Multiple Perspectives on the Need for Real-World Evidence to Inform Regulatory and Health Technology Assessment Decision-Making: Scoping Review and Stakeholder Interviews.

Purpose: Real-world evidence (RWE) is increasingly considered in regulatory and health technology assessment (HTA) decision-making, though perspectives on its relevance may vary. Expanding on a recent review regarding regulatory decisions, this study aimed to identify factors influencing the need for RWE in HTA decision-making, confirm and enrich factors with stakeholder views, and evaluate similarities and differences between regulatory and HTA needs.

Methods: Previous scoping review methodology was used to identify factors influencing the need for RWE in HTA decision-making. Semi-structured interviews with stakeholders were conducted to confirm and enrich literature-derived factors for both regulatory and HTA contexts. Insights from the reviews and interviews were combined to explore similarities and differences in RWE needs across these domains.

Results: The HTA review, featuring 118 articles, revealed two major themes and six subthemes, encompassing 45 factors. The need for RWE depended on (1) questions addressable with RWE, and (2) contextual factors. Stakeholder interviews confirmed literature-derived factors. While contextual factors aligned between regulatory and HTA decision-making, question-related factors partly differed. Unlike the benefit-risk assessment in regulatory decision-making, RWE serves as direct input for the HTA, and involves specific details and a broader scope. Regulators require RWE for orphan status submissions, alternative approval pathways and to evaluate the impact of risk minimization measures, whereas HTA uses RWE to guide comparator selection, evaluate treatment implementation, quality of care and general healthcare impacts.

Conclusion: Contextual factors that influence the need for RWE are similar between regulatory and HTA decision-making, with variations seen in questions addressable with RWE.