Stakeholders’ Perspectives Toward the Use of Patient Registry Data for Decision-Making on Medicines: A Cross-Sectional Survey

Background The use of patient registries in regulatory, health technology assessment (HTA), and payer decision-making has gained increasing attention in recent years. Stakeholders’ perspectives toward the use of registry-based real-world evidence (RWE) are unknown. Objectives The purpose of this study was to assess stakeholders’ perspectives toward the use of RWE from patient registries in decision-making on medicines and explore factors influencing their intention to use registry data in the future. Methods European regulators, HTA/payers, and other stakeholders (industry, academia, healthcare professionals, patient representatives) were invited by email to participate in a web-based survey. The survey was open between November 2023 and January 2024 and contained 24 questions including demographics and questions about perspectives toward registry-based data for decision-making purposes. The latter consisted of 5-point Likert scale items based on the theory of planned behavior (TPB), i.e., attitudes, subjective norm, perceived behavioral control, and intention. Descriptive analyses and a logistic regression analysis (outcome: intention; determinants: demographics, attitudes, subjective norm, behavioral control) were performed. Results Included were 191 respondents (response rate: 16%), of whom 110 were regulators (58%), 24 HTA/payers (13%), and 54 other stakeholders (28%). Most respondents were between 41 and 50 years old (32%), 65% were women, and 53% had > 10 years work experience. Respondents considered registry data in the medicinal product lifecycle most informative for characterization of disease epidemiology (mean 4.4; 95% confidence interval (CI) 4.2–4.5), and least informative for comparative effectiveness (mean 3.6; 95% CI 3.4–3.7). Reaching the relevant patient population was perceived as the biggest strength (mean 3.6; 95% CI 3.4–3.8), and data quality as the largest weakness of patient registries (mean 2.4; 95% CI 2.2–2.6). Compared with regulators, HTA/payers had a similar intention to use registry data (Odds ratio (OR) 1.56; 95% CI 0.47–5.16), while other stakeholders were more frequently very open (intention) to using registry data in the future (OR 8.48; 95% CI 3.00–23.98). Respondents from organizations in Northern Europe were less often very open to using registry data in the future than respondents from multinational organizations (OR 0.19; 95% CI 0.04–0.85). Finally, respondents with a high perceived behavioral control concerning the use of registry data were more often very open to using registry data in the future than respondents with a neutral or low perceived behavioral control (OR 3.45; 95% CI 1.37–8.64). Conclusions The participants in our survey were generally open to increasing the use of registry data in the future. Nevertheless, perceived weaknesses such as data quality and accessibility will need to be addressed to align and improve stakeholders’ perspectives on the use of patient registries as an evidence basis for medicines decision-making.