Overview

In line with the MHRA data strategy, the MHRA is producing a guidance series to provide general points to consider for sponsors planning to conduct clinical research using real-world data (RWD) to support regulatory decision making.

The guidance provides information to aid the design of studies aiming to provide evidence suitable for supporting regulatory decisions. This guidance is applicable to studies regardless of geographical location.

Two guidance documents have been published and a third draft guideline on the use of External Control Arms Based on Real-World Data is now open for consultation. See MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions

Published guidance

MHRA guidance on the use of real-world data in clinical studies to support regulatory decisions:

This provides an introduction to the MHRA’s RWD guideline series, and points to consider when evaluating whether a RWD source is of sufficient quality for the intended use.

MHRA guideline on randomised controlled trials using real-world data to support regulatory decisions

This provides points to consider when planning a prospective randomised trial using RWD sources with the intention of using the trial to support a regulatory decision. The guideline covers clinical trial authorisation (if applying for approval to run such a trial wholly or in part in the UK), and clinical trial design including choice of endpoints and safety data requirements.

Consultations

Current consultation

On 20 May 2025, the MHRA launched a 6-week consultation with stakeholders on the draft guideline on the use of external control arms based on real-world data to support regulatory decisions. This consultation ran from 12:01am on 20 May 2025 to 11:59pm on 14 July 2025. An analysis of the responses will be conducted to inform the final guidance document.

Previous consultation

In October 2020, the MHRA ran a consultation on the MHRA draft guidance on randomised controlled trials generating real-world evidence to support regulatory decisions. This resulted in the current MHRA guideline on randomised controlled trials using real-world data to support regulatory decisions and MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions. See details on the previous consultation and the public feedback and detail of the outcome.

Contact us

For more details, please contact MHRA Customer Experience Centre: info@mhra.gov.uk.