Global Stakeholder Perspectives on Real-World Data and Evidence in Health Technology Assessment: An Exploratory Study

Objective: This exploratory study presents an international, multi-stakeholder snapshot of perceptions regarding real-world data and real-world evidence in health technology assessment. The aim is to identify perceived opportunities, barriers, and enabling conditions rather than to generate generalizable conclusions. Methods: A 21-item, expert-validated questionnaire was distributed via LimeSurvey to diverse health technology assessment stakeholders, including academia, industry, health technology assessment agencies, healthcare providers, policymakers, patients, and payers. The survey explored perceptions of value, methodological and regulatory challenges, and future outlooks for RWD/RWE use in HTA. Ethical approval was obtained by the University of West Attica Ethics Committee, and pilot testing was conducted prior to dissemination. Data were analyzed using descriptive statistics, consistent with the study's exploratory intent and acknowledging that results are preliminary and not statistically generalizable. Results: Thirty-two completed responses demonstrated preliminary stakeholder support for integrating real-world data and real-world evidence into health technology assessment. Respondents represented academia, industry, HTA agencies, healthcare providers, policymakers, and patient/advocacy groups; however, no payer responses were obtained. Respondents emphasized the value of real-world data in complementing clinical trials by capturing real-world effectiveness, patient diversity, and long-term outcomes, especially in rare diseases and cancer. Key challenges included poor data quality, confounding biases, and regulatory barriers. Stakeholders highlighted the importance of standardization, transparency, and international collaboration. Opportunities included better decision-making, personalized healthcare, and improved post-market monitoring, with strong calls for robust infrastructure, clear methodologies, patient involvement, and supportive health policy frameworks. Conclusions: Real-world data and evidence enhance health technology assessment by supporting better decisions and personalized care. However, issues like data quality, methods, and trust must be addressed through standardization, strong infrastructure, and collaboration to ensure effective and impactful implementation in healthcare, while acknowledging these insights are based on a small exploratory sample.