MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions, Public Sector article

MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions

Summary

We invite feedback on the clarity and wording of our new draft guideline on the use of external control arms based on real-world data to support regulatory decisions.

This consultation closes at
11:59pm on 30 June 2025

Consultation description

A real-world data (RWD) external control arm (ECA) comprises patient level data collected outside of a clinical study which will be used as a control or part of a control arm to estimate the comparative efficacy and safety of an intervention being studied in a clinical trial. As part of our ongoing series of guidelines on using RWD to support regulatory decisions, the MHRA, with independent scientific advice from the Commission on Human Medicines RWD ad hoc group, has drafted a new guideline.

This draft guideline provides clinical trial sponsors with points to consider and key principles that should be taken into account when planning a clinical trial which will include a RWD ECA and which will require regulatory approval. While the guideline is specifically aimed at sponsors planning to use RWD ECAs, many of the general principles would be relevant for external controls drawn from other sources, such as previously completed clinical trials.

The 6-week consultation aims to get feedback from relevant stakeholders regarding the clarity and wording of this guideline, including any perceived contradictions or omissions.

Following the consultation, the MHRA will amend and publish the guideline document as well as publishing an anonymised summary of the public feedback.

Please download the draft guideline and consultation questions for reference before responding to this consultation. You can also leave specific line-by-line comments on the draft guideline. To leave such feedback, please download the MHRA spreadsheet and refer to the draft guideline while completing it.