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NEJM Articles Address Comparative-Effectiveness Research

NEW YORK (GenomeWeb News) – Three perspectives articles appearing online in the New England Journal of Medicine today are examining some key issues related to the goals and execution of comparative-effectiveness research and its implementation in the US.

Comparative-effectiveness research, or CER, received more than a billion dollars of funding through the American Reinvestment and Recovery Act earlier this year. The US Department of Health and Human Services subsequently organized a new body, the Federal Coordinating Council for Comparative Effectiveness Research, to oversee CER supported by stimulus funds.

But the enhanced emphasis on CER is not without controversy. Some politicians, advocacy groups, and industry leaders argue that CER could lead to one-size fits all medicine or unwelcome government interference in health care.

"Industry and free-market advocates have expressed concerns about the role of cost-effectiveness analyses within CER and subsequent governmental intrusion into doctor-patient decisions," Aanand Naik and Laura Petersen, researchers at the Michael E. DeBakey Veterans Affairs Medical Center in Houston and the Baylor College of Medicine, explained in one of the perspectives articles published today.

Although HHS stated that the Federal Coordinating Council for Comparative Effectiveness Research "will not recommend clinical guidelines for payment, coverage, or treatment," some critics have expressed concerns that it could morph into a body such as the UK's National Institute for Clinical Excellence, which determines which treatments will be reimbursed based on cost-benefit calculations and quality-adjusted life years.

Others are calling for pharmacogenomics to play a central role in CER from the outset in order to prevent a situation in which CER hinders the translation of genomics research to the clinic and adoption of personalized medicine.

In their perspectives article, Alan Garber, director of Stanford University's Center for Primary Care and Outcomes Research and the Center for Health Policy, and Sean Tunis, founder and director of the Center for Medical Technology Policy in Baltimore, tried to appease CER concerns related to personalized medicine.

Garber and Tunis argued that CER will aid rather than thwart progress in personalized medicine. "[F]ar from impeding personalized medicine, CER offers a way to hasten the discovery of the best approaches to personalization, providing more and better information with which to craft a management strategy for each individual patient," they wrote.

Despite notable exceptions such as HER2 and KRAS testing, the researchers noted that personalized medicine has done little to change clinical practice so far. They suggested that a lack of appropriate studies for evaluating the clinical utility of genomics-based personalized approaches is the "greatest obstacle" to increased adoption of such methods in health care.

The pair noted that CER should help to distinguish between genomic tests that improve treatments or outcomes in the clinic and those that don't, provided studies are designed appropriately.

In answer to those who argue that the misuse of CER may have detrimental consequences for policy and payment choices, Garber and Tunis countered that such decisions "will not be improved by discouraging the use of the most relevant and valid information about what works and in whom." Instead, they suggested, new innovations and clinical trial design will likely be necessary before personalized medicine realizes its full potential.

Meanwhile, Harvard Medical School and Brigham and Women's Hospital researcher Jerry Avorn argued in his perspectives article that CER has been misconstrued and characterized by an "avalanche of non-facts."

Calling the opposition to CER funding "a vigorous and well-coordinated backlash," Avorn suggested that many of the criticisms leveled at CER so far — including fears of health care rationing — have been based on "overt mischaracterization of the legislation's content."

While he conceded that there are legitimate concerns related to CER, health care reimbursement, and strategies for treating patients who don't respond to conventional or cost-effective treatments, Avorn's article emphasized the notion that disagreement over the details of CER should not preclude its use.

"[W]e must stay focused on the methodological, practical, and ethical issues we will face in the new era of CER, while avoiding the disinformation and distractions that seek to equate generating knowledge with denial of care," Avorn concluded.

In their NEJM article, Naik and Petersen addressed some of these methodological issues, noting that they hope to see a shift from research recommendations to implementation in the CER field. The duo pointed out that although basic research is relatively well supported, translating that research to new clinical treatments is often time-consuming and poorly funded.

Much of the CER done at the moment consists of making conclusions and recommendations based on existing research, Naik told GenomeWeb Daily News. But the uptake of such recommendations by physicians and/or patients is often slow or non-existent.

"We really need to move forward from just making recommendations and writing guidelines," Naik said. Such implementation will likely involve establishing priority levels with input from patients and clinicians and investigators, he explained, which may involve some health care differences between hospitals and health regions.

In addition, Naik and Petersen noted, randomized clinical trials aren't usually designed to tease apart the potential benefits of different treatment options. Even so, Naik said he doesn't see a conflict between CER and randomized clinical trials, since it may not be ethical, cost-effective, or feasible to do traditional randomized clinical trials in some patient sub-populations.

In such cases, he said, it may be better to compare new treatments with existing treatments. Consequently, Naik suggested that CER actually has the potential to determine effective treatment groups that would otherwise be neglected.

"Above all, the Federal Coordinating Council must remain mindful that the primary goal of CER is to enhance the translation of new medical discoveries into safe and high-quality health care for all Americans," Naik and Petersen wrote. "This goal can be achieved only if our renewed investment in CER includes a commitment to implementation research."

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