A Cross-Sectional Survey of Evidence Needs for Medicinal Products in Europe With a Focus on Real-World Evidence.

purposeTo assess views among different stakeholders around evidence needs during assessment of clinical effects across the medicinal product life cycle, with a focus on real-world evidence (RWE) in regulatory and health technology assessor (HTA)/payer decision-making.

methodsA cross-sectional survey was distributed between November 2023-January 2024 to 1210 European regulators, HTAs, payers, and other stakeholders (i.e., industry, academia, healthcare professionals, and patient representatives). The survey included questions on their experience and views on evidence needs, which were analysed descriptively and with non-parametric testing.

results191 respondents (110 regulators, 24 HTA/payers and 57 others) from 32 European countries completed the survey (response rate: 16%). Most respondents were between 41-60 years and 53% had > 10 years of work experience. Respondents were least confident in assessing evidence based on adjusted indirect comparisons and network meta-analyses. Randomization and clinically relevant endpoints were considered most relevant in studies, while country-specific data and patient preferences were considered least important. Respondents acknowledged that there are uncertainties on the clinical effect of medicinal products in the different stages of the medicinal product life cycle, but the views on the usefulness of RWE to address these uncertainties varied across stakeholder groups and life-cycle stages. RWE was considered most likely to be accepted as supplementary evidence for orphan diseases and when randomized controlled trials (RCTs) are infeasible.

conclusionOur findings highlight opportunities and challenges in using RWE to reduce uncertainties in decision-making around medicinal products. This may contribute to a dialogue on how and when to augment evidence from RCTs with RWE.