Workshop on the use of external controls for evidence generation in regulatory decision-making

• Date:
  Monday, 03 November 2025 , 10:00 - 17:30 Amsterdam time (CET)
• Location:
  Online
  European Medicines Agency, Amsterdam, the Netherlands

Event summary

The need for guidance on the 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 the development of a reflection paper on the use of external controls for evidence generation in regulatory decision-making - Scientific guideline. (Draft Concept Paper (CP) published for public consultation – deadline for comments: 31 October 2025).

As part of the drafting process, the MWP would like to engage with external stakeholders to explore the opportunities and the potential use of external controls in the regulatory setting and to discuss related methodological challenges to draw causal conclusions.

As stated in the ACT EU multi-annual workplan 2025-2026, a hybrid workshop is organised on 3 November 2025.

The sessions of the event address:

• Session 1 provides an overview of the use of external controls in submission to the EMA and outlines the motivation for the planned reflection paper on the use of external controls for evidence generation.
• Session 2 focuses on structured pre-planning of the use of external controls and touches upon related challenges as well as outlining the role of the estimand framework and target trial emulation.
• Session 3 focuses 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 concludes with a panel discussion.
• Session 4 reflects 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 are the key focus of this session. 

A video recording is made available after the event. Processing and publication of the video recording typically takes up to 60 days.

Documents

Agenda - Workshop on the use of external controls for evidence generation in regulatory decision-making

Draft

Registration

• Registration for online participation is open until 2 November
• Registration is required for online participation.