Comparative effectiveness efforts expanding but still raising concerns

Supporters hope a new institute launching in September will help guide the research and offset predictions of a rough road ahead.

By Doug Trapp — Posted July 12, 2010

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Efforts to compare the effectiveness of different medical interventions on the same conditions are growing, thanks to federal funding and the creation of a new public-private body to steer the federal efforts.

Supporters of comparative effectiveness research see it as a way to understand which treatments work best for the most people, possibly lessening waste in the health system. But critics warn that the complexity of patient populations will confound these studies. Also, such research could be used to limit public and private health plan coverage unjustly for treatments that are most effective for patient subpopulations, critics argue.

Flawed methodologies or poor public outreach could hurt public support for comparative effectiveness research, issue experts said at two recent forums held in Washington, D.C.

"If this research and the findings aren't trusted ... it will not have an impact on patient health and patient-centered outcomes," said Carolyn Clancy, MD, director of the Agency for Healthcare Research and Quality. She spoke at a June 24 comparative effectiveness symposium sponsored by pharmaceutical and health insurance industry associations.

Much of this trust building will fall to the 21-member Patient-Centered Outcomes Research Institute, which was created by the health reform law and replaces the Federal Coordinating Council for Comparative Effectiveness Research. The new research institute -- whose members will be appointed before the end of September -- will set the agenda for federal comparative effectiveness research efforts. But the health reform law prohibits the new institute from issuing practice guidelines and coverage recommendations.

Significant federal funding already is in place. The stimulus package allocated $1.1 billion for comparative effectiveness research to three federal agencies: the Dept. of Health and Human Services, AHRQ, and the National Institutes of Health. The research institute will receive hundreds of millions of dollars each year.

One size doesn't fit all

Comparative effectiveness research won't necessarily lead to better, more cost-effective treatments for everyone. It could shortchange certain patient subpopulations, said Tomas J. Philipson, PhD, a health economist and Daniel Levin Professor of Public Policy Studies at the University of Chicago. Philipson spoke at a June 23 event on comparative effectiveness research sponsored by the conservative American Enterprise Institute.

Suppose a randomized clinical trial concludes that the first of two treatments is more effective for a broad population, Philipson said. But what about the people who respond better to the second treatment? Health plan coverage decisions and subsidies could favor the first treatment based on the research, driving up the cost of more effective health care for the minority population.

"The question is not so much whether the blue or red is the better pill, the question is for whom is the red or blue pill better," Philipson said.

By example, he noted a 2006 National Institute of Mental Health study of the effectiveness of antipsychotic drugs on schizophrenics that found that older drugs were more cost effective at managing the disease than newer drugs. Medicaid would have saved $1.3 billion each year if it had stopped covering the newer drugs. But Medicaid also would have paid at least that much for increased hospitalizations for people who responded better to the newer drugs and had been denied coverage for them.

This scenario is uncommon, said Sean Tunis, MD, former chief medical officer at the Centers for Medicare & Medicaid Services. Dr. Tunis, who also spoke at the June 23 AEI event, is the founder and director of the Baltimore-based Center for Medical Technology Policy, which focuses on improving the quality of research.

He said Philipson picked the most striking evidence to support his position. Hospitalizing schizophrenics is more expensive and occurs faster than the fallout from most other failed interventions. Also, researchers understand that studies such as the 2006 NIMH effort can't simply be used to justify such an across-the-board coverage policy, he said.

Researchers need to address the huge gaps in understanding of the effectiveness of medical care, according to Dr. Tunis. For example, few studies have examined the various radiation treatments for prostate cancer, even though the procedures are performed routinely, he said.

Swinging for the fences

Despite all of the recent interest in the issue, federally supported comparative effectiveness research is in a "fragile state," said Gail Wilensky, PhD, an economist and senior fellow at Project HOPE, an international health foundation.

"There are going to be enormous implementation challenges," said Wilensky, speaking at the June 24 forum sponsored by drug and health plan associations. One key will be writing federal guidelines for how much evidence will be considered meaningful.

Several health professionals, patients and lawmakers are concerned about the concept of the research, Wilensky said. Similar worries about care rationing fueled an uproar over breast cancer screening recommendations issued in November 2009 by the U.S. Preventive Services Task Force that contradicted longstanding guidelines and practices.

Proponents hope the new research institute will allay those concerns. The institute's members will be appointed by the U.S. comptroller general, the head of the Government Accountability Office -- not the president and members of Congress, as was the case for the council.

Despite the controversy, comparative effectiveness research has the potential to improve patient outcomes greatly, said Michael Lauer, MD, director of the Division of Cardiovascular Sciences at the NIH's National Heart, Lung and Blood Institute. Dr. Lauer noted that health systems such as Kaiser Permanente have reduced the incidence of heart attacks in their facilities in the last decade, apparently due in part to an increase in the use of stents as opposed to anti-clotting drugs. This is the conclusion of a study by Dartmouth College researchers published in the June 10 New England Journal of Medicine.

Perhaps the greatest challenge of comparative effectiveness research, Wilensky said, will be translating its results into new health care practices.

Said Dr. Clancy: "We have a lot to learn about how to get this information to patients and clinicians when they need it."

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A new approach

The health reform law terminated the Federal Coordinating Council for Comparative Effectiveness Research, created by the federal stimulus package in 2009. The reform law replaced the council with the Patient-Centered Outcomes Research Institute, which will:

  • Set priorities for comparative effectiveness research and coordinate with agencies supporting such work.
  • Consider the potential cost of the research compared with the potential value of the results, among other factors.
  • Refrain from setting practice guidelines, payment recommendations or coverage decisions.
  • Have a 21-member board of governors, including the directors of the National Institutes of Health and the Agency for Healthcare Research and Quality. The comptroller general will appoint the remaining 19 members by mid-September.
  • Work with AHRQ to disseminate research findings.
  • Receive baseline funding of $10 million in fiscal year 2010, $50 million in fiscal 2011 and $150 million each year from fiscal 2012 through fiscal 2019. The institute will receive substantial additional funding from fees on health insurance plans and transfers from the Medicare trust fund.

Sources: Assn. of American Medical Colleges, Agency for Healthcare Research and Quality

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