The European Medicines Agency (EMA) has opened a public consultation on the “Reflection Paper on the use of external controls for evidence generation in regulatory decision-making”.
Recent developments in data availability and statistical/pharmacoepidemiological methodology have led to an increase in proposals using external controls to support regulatory decision-making. External controls are data derived from clinical trials, real-world data (RWD) or other data sources that could replace randomized control groups in clinical trials.
The reflection paper aims to provide a standard definition of external controls, outline when their use may be appropriate for regulatory decision-making and discuss the associated methodological challenges and limitations.
The draft document is open for consultation until 31 October 2025 on the EMA website: Development of a reflection paper on the use of external controls for evidence generation in regulatory decision-making – Scientific guideline | European Medicines Agency (EMA).
Comments should be provided using this EUSurvey form.