News Release

Comparative Effectiveness Research and Medical Innovation

Policy Analysis Explores How CER Could Influence Medical Advances

WASHINGTON, D.C. –– Determining what treatments work best for which patients in real–world settings—known as comparative effectiveness research (CER)—can help foster beneficial medical innovation, according to a new Policy Analysis from the nonprofit, nonpartisan National Institute for Health Care Reform (NIHCR).

Written by researchers at the Center for Studying Health System Change (HSC) and Mathematica Policy Research, the analysis identifies key policy considerations to help ensure that CER encourages beneficial innovation while strengthening the evidence base for medical decisions.

“A major goal of CER is to encourage the use of effective therapies and discourage ineffective therapies. By promoting effective therapies, CER stands to increase the rewards and incentives for beneficial innovations in medical care. However, CER could dampen development of new, potentially effective therapies by creating additional hurdles for innovators,” according to the analysis.

“Policy makers can take steps to ensure that CER encourages beneficial innovation by creating a stable, transparent and predictable environment to compare therapies and setting explicit time frames for evaluation that are in sync with the innovation process. Policy makers and payers also could ease patient access to promising therapies when relative effectiveness is unknown by extending coverage with evidence development and sharing financial risk with innovators,” the analysis states.

The analysis suggests that policy makers consider the following priority areas when crafting an overall strategy that optimizes the influence of CER on innovation:

  • the role of policy in promoting societal priorities;
  • the importance of stability to innovation;
  • setting consistent evidence standards;
  • encouraging providers and patients to participate in CER studies;
  • supporting personalized medicine;
  • acknowledging uncertainty;
  • encouraging patients and clinicians to understand and use CER; and
  • leveraging public investments in CER.

Written by Emily Carrier M.D., M.S.C.I., an HSC senior researcher; Hoangmai H. Pham, M.D., M.P.H., a former HSC senior researcher, and Eugene Rich, M.D., director of Mathematica Policy Research’s Center on Health Care Effectiveness, the new Policy Analysis—Comparative Effectiveness Research and Innovation: Policy Options to Foster Medical Advances.

Building on a $1.1 billion investment in comparative effectiveness research in the 2009 economic stimulus legislation, the recently enacted health care reform law, known as the Patient Protection and Affordable Care Act (PPACA), creates a formal structure to guide publicly financed CER going forward.

The law creates a private, nonprofit corporation—the Patient–Centered Outcomes Research Institute (PCORI)—whose purpose is to help patients, clinicians, purchasers and policy makers make “informed health decisions by advancing the quality and relevance of evidence” of various approaches to preventing, diagnosing, treating, monitoring and managing diseases and other health conditions. The institute will identify comparative effectiveness research priorities, establish a research agenda, adopt methodological standards and administer a federal trust fund dedicated to CER.

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The National Institute for Health Care Reform (NIHCR)is a nonpartisan, nonprofit 501 (c)(3)organization created by the International Union, UAW; Chrysler Group LLC; Ford Motor Company; and General Motors. Between 2009 and 2013, NIHCR contracted with the Center for Studying Health System Change (HSC) to conduct high-quality, objective research and policy analyses of the organization, financing and delivery of health care in the United States. HSC ceased operations on Dec. 31, 2013, after merging with Mathematica Policy Research, which assumed the HSC contract to complete NIHCR projects.