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Cost-effectiveness analysis in US healthcare decision-making: where is it going?

A recent survey of pharmacoeconomic assessment in eleven Organisation for Economic Co-operation and Development (OECD) countries reported that just under half of these countries now use the techniques of cost-effectiveness analysis (CEA) to determine the “value-for-money” of new pharmaceuticals. For many of these countries, and for others such as Australia and the Canadian Province of Ontario, systems for integrating cost-effectiveness information into reimbursement decisions have been in place for over a decade. Analysts in these countries are occupied with refining their use of costeffectiveness information: evaluating its quality, reflecting on experience to date, and revising their systems for incorporating economic information into reimbursement decisions. In the United States, in contrast, although cost-effectiveness is frequently invoked as a desirable goal—and even as a basic requirement for improving our health care—costeffectiveness information has not been widely used as an input to healthcare decisionmaking. Processes such as those in the OECD and other countries are not in place. This is in part because of the differences in the underlying healthcare systems. Whereas the European, Australian, and Canadian systems are national (or provincial) with single payers and accountable planners, the US system includes multiple systems and multiple financing sources. There are also other significant differences, as many authors have noted: differences in the histories of healthcare systems and differences in cultures and expectations of the population, to name two of the most basic. Although the United States may never approach the matter-of-fact manner with which many other countries view the necessity of factoring resource constraints into their choices and implement this imperative, there has nonetheless been a noticeable evolution in the use of CEA over the past several years. Important changes in the healthcare environment, including rapid increases in spending on pharmaceuticals, some significant regulatory changes, and growing familiarity with CEA, have led to the development of mechanisms and institutions that are better able to manage CEA. The goal of this volume is to collect and describe recent developments in CEA for healthcare decision-making in the United States. Included are an analysis by authors from the United Kingdom and an update from our northern neighbor to offer context and contrast. The discussions in this volume relate almost entirely to decision-making concerning pharmaceuticals, a reflection of the unique characteristics of this sector and the high level of activity therein.