Embargoed Until:
June 25, 2009
12:01 a.m. Eastern Time



Christopher Fleming

A New Center For Comparative Effectiveness Research Should Leave Cost-Effectiveness Analysis To Others, Wilensky Says

In Second Article, Researchers Describe The Types Of “Marginal Medicine” That Might Be Revealed By Comparative Effectiveness Research

Bethesda, MD -- If Congress establishes a new center for research on the comparative effectiveness of drugs and medical devices, the center should not include calculations of cost and cost-effectiveness in its analyses, a former administrator of the Health Care Financing Administration (HCFA) argues in an article published today on the Health Affairs Web site. http://content.healthaffairs.org/cgi/content/abstract/hlthaff.28.4.w719

The economic stimulus bill passed earlier this year included $1.1. billion over two years for comparative effectiveness research, writes Gail Wilensky, a senior fellow at Project HOPE who ran HCFA, the predecessor agency to the Centers for Medicare and Medicaid Services (CMS), during the administration of President George H.W. Bush. Her article surveys the comparative effectiveness provisions of the stimulus bill and the longer-term decisions that still need to be reached on the location, governance, and funding of a new center for comparative effectiveness research; the role of costs in the center’s activities; and other issues.

Wilensky’s article is one of two papers published online today by Health Affairs. In the second paper, researchers set forth a taxonomy of the categories of “marginal medicine” that might be revealed through comparative effectiveness research. “By delineating how evidence can be used to define each of these categories, we seek to provide a conceptual basis to guide clinicians, researchers, and policymakers as they consider how to prioritize the targets and methods for future comparative effectiveness research,” write Ari Hoffman, a postdoctoral fellow in bioethics at the National Institutes of Health, and Steven Pearson, president of the Institute for Clinical and Economic Review (ICER) at the Massachusetts General Hospital Institute for Technology Assessment. http://content.healthaffairs.org/cgi/content/abstract/hlthaff.28.4.w710

Cost-Effectiveness Analysis Should Be Undertaken By Public And Private Payers, Not A New Comparative Effectiveness Research Center

The clinical effectiveness information generated by a comparative effectiveness research center “must be regarded as objective and credible and must be protected from the political process if it is to be used to drive changes in how medical conditions are treated,” Wilensky says. “I believe that having cost-effectiveness information included as part of the comparative effectiveness analyses or as part of the work of an institute or center for comparative effectiveness research will taint the clinical effectiveness analyses that are produced or at least will make some of the results suspect.”

Wilensky emphasizes that not including cost information in the activities of a new center would not mean ignoring cost and cost-effectiveness analyses in making reimbursement and even coverage decisions. She proposes that the CMS be funded to do cost-effectiveness analyses for procedures that are important to the Medicare population. “The CMS would also have to be given the statutory authority to make use of cost information in setting reimbursement rates or coverage -- authority it does not now have,” she notes. Wilensky suggests that “private payers may choose to contract the cost-effectiveness work to not-for-profit groups already doing technology assessments such as the ECRI Institute or the Blue Cross Blue Shield Association Technology Evaluation Center, to add credibility to the analyses and provide some distance between the assessment and the payers.”

A New Taxonomy Of Marginal Medicine

In their article, Hoffman and Pearson use the term “marginal medicine” because “the term waste is often not a helpful guide; it triggers strong emotions among clinicians by implying that there are plentiful, clear-cut examples of interventions just waiting to be identified that harm patients or produce no benefit.” Unfortunately, things “are not that simple,” the authors warn. “Given the challenge of reducing wasteful spending in health care, policymakers must transform the vague concept of ‘waste’ into a clearly defined set of targets that can be more precisely addressed with research and policy.”

Hoffman and Pearson identify four categories of marginal medicine and use these categories to suggest which tools of comparative effectiveness research might be most appropriate for studying particular interventions. Their first two categories are driven by uncertainty because of inadequate evidence of clinical benefit: (1) interventions with inadequate evidence of comparative benefit for any indication; and (2) interventions that are used beyond the boundaries of established benefit. Hoffman and Pearson’s last two categories of marginal medicine are driven by considerations of cost-effectiveness: (3) interventions with established benefits that are comparable to other options but which cost more; and (4) interventions with established benefits that are greater than other options, but for which the incremental benefits come at a relatively high cost.

The authors say their taxonomy of marginal medicine can help direct researchers, clinicians, and policymakers to the most appropriate comparative effectiveness research tool. For example, if an intervention is relatively new, researchers might determine whether the intervention belongs in the first category of marginal medicine by employing a “systematic review,” which synthesizes existing published evidence, to determine the level of certainty or uncertainty about the intervention’s benefits. If an intervention is placed in category one, evidence on the intervention’s benefits or lack of benefits might be developed through “pragmatic randomized clinical trials” (RCTs), which “seek to expand traditional RCT methods to better reflect real-world conditions.”

After the embargo lifts, you can read the article by Wilensky at http://content.healthaffairs.org/cgi/content/abstract/hlthaff.28.4.w719

You can read the article by Hoffman and Pearson at


Health Affairs, published by Project HOPE, is the leading journal of health policy. The peer-reviewed journal appears bimonthly in print with additional online-only papers published weekly as Health Affairs Web Exclusives at www.healthaffairs.org.


©2009 Project HOPE–The People-to-People Health Foundation, Inc.