Overview of Comparative Effectiveness

What is it?

Comparative effectiveness research examines the degree to which alternative treatments for the same health problem produce equivalent or different health outcomes. The research can be structured to evaluate treatments within a modality (e.g., different choices of medication therapy), treatments across modalities (e.g., medication versus surgery), treatment versus no treatment (sometimes referred to as "watchful waiting"), or alternative methods to deliver health care services. The research can consider a range of clinical outcomes (e.g., mortality rates, functional status, laboratory results, physical examination findings, or presence of side effects) and may also evaluate the costs associated with different options.

How would it work?

This policy option generally includes increased funding to conduct comparative effectiveness research. Three key issues are frequently raised in this policy option: (1) who is responsible for managing the research portfolio, (2) what type of research will be done, and (3) what will or can be done with the results of the research?

The debate over the entity conducting the research concerns whether it should be within government, independent, or a public–private collaboration. The type of research may be clinical trials, observational studies using existing databases, or reviews of prior research (particularly meta–analyses). The research may be restricted to the study of clinical outcomes, or it may also include financial outcomes. The products of comparative effectiveness research can be used in a variety of ways, including to provide information to physicians and patients in choosing appropriate treatments, as input into decisions about whether and/or for whom a treatment will be covered under public or private insurance policies, and for setting the level of reimbursement that will be allowed under private or public insurance policies.

Has it been tried before?

Federal, state, private, and international efforts to examine the comparative effectiveness of medical treatments have been undertaken previously or are ongoing. Several examples are highlighted below.

Federally, the Office of Technology Assessment, which operated between 1972 and 1995, provided Congress with analyses of technical and scientific issues, including the effectiveness of health technologies (Eisenberg and Zarin, 2002). Current funding for comparative effectiveness comes from the National Institutes of Health, as well as the Agency for Healthcare Research and Quality (AHRQ), which funds comparative effectiveness research through its Evidence–based Practice Centers Program (AHRQ, 2002). Furthermore, the Food and Drug Administration assesses safety and efficacy of drugs and devices. In addition, established in 1999, the Medicare Coverage Advisory Committee serves an advisory role for Medicare in analyzing the comparative effectiveness of some new services and technologies (Garber, 2001).

At the state level, the Washington State Health Technology Assessment program performs systematic evidence–based assessments of the cost, efficacy, and safety of medical devices and procedures, equipment, and diagnostic tests. The assessments are reviewed by a clinical panel, which makes coverage recommendations for state insurance programs.

The Drug Effectiveness Review Project and the Medicaid Evidence–based Decisions Project are collaborations between states to evaluate the comparative effectiveness of drugs and medical treatments. Within the private sector, the Blue Cross Blue Shield Association established the Technology Evaluation Center, which provides comprehensive reviews of a variety of therapies, drugs, and devices (Wilensky, 2006).

Internationally, the National Institute for Health and Clinical Excellence (NICE), which is funded by the U.K. government and is part of the nation's National Health Service, conducts systematic reviews of existing literature on the cost–effectiveness of medical interventions and prescription drugs. NICE uses the reviews as the basis for coverage decisions for the National Health Service.