

This model would be just as useful in creating a modernized comparative effectiveness research program. By utilizing the most up-to-date scientific knowledge, treatment potency could be assessed in a manner that gives the utmost attention to the genetic, clinical, and demographic factors that affect how different patients react to different treatments.

Obama is right to see the potential benefits associated with comparative effectiveness research. As he moves forward with his plan, however, it’s imperative that he also be aware of the serious risks this research poses.

Peter J. Pitts is president of the Center for Medicine in the Public Interest and a former associate commissioner of the U.S. Food and Drug Administration. An earlier version of this article appeared in the New Jersey Star-Ledger. Reprinted with permission.