Real World Evidence: Final Reflection Paper on RWD in NIS

The use of real-world data (RWD) in the development, approval and monitoring of medicinal products continues to increase. In May 2024, the EMA published a draft Reflection Paper “On use of real-world data in non-interventional studies to generate real-world evidence for regulatory purposes” for comment. This Reflection Paper discusses methodological aspects of non-interventional studies (NIS) using real-world data (RWD) to generate real-world evidence (RWE) for regulatory purposes. Now, the final version of the Reflection Paper was published on the EMA's RWE page.

While clinical trials are the main source of information for assessing the benefits and risks of drugs in approval procedures, NIS have so far tended to be 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 or sponsor-defined and thus highly controlled settings. As part of the 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 RWE.

Guidance on Real-World Evidence

The paper is part of a series of guidance documents to be developed and published by the EMA for the area of real world data. An overview can be found on the EMA's website on real-world evidence in the chapter “Guidance on Real-World Evidence”. In the document “Journey towards a roadmap for regulatory Guidance on real-world evidence”, the EMA has listed which areas are to be supported by guidance documents in the coming years (e.g. use of RWD in clinical trials).