Toward Relevant and Credible Cost-Effectiveness Analyses for Value Assessment in the Decentralized U.S. Health Care System.

In the United States, there is an increased interest to understand the value of health technologies. Cost-effectiveness analysis is arguably the most appropriate framework to quantify value and to inform reimbursement decision making regarding medical interventions; however, a thorough analysis is resource intensive and complex. In many countries, the cost-effectiveness of medical interventions is evaluated by expert agencies at the national level, but in the United States, reimbursement decision making occurs at the local level. This raises the question of how we can provide a means to transparent cost-effectiveness analysis that reflects the local context and patient population and is based on the latest evidence and scientific insights. In other words, how can we maximize the relevance and credibility of cost-effectiveness evaluations in the context of a decentralized decision-making environment? Published cost-effectiveness analyses typically fail on these dimensions. Access to transparent open-source models that can be adapted to reflect the local setting in a relatively straightforward manner is an essential step toward such a goal. However, no model for cost-effectiveness analysis is ever truly "right" or "complete," and it must evolve along with clinical evidence and improvements in scientific methodology to ensure that its credibility remains. We propose a transparent approach of iterative development and collaboration between content and methodology experts to produce up-to-date, open-source consensus-based cost-effectiveness models that account for parameter and structural uncertainty to help local decision makers understand the confidence with which they might make a decision. Our proposed approach provides a way to adapt formal assessments of value-long the province of centralized health care systems-into the decentralized U.S. health care landscape. DISCLOSURES: This research was funded through the Innovation and Value Initiative, a nonprofit multistakeholder research organization. The Innovation and Value Initiative contracted with Precision Medicine Group for research activities related to this article. Jansen and Incerti are salaried employees and shareholders of Precision Medicine Group. Curtis is a paid consultant for the Innovation and Value Initiative. Curtis also reports consulting fees and grants from Amgen, AbbVie, BMS, Corrona, Janssen, Lilly, Myriad, Pfizer, Roche/Genentech, Radius, and UCB, unrelated to this article.