(SLC-047) EU-Wide Survey on Key Stakeholders’ Knowledge, Opinions and Interests on Real-World Evidence in the Regulatory Process

Background: Accounting for views of all relevant stakeholders is important for optimal utilisation of real-world evidence (RWE) in the regulatory process. To achieve this, we conducted a survey to relevant stakeholders to gather their insights and opinions on the use of RWE and artificial intelligence (AI)/machine learning (ML) methodology in medicines regulation and health technology assessment (HTA).

Objectives: To describe key stakeholders’ views and needs regarding RWE and to gain an overview of real-world data (RWD) usage in the regulatory process.

Methods: Key stakeholders relevant to the European Union (EU) medicines regulatory process were identified via stakeholder analysis. Using EUSurvey, the open-source online survey management tool of the European Commission, four surveys were created, tailored to i) pharmaceutical industry and regulatory/HTA bodies, ii) academia, iii) payers and iv) patients and physicians. The surveys contained between 38 and 42 questions, which were structured into the domains of knowledge, opinions, current usage and interest in RWE and AI/ML methodology.
The surveys were conducted online from May until June 2024. Invitations for stakeholders to participate were sent to 448 e-mail addresses (both personal and institutional) identified during the stakeholder analysis. Additional stakeholders were invited via social media. Responses were recorded anonymously.
Descriptive statistics and qualitative content analysis using an inductive coding approach were used to analyse the survey results.

Results: A total of 221 respondents participated in the survey, including 126 from industry, regulatory or HTA organisations, 36 from academia, 6 from payers, and 53 patients or physicians. Gaps in knowledge were identified across stakeholder groups especially in the topics of AI/ML, statistics and programming. Stakeholders from industry, regulatory and HTA bodies already using RWD (n = 56) reported data quality (n = 50, 89.3%) and data access (n = 35, 62.5%) as the largest challenges. Those stakeholders not using RWD (n = 56) reported no need for RWD (n = 28, 50%) as the main reason, followed by a lack of clear regulatory guidelines (n = 15, 26.8%).
Both patients and physicians reported privacy as the main concern about the use of RWD for clinical research purposes (Patients n = 22, 81.5%; Physicians n = 16, 69.6%). Most of the responding patients (n = 26, 96.3%) were willing to share their health data – particularly for public health research & monitoring (n = 26, 96.3%), academic research (n = 25, 92.6%) and regulatory /government agencies (n = 24, 88.9%).

Conclusions: Education, cooperation and improved guidance for all stakeholders, as well as better data quality and access are needed to improve the use of and trust in RWE in the EU.