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In their December 2007 Document, styled Research on the Comparative Effectiveness of
Medical Treatments: Issues and Options for an Expanded Federal Role, the Congressional Budget Office stated:
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As applied in the health care sector, an analysis of comparative effectiveness is simply a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients. Such a study may compare similar treatments, such as competing drugs, or it may analyze very different approaches, such as surgery and drug therapy. The analysis may focus only on the relative medical benefits and risks of each option, or it may also weigh both the costs and the benefits of those options. In some cases, a given treatment may prove to be more effective clinically or more cost-effective for a broad range of patients, but frequently a key issue is determining which specific types of patients would benefit most from it. Related terms include cost–benefit analysis, technology assessment, and evidence-based medicine, although the latter concepts do not ordinarily take costs into account.
In October 2007 Congressional Research Service provided a Report to Congress called “Comparative Clinical Effectiveness and Cost-Effectiveness Research: Background, History, and Review.” This report stated:
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Comparative effectiveness research is a term that has been defined by people in many different ways. All agree that comparative effectiveness research compares the effectiveness of two or more health care services or treatments, and is one form of health technology assessment. It compares outcomes resulting from different treatments or services, and provides information about the relative effectiveness of treatments.
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Additional specifics about the research and its definition are sources of contention. In particular: (A) Effectiveness — How should effectiveness be measured? Should the research compare only the effectiveness (the effect in routine clinical practice) or also the efficacy (the effect under optimal conditions) of treatments or services? (B) Costs — Should costs be included in the research? Should the costs be reported separately from the effectiveness results? Or should a cost-effectiveness ratio be the ultimate goal?
In June 2007 the Medicare Payment Advisory Commission (Medpac) issued a Report to Congress on Promoting Greater Efficiency in Medicare. Medpac stated:
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Comparative-effectiveness analysis compares the relative value of drugs, devices, diagnostic and surgical procedures, diagnostic tests, and medical services. By value, we mean the clinical effectiveness of a service compared with its alternatives. Comparative-effectiveness information has the potential to promote care of higher value and quality in the public and private sectors. Comparative information would help patients and providers become better informed and make value-based decisions. Most public payers—including Medicare—and private payers do not encourage patients or providers to consider the value of a service when making health care decisions. Information about the value of alternative health strategies might improve quality and reduce variation in practice styles. Use of comparative effectiveness research might improve health but will not necessarily reduce spending. Many effective treatments are underused, and effectiveness research might encourage their greater and more appropriate use (McGlynn et al. 2003). On the other hand, comparative-effectiveness research might reduce spending if, among a set of clinically comparable services, less costly services replace more costly services.
In 2007 Institute of Medicine issued work styled “Learning What Works Best: The Nation’s Need for Evidence on Comparative Effectiveness in Health Care.” That work states:
Within the overall umbrella of clinical effectiveness research, the most practical need is for studies of comparative effectiveness, the comparison of one diagnostic or treatment option to one or more others. In this respect, primary comparative effectiveness research involves the direct generation of clinical information on the relative merits or outcomes of one intervention in comparison to one or more others. Secondary comparative effectiveness research involves the synthesis of primary studies (usually multiple) to allow conclusions to be drawn. Secondary comparisons of the relative merits of different diagnostic or treatment interventions can be done through collective analysis of the results of multiple head-to-head studies, or indirectly, in which the treatment options have not been directly compared to each other in a clinical evaluation but reside in larger databases. Conclusions utilize inferential adjustments based on the relative effect of each intervention to a specific comparison, often a placebo.
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