AMCP real-world evidence standards: Overcoming barriers to using real-world evidence in US payer decision-making.

Background: US payers are interested in incorporating real-world evidence (RWE) into their pharmaceutical coverage and reimbursement decisions. One barrier to using RWE to inform decisions is the lack of standards for RWE assessment and interpretation specific to US payer needs.

Objective: To develop RWE standards supporting US payer decision-making through a framework outlining study types and potential endpoints important to US payers throughout a product's lifecycle and a set of criteria, tailored to US payer needs, for assessing and applying RWE.

Methods: The Academy of Managed Care Pharmacy (AMCP) Research Institute, in partnership with IQVIA, convened a multistakeholder group of RWE experts to complete a survey and participate in a series of workshops with the goal of developing RWE standards tailored to payer decision-making needs. Informed by a targeted literature search, we created an initial list of RWE assessment criteria relevant to payers and refined by consensus and a framework describing RWE appropriate to different product lifecycle stages from pre- to postapproval.

Results: A total of 36 payers completed the survey, and few (18%) reported regularly using RWE as part of their decision process; however, most (80%) were interested in using it. There was consensus on the need for a set of payer-specific standards. Through 2 focus groups and an AMCP Partnership Forum, participants vetted, refined, and finalized this payer-specific framework and developed a checklist that comprised a total of 29 RWE assessment criteria across 6 categories. This framework and set of criteria became the final AMCP RWE standards.

Conclusions: The AMCP RWE standards provide a tool to facilitate payer-specific assessments of RWE and guidance for designing RWE studies and communicating results that will be most impactful for payer formulary and coverage decisions.