Speaker
Description
Randomised controlled trials (RCTs) are the gold standard of evidence to support causal conclusions on the benefits and risks of medicines in regulatory decision making along the lifecycle [1]. However, single-arm trials (SATs) are also frequently used for various reasons during drug development. While RCTs allow adjustment for confounding via design, the contextualization of SATs requires more thoughtful consideration. This triggers the need of guidance. In response, the European Medicines Agency (EMA) has begun developing a concept paper on the use of external controls.
This presentation will provide a regulator’s perspective on the opportunities and challenges associated with using external controls in evidence generation throughout the lifecycle of a drug.
1] European Medicines Agency (2025). Draft Concept Paper on the Development of a Reflection Paper on the Use of External Controls for Evidence 5 Generation in Regulatory Decision-Making, EMA/CHMP/225255/2025.
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