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

AI Summary

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.