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

Source: https://www.keypedia.com/records/uk_mhra_guidance/uk-mhra/3d3d4b70-ea33-41a5-a97a-5fc19453eca9
Type: Guidance
Published: December 16, 2021
Updated: July 17, 2025

> The Medicines and Healthcare products Regulatory Agency (MHRA) has released a comprehensive guidance series addressing the use of real-world data (RWD) in clinical research to support regulatory decision-making. Aligned with the MHRA data strategy, this regulatory framework provides guidelines for sponsors globally who intend to use RWD sources for UK-based clinical trial authorizations. The guidance series includes an introductory document on assessing the quality of RWD sources and a specific guideline for prospective randomized controlled trials. The randomized trial guideline outlines critical requirements for trial design, including the selection of endpoints and the management of safety data. Additionally, the MHRA recently concluded a stakeholder consultation period running from May 20, 2025, to July 14, 2025, regarding a new draft guideline for using external control arms based on RWD. While this document is a general guidance series rather than an inspection report for a specific company or date, it establishes the expectations for all research sponsors. Required actions for organizations include performing rigorous quality evaluations of their RWD sources and adhering to specific trial design and safety reporting standards when seeking regulatory approval. Sponsors are further encouraged to monitor the outcomes of these consultations to ensure their methodologies remain compliant with the evolving regulatory landscape.

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## Content

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 .

## Attachments

- [MHRA guidance on the use of real-world data in clinical studies to support regulatory decisions](/government/publications/mhra-guidance-on-the-use-of-real-world-data-in-clinical-studies-to-support-regulatory-decisions/mhra-guidance-on-the-use-of-real-world-data-in-clinical-studies-to-support-regulatory-decisions)
- [ MHRA guideline on randomised controlled trials using real-world data to support regulatory decisions](/government/publications/mhra-guidance-on-the-use-of-real-world-data-in-clinical-studies-to-support-regulatory-decisions/mhra-guideline-on-randomised-controlled-trials-using-real-world-data-to-support-regulatory-decisions)