The European Medicines Agency (EMA) has launched two public consultation processes:
Patient experience data
Feedback from medicine developers, patient groups, researchers and other decision-makers on this consultation will help shape the EU approach to patient experience data with the aim to improve evidence generation and optimise the development of future medicines towards outcomes most relevant to patients.
The consultation is open until 31 January 2026. Further information is available on the EMA website.
Use of external controls
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 EU Survey form.
Why this matters for rare diseases
People living with a rare disease and their advocates bring essential expertise to define meaningful outcomes and acceptable trade‑offs.
Thoughtful contributions to these consultations can help ensure that future EU guidance supports earlier diagnosis, better care and more inclusive research.
