Real-World Evidence to Support EU Regulatory Decision Making-Results From a Pilot of Regulatory Use Cases

Stefanie Prilla*, Sophie Groeneveld, Alexandra Pacurariu, Maria Clara Restrepo-Mendez, Patrice Verpillat, Carla Torre, Christian Gartner, Peter G. M. Mol, Frauke Naumann-Winter, Kieran C. Breen, Nathalie Gault, Liana Gross-Martirosyan, Sylvie Benchetrit, Brian Aylward, Violeta Stoyanova-Beninska, Maura O'Donovan, Sabine Straus, Jesper Kjaer, Peter Arlett

*Corresponding author for this work

Research output: Contribution to journalReview articleAcademicpeer-review

6 Citations (Scopus)
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Abstract

Studies using real-world data (RWD) can complement evidence from clinical trials and fill evidence gaps during different stages of a medicine's lifecycle. This review presents the experience resulting from the European Medicines Agency (EMA) pilot to generate RWE to support evaluations by EU regulators and down-stream decision makers from September 2021 to February 2023. A total of 61 research topics were identified for RWE generation during this period, covering a wide range of research questions, primarily generating evidence on medicines safety (22, 36%), followed by questions on the design and feasibility of clinical trials (11, 18%), drug utilization (10, 16%), clinical management (10, 16%), and disease epidemiology. A significant number of questions were related to the pediatric population and/or rare diseases. A total of 27 regulatory-led RWD studies have been conducted. Most studies were descriptive and aimed at estimating incidence and prevalence rates of clinical outcomes including adverse events or to evaluate medicines utilization. The review highlights key learnings to guide further efforts to enable the use and establish the value of real-world evidence (RWE) for regulatory decisions. For instance, there is a need to access additional fit-for-purpose and representative data, and to explore further means to provide timely evidence that meets regulatory timelines. The need for early interactions and close collaboration with study requesters, e.g., from the Agency's scientific Committees, to better understand the research question is equally important. Finally, the review provides our perspective on the way forward to maximize the potential of regulatory-led RWE generation.
Original languageEnglish
Pages (from-to)1188-1197
Number of pages10
JournalClinical Pharmacology & Therapeutics
Volume116
Issue number5
DOIs
Publication statusE-pub ahead of print - 4 Jul 2024

Bibliographical note

Publisher Copyright:
© 2024 The Author(s). Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.

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