EMA's Reflection Paper on the Use of Real-World Data in Non-Interventional Studies

The EMA has published a draft Reflection Paper on the use of real-world data in non-interventional studies (NIS) to generate real-world evidence (RWE) for comment. The Reflection Paper discusses methodological aspects of NIS using real-world data (RWD) to generate RWE for regulatory purposes. While clinical trials are the main source of information for the assessment of benefits and risks of medicinal products in marketing authorization procedures, NIS are so far more accepted in post-authorization safety assessments. Their acceptance for assessing the efficacy of medicinal products is currently hampered by methodological restrictions, as they are not allowed to use randomization or blinding. As part of EMA's projects to generate structured RWD and their use in regulatory decision-making processes, NIS are also becoming increasingly important as a source of information and data for RWD. The draft document on the use of real-world data in NIS to generate RWE is open for comment until 31 August 2024.

Previously, a reflection paper issued by the ICH was available for public consultation aiming to harmonize RWE terminology and enable the convergence of general principles for planning and reporting studies using real-world data to support regulatory decision-making. In addition, the FDA published a draft guidance Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products.

More information is available in EMA's Guidance on Real-world evidence and the Reflection paper on use of real-world data in non-interventional studies to generate real-world evidence published on EMA's Big data website.