Evidence and Pricing: Bridging the Comparative Effectiveness Gap in Federal and State Drug Policy

OBJECTIVES: Federal and state drug pricing programs typically involve a review of comparative clinical evidence to understand the added benefit that interventions provide for the indicated and covered patient population. Contemporary (recent) comparative effectiveness research (CER) can be instrumental in illustrating benefit under conditions of current, real-world use to support these decisions. METHODS: We convened 7 subject-matter experts plus 2 expert moderators to discuss gaps, challenges, and opportunities around the use of contemporary CER for federal and state drug pricing programs. We synthesized and structured key recommendations for the use of CER for policy and pricing decision makers. RESULTS: Key gaps and challenges to using contemporary CER to support pricing decisions by the Centers for Medicare and Medicaid Services and state Prescription Drug Affordability Boards are centered around soliciting and generating fit-for-purpose evidence, improving transparency on how evidence is used in decision making, and mitigating resource constraints. Recommendations for policymakers include asking clear questions to solicit necessary data, considering a Population-Intervention-Comparator-Outcomes-Time framework, balancing usefulness with limitations, improving transparency on decision-making, and streamlining processes. Recommendations for manufacturers include leveraging resources, supporting scientific credibility, and tailoring evidence submissions to decision-maker realities. CONCLUSIONS: Incorporating contemporary CER is critical for informing policymakers on the value of interventions for drug pricing negotiations. Evaluating evidence, answering research questions, and inferring how evidence applies to different populations are core principles in health economics and health policy, which should be applied more rigorously and transparently in drug pricing programs.