COMMON THREADS: REGULATORY AND HEALTH TECHNOLOGY ASSESSMENT (HTA) AGENCIES’ RECOMMENDATIONS ON REAL-WORLD EVIDENCE (RWE) USE IN DECISION-MAKING
Author(s)
Jaksa A1, Gatto N2, Case A2
1Aetion, Inc., Boston, MA, USA, 2Aetion, New York, NY, USA
OBJECTIVES: Over the past decade, there has been a debate in the literature on how to use RWE for decision-making while promoting transparency, quality, and confidence in RWE. Regulatory and HTA agencies are proactively evaluating the role of RWE to inform decision-making. However they are at different stages of guidance development and adoption. The objective of this study was to compare guidance from regulatory and HTA agencies, catalogue existing recommendations, and identify commonalities among the agencies. METHODS: Reports from prominent regulatory and HTA bodies on RWE were selected for evaluation. Key elements extracted included the objective of the publication, justification for use of RWE, recommendations for RWE generation, and use in decision-making. Themes were identified and categorized. RESULTS: A total of seven reports were identified from regulatory (FDA; EMA; Health Canada) and HTA (NICE; CADTH; ICER; INAMI) agencies. A need to accelerate patient access to treatment and technological advances in data rationalized the need to incorporate RWE into decision-making. Publication aims centered around outlining situations where RWE can be influential and the methodological and data challenges associated with RWE. Almost all publications discussed an evidence hierarchy, with RWE secondary to randomized trials. There were six main themes that emerged: transparency, fit-for-purpose real-world data, collaboration between stakeholders, principled database epidemiology, replicability, and supporting data assets and governance. While regulatory and HTA agencies aligned on common themes, clarity on how manufacturers should operationalize the themes was missing. There was a lack of concrete recommendations for how to generate RWE, beyond protocol design considerations and frameworks on how RWE will inform decision-making was likewise missing. CONCLUSIONS:Regulatory and HTA bodies are actively working to formulate positions on RWE generation and use and there is a need for transparent recommendation to better inform manufacturers.
Conference/Value in Health Info
2020-05, ISPOR 2020, Orlando, FL, USA
Value in Health, Volume 23, Issue 5, S1 (May 2020)
Code
PNS174
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices, Real World Data & Information Systems
Topic Subcategory
Approval & Labeling, Best Research Practices, Reproducibility & Replicability, Value Frameworks & Dossier Format
Disease
No Specific Disease