Abstract
In 2016, The Professional Society for Health Economics and Outcomes Research (ISPOR) formed a special task force (STF) to review approaches and methods to support the definition and use of high-quality U.S. value frameworks. As the leadership group of that initiative, we present our perspective, focusing on implications for the managed care pharmacy community. Our reflections are organized by 9 key observations and conclude with a summary recommendation. We begin by emphasizing the importance of distinguishing among "perspectives" and "decision contexts." Possible perspectives include patient, payer, provider, health care sector, and societal. Decision contexts range from formulary inclusion to guideline development to clinical shared decision making, and multiple perspectives can be taken on each of these decisions. The STF focused on value in the context of including a new medicine in a formulary and, thus, health plan, using a health economics approach that compares marginal benefit (gross value) and marginal (opportunity) cost, yielding the net value. Health care is unique compared with other markets. While economists often use market purchases as indicators of value, they also recognize that this does not work well in health care, since most patent-protected drugs are covered by insurance. To assess the likely health and economic impact, health economists often employ cost-effectiveness analysis, using the quality-adjusted life-year (QALY), a metric that combines mortality and morbidity into a single preference-based index. We strongly endorse the STF's recommendation that payers should use the cost-per-QALY metric as a starting point. However, like the STF, and many of those stakeholders who provided input, we recognize that this metric has some limitations in theory and in practice. Nonetheless, the cost-per-QALY metric is a pragmatic tool that can be augmented to address some of its limitations by integrating other elements of value, particularly those related to uncertainty, such as financial risk protection, health risk protection, the value of hope, real option value, and the value of knowing. The resulting adjusted ratio can be compared with a willingness-to-pay threshold or combined in a measure of net monetary benefit. Alternatively, the array of elements can be valued using multi-criteria decision analysis. We end with the key recommendation that further development and testing of these promising approaches is needed to improve the deliberative process of health technology assessment. DISCLOSURES: No outside funding supported the writing of this article. The authors are leaders of the ISPOR Special Task Force on U.S. Value Frameworks. Willke is employed by ISPOR. Garrison and Neumann have nothing to disclose. The opinions expressed in this article should be considered as belonging only to the authors.