Comparing the effectiveness of drug treatments

January 14, 2008

Most Medicare providers and beneficiaries are unaware of the provision for comparative effectiveness research which is part of the Medicare Modernization Act. Politicians, policymakers, and manufacturers view it as a key initiative for the future of Medicare and beyond.

Insurance plans often lack the data needed to decide which healthcare option represents the best value.

Purchasers presumably would spend resources more efficiently if they had information about how and when the various therapeutic options work best. Comparative effectiveness research evaluates the outcomes of different therapies for the same condition.

Overall costs per Medicare beneficiary vary widely in different sections of the country. This variation is not explained by differences in the respective populations nor does it result in better health outcomes in the higher-spending regions. Moreover, the variation in expenditures is the greatest for conditions for which comparative data are lacking. This suggests that it may be possible to achieve the same, or better, results at lower costs.

Expense is a major reason few comparative clinical trials are done. The recent study comparing coronary stents with drug treatment cost approximately $33.5 million, while the ALLHAT study (comparing different antihypertensive and lipid-lowering drugs) cost more than $83 million. Although several federal agencies conduct some health services research, none focuses on supporting comparative studies. Clinical trials by the private sector focus on establishing efficacy for FDA approval rather than comparing treatment options.

The Agency for Healthcare Research & Quality has received roughly $15 million per year for comparative effectiveness projects pursuant to Section 1013 of MMA. AHRQ set up the Effective Health Care Program that includes Evidence-based Practice Centers to perform systematic reviews of existing research, the Centers for Education & Research on Therapeutics (CERTs) and the DeCIDE network (Developing Evidence to Inform Decisions about Effectiveness) to support new research, and the John M. Eisenberg Clinical Decisions and Communications Science Center to disseminate the knowledge.

Call for legislation

MedPAC (Medicare Payment Advisory Commission), the Blue Cross Blue Shield Association, and the Institute of Medicine have all issued calls for more legislation and resources to compare the effectiveness of new and existing procedures, drugs, devices, and biologics. Emerging from the ongoing debate are several areas of agreement, including:

1. Priorities for comparative effectiveness data should be set, and the research should be conducted by an institute that is governed by both public and private sectors.

2. Adequate and stable funding should be provided by both government and private payers.

3. An institute should be protected from political pressures so it can establish and maintain a reputation as a credible source for information.

4. Given the mission and requisite budget, the operations and work of the institute must be transparent.

5. A broad range of research is needed, including syntheses of existing research and clinical trials that compare options head-to-head.

6. Significant education programs and incentives must be included, and electronic health records should be required to incorporate the findings into clinical decision support tools.

An analysis prepared by the Congressional Budget Office suggests that it could take a decade or more before an investment in comparative effectiveness would amass enough data to offset the required investment in that research. Underutilized services that prove to be cost-effective could even result in increased Medicare spending. In its model, CBO projects that expenditures of $600 million in comparative effectiveness research over the next five years might generate $100 million in savings. Expenditures totaling $2.4 billion over 10 years could produce $1.3 billion in savings, after which continued investments could begin to generate savings in excess of research expenditures.

THE AUTHOR is associate professor, Department of Pharmacy Health Care Administration, University of Florida College of Pharmacy.