OBJECTIVES: The objective of this primary research study was to obtain perspectives around the role and importance of evidence in therapeutic assessment to inform the formulary decision-making process by diverse US payer organization stakeholders.

METHODS: Experienced stakeholders from US payer organizations were recruited to participate in an online quantitative and qualitative primary research study in May 2024. The survey explored 12 key themes, consisting of 53 questions. To analyze the results, we conducted descriptive statistics and contextual analyses.

RESULTS: The survey included 20 participants, including 4 medical directors, 11 pharmacy directors, 4 industry/trade relations, and 1 actuary representing national and regional MCOs, PBMs, and IDNs.

The most frequent sources of disease burden evidence used by payers in preparation for formulary decisions were RWE from their organization (weighted average, 4.0/5) and manufacturer-provided materials (3.45/5). For the evaluation of product-specific clinical evidence, the most frequent sources were published clinical trials (weighted average 4.45/5), professional guidelines (4.2/5), and ICER reviews (3.9/5). Of evidence to evaluate economic burden, the most frequent sources were ICER reports (weighted average, 3.6/5), own institution-built economic models (3.55/5), and AMCP dossiers (3.25/5). Overall, the most important sources of evidence to support formulary decisions were clinical guidelines (weighted average, 4.45/5), peer-reviewed publications (4.2/5), and internal claims data (3.6/5).

More specifically, payers perceived head-to-head clinical trials as the most important in competitive disease states with many therapeutic options (weighted average, 4.55/5) and for a new mechanism of action that challenges the standard of care (4.2/5). When head-to-head clinical trials were unavailable, most participants (75%) preferred to conduct their own informal indirect comparison.

CONCLUSIONS: US payers leverage various evidence sources to inform formulary decisions, but some sources are more frequently utilized. While preferences for specific evidence types vary, there is a consistent preference toward scientifically robust evidence.