Generation and Utilization of Real-World Evidence by US Payer Organization Stakeholders to Support Formulary Decisions

OBJECTIVES: The objective of this primary research study was to obtain perspectives around real-world evidence utilization and generation from diverse US payer organizations.

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

RESULTS: The survey included 20 participants, including 4 medical directors, 11 pharmacy directors, 4 industry/trade relations professionals, and 1 actuary representing national and regional MCOs, PBMs, and IDNs. Most participants (80%) currently use RWD from their institution to inform treatment coverage decisions. Those participants use RWD to analyze current drug utilization (100%), analyze current treatment patterns (88%), track HCRU (75%), assess economic burden (75%), and/or assess disease burden (56%). For disease burden evaluation specifically, 80% conduct in-house analyses at their organization, with the highest value for diabetes (weighted average, 4.2/5) and gene therapy (4.0/5). Participants felt the most useful RWE provided by a manufacturer to be RW comparative evidence (weighted average, 3.5/5), followed by RW clinical safety (3.4/5), RW economic impact (3.4/5), and RW clinical effectiveness (3.4/5). Most participants (65%) shared that post-launch RWE has played a role in product coverage or formulary within their organization. Further, most participants were moderately (45%) or slightly (30%) supportive of using RWD to substantiate label extensions or changes post-FDA approval.

CONCLUSIONS: The role of real-world evidence in formulary decision-making and its influence in the US is progressively expanding. While clinical trial evidence remains the gold standard, RWE offers strategic advantages for payers and other stakeholders in numerous scenarios, enhancing and supporting the formulary decision-making process.