Research on the Comparative Effectiveness of Medical Treatments: Isues and Options for an Expanded Federal Role

Preface Ri sing costs for health care represent a central challenge both for the federal government and the private sector, but opportunities may exist to constrain costs in both sectors without adverse health consequences. Perhaps the most compelling evidence of those opportunities involves the significant geographic differences in spending on health care within the United States, which do not, on average, translate into higher life expectancy or substantial improvements in other health statistics in the higher-spending regions. At the same time, only a limited amount of evidence is available about which treatments work best for which patients and whether the added benefits of more-effective but more-expensive services are sufficient to warrant their added costs. Together, those findings suggest that generating better information about the costs and benefits of different treatment options—through research on the comparative effectiveness of those options—could help reduce health care spending without adversely affecting health overall. This Congressional Budget Office (CBO) paper—prepared at the request of the Chairmen of the Senate Budget and Finance Committees—examines options for expanding federal support for research on comparative effectiveness. It reviews the current state of such research in both the public and private sectors and discusses several mechanisms for organizing and funding additional research efforts. It also discusses the different types of research that could be pursued and their likely benefits and costs. Finally, it considers the potential effects that such research could have on health care spending and the difficult steps that public and private insurers would probably have to take to achieve substantial savings on the basis of that research—in particular, changing the financial incentives for doctors and patients to reflect that information. In accordance with CBO's mandate to provide objective, impartial analysis, this paper contains no recommendations. Philip Ellis of CBO's Health and Human Resources Division prepared the paper, with valuable contributions from Colin Baker and Morgan Hanger. The analysis benefited from comments by Dr. (The assistance of external reviewers implies no responsibility for the final product, which rests solely with CBO.) John Skeen edited the paper, and Maureen Costantino prepared it for publication and designed the cover. Lenny Skutnik printed the initial copies, Linda Schimmel handled the print distribution, and Simone Thomas prepared the electronic version for CBO's Web site (www.cbo.gov).