Cost-Effectiveness Analysis 2.0.

n engl j med 376;3 nejm.org January 19, 2017 C analysis in U.S. health care seems poised for a second act of sorts. Although it has never actually gone away, efforts to apply it have encountered resistance, and the federal government and some health care organizations have sometimes prohibited its use or relegated it to a minor role. But several developments are helping to recharge the field. One is the embrace of its methods by prominent groups that are using it to measure and communicate the value of new drugs and other interventions. Another is the publication of new guidelines for such analysis from a national panel that is updating recommendations for the field from 1996.1-3 Cost-effectiveness analyses reveal the trade-offs involved in choosing among alternative interventions, with the goal of obtaining the most health possible for the available resources. Such analyses help define and illuminate the potential health benefits lost when the best alternative is not selected. Researchers have conducted cost-effectiveness analyses on a wide range of topics. Some organizations, such as the Advisory Committee for Immunization Practices, which establishes national immunization policy recommendations on behalf of the Centers for Disease Control and Prevention, have used these analyses in their deliberations. The Medicare program, however, does not consider cost-effectiveness when deciding whether to cover and pay for new therapies, though it has made exceptions for certain preventive services. The Affordable Care Act forbids the Patient Centered Outcomes Research Institute from considering ratios of cost per quality-adjusted life-year (QALY), the usual way of presenting the results of cost-effectiveness analysis, as thresholds in establishing which health care services are cost-effective or recommended. Reasons for the opposition to costeffectiveness analysis are multifaceted and reflect mistrust of the underlying methods or the motives of the parties conducting the analyses, or a desire on the part of many Americans to deny or downplay the underlying problem of resource scarcity in health care.3 Developments over the past year may signal a shift, however. In 2014, the American College of Cardiology and the American Heart Association released new guidance for developers of clinical practice guidelines emphasizing the importance of “value” considerations in such guidelines and have chosen to highlight ranges of cost-per-QALY thresholds as complements to traditional grading methods based on the strength of the clinical evidence.4 At the same time, the Institute for Clinical and Economic Review, a nonprofit organization that uses cost-effectiveness analysis as part of a process for assessing the value of drugs and other technologies, has received widespread attention for a series of reports on therapies for heart failure, multiple myeloma, and other conditions.5 Other organizations, such as the American Society for Clinical Oncology, Memorial Sloan Kettering Cancer Center, and the National Comprehensive Cancer Network, have released their own value frameworks that examine components such as clinical benefit, adverse events, and quality of life, though they do not aggregate these measures into formal cost-effectiveness analyses. Then, in September 2016, the Second Panel on Cost-Effectiveness in Health and Medicine, which we cochaired, published a report revising guidance on various aspects of cost-effectiveness analysis reflecting advances in the field.2,3 A key change pertains to the recommended perspective for analyses. The original panel on costeffectiveness, convened in 1993 by the U.S. Public Health Service, recommended that to improve the quality and comparability of costeffectiveness analyses, analysts should use a “reference case” incorporating a set of standard methodologic practices. It further advised that reference-case analyses should assume a “societal perspective,” reflecting the viewpoint of a decision maker considering the broad allocation of resources across the population. Under such a perspective, the analyst considers all parties affected by the intervention and counts all significant outcomes and costs that f low from it, regardless of who experiences them. Our panel (which was convened by a leadership group that Population Health C st-Effectiv ness Analysis 2.0