Within Work Package 5 (WP5) of the More-EUROPA project, which includes examining how patient registries are integrated into regulatory decision-making across Europe, a recent study analyzed the use of real-world data (RWD) in post-authorisation measures (PAMs) for advanced therapy medicinal products (ATMPs) approved in the European Union.
The authors systematically reviewed post-authorization measures (PAMs) for all ATMPs approved in the European Union between 2013 and 2024, extracting information from European Medicines Agency (EMA) documents and complementary databases such as the HMA-EMA Catalogue of RWD studies and sources and ClinicalTrials.gov.
Out of 25 ATMPs approved by the European Commission in this period, 118 PAMs were identified, with 49 (41.5%) involving the use of RWD. Most RWD-PAMs were imposed by regulators (69.4%) and relied primarily on secondary data sources (57.1%). Patient registries were the dominant source of RWD (53.1%), underscoring their central role in monitoring the long-term safety and effectiveness of ATMPs.
Overall, the findings illustrate the increasing importance of RWD in the post-authorization setting for ATMPs, reflecting both regulatory flexibility and the need to address benefit–risk uncertainties that persist at the time of approval. Strengthening the use of RWD, particularly through structured registry-based data collection, could enhance evidence generation, reduce residual uncertainties, and ultimately support more informed and adaptive regulatory decision-making for innovative therapies in the European Union.
The full paper can be found via the following link: Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024
