Presentation (CTI)

OBJECTIVES: US payers increasingly recognize that real-world evidence (RWE), when combined with data from randomized controlled trials (RCTs), can be a powerful tool to guide formulary and coverage decisions. RWE has gained traction in regulatory submissions following Food and Drug Administration (FDA) guidance on its use for supporting initial drug approvals and new indications. However, no comparable guidance exists for payers on how to evaluate and incorporate RWE into coverage and reimbursement decisions. As a result, despite its potential to inform comparative effectiveness, safety, and economic outcomes, payer adoption of RWE remains limited. The objective of this research is to identify key barriers, priorities, and frameworks for integrating RWE into US payer decision making.
METHODS: A targeted literature review was conducted to identify recent studies and policy initiatives addressing the role of RWE in US payer decision making.
RESULTS: Payers acknowledged the value of RWE for informing reimbursement decisions, as reported in published interviews showing strong interest among payer stakeholders. In these interviews, 100% of respondents agreed that RWE is becoming influential, and 75% expressed an appetite for its use. Non-experimental studies, such as registries, were considered useful for assessing effectiveness and safety of medical products. Despite this, RWE is not routinely incorporated into payer value assessments. Key barriers include the timeliness of evidence (often generated post-launch, after coverage decisions), limited understanding of optimal use, absence of standardized guidelines, and lack of transparency in study methods. Recommended solutions include improving methodological transparency, engaging payers early in study design, and developing consensus-based frameworks to support payer education and interpretation of RWE.
CONCLUSIONS: RWE, as a complement to data from traditional RCTs, holds significant promise for payer decision making. Establishing common standards, improving transparency, and fostering early collaboration can enable consistent and trusted use of RWE in US payer decision making.