Key Stakeholders' Knowledge, Opinions, and Interests on Real-World Evidence in the Regulatory Process-Results of an EU-Wide Survey.

Real-world data (RWD) and real-world evidence (RWE) are increasingly gaining attention in supporting drug regulatory decision making. This study assessed knowledge, opinions, and usage patterns of key stakeholders regarding the status of RWD/RWE and AI implementation in health technology assessment (HTA) and drug regulation and aimed to identify the primary obstacles hindering adoption of these technologies. Four surveys tailored to different stakeholders were created and disseminated online to the respective target groups, including I) regulatory authorities, HTA bodies, and the pharmaceutical industry, II) academia, III) payers, and IV) patients and physicians. The responses were analyzed using descriptive statistics or qualitative content analysis with inductive coding. The survey was active from May 6, 2024 to June 30, 2024. Altogether, 221 respondents participated in the survey. Among respondents from regulatory/HTA authorities or industry, 75.4% viewed RWD/RWE as important for future decision making. Respondents from this group already using RWD (n = 56) most frequently reported obstacles regarding data quality (89.3%), data access (62.5%), and data-coding standardization (57.1%). Patients and physicians predominantly had positive expectations about the use of RWD/RWE, and 94.3% indicated willingness to share healthcare data for research, but all respondents also expressed concerns, with data privacy being most frequently mentioned (75.5%). The results show that although stakeholders are optimistic about RWD/RWE implementation into regulatory practice, our survey suggests that successful implementation may benefit from further development in several areas, including guideline harmonization, RWD infrastructure optimization and accessibility, and professional education.