Workshop on the use of external controls for evidence generation in regulatory decision-making
Event
Human
Date
- Monday, 3 November 2025, All day
Location
- OnlineEuropean Medicines Agency, Amsterdam, the Netherlands
Event summary
The need for guidance on use of external controls was prioritised by the Methodology Working Party (MWP) in its consolidated 3-year workplan 2025-2027 and work has started on a draft reflection paper on the use of external controls for evidence generation in regulatory decision-making.
As part of the drafting process, MWP would like to engage with external stakeholders to explore opportunities and the potential use of external controls in the regulatory setting and discuss related methodological challenges to draw causal conclusions.
A hybrid workshop is planned for 3 November 2025. The sessions for the event will address:
- Session 1 will provide an overview of the use of external controls in submission to the EMA and will outline the motivation for the planned reflection paper on the use of external controls for evidence generation.
- Session 2 will focus on structured pre-planning of the use of external controls and will touch upon related challenges as well as outlining the role of the estimand framework and target trial emulation.
- Session 3 will focus on methodological considerations for various design aspects (for example on metrics to assess the suitability of endpoints, the definition of time zero for time-to-event data and the effect size relevance). The session will conclude with a panel discussion.
- Session 4 will reflect on the identification and evaluation of data sources for external controls. Understanding relevant parameters to identify suitable and reliable data sources as well as highlighting new developments will be the key focus of this session.
Registration is open and needed for online participation.
A video recording is made available after the event. Processing and publication of the video recording typically takes up to 60 days.