UK – MHRA – Use of RWD to Support Regulatory Decisions

RWE 201 – UK – MHRA – Use of RWD to Support Regulatory Decisions


MHRA RWD Guidance:

The MHRA (Medicines and Healthcare products Regulatory Agency) has produced a comprehensive series of guidelines addressing the use of Real-World Data (RWD) in clinical research, particularly aiming to guide sponsors who intend to use RWD to support regulatory decisions.

Key Highlights:

[1] Definition and Sources: RWD is defined as data relating to patient health status or healthcare delivery, collected outside the controlled environment of a clinical study. EHR, primary and secondary care records, and disease registries are key sources of RWD. The guidelines also recognize newer sources like wearable devices and specialized websites.

[2] Purpose and Scope: The guidelines are intended to inform the design of studies that leverage RWD for evidence suitable for regulatory decisions, irrespective of geographical location. It outlines requirements for gaining approval for such studies to be conducted in the UK.

[3] Benefits: Using RWD can potentially expedite research and reduce costs. RWD can also make certain previously unfeasible studies viable, resulting in a broader understanding of treatments’ real-world effectiveness.

[4] Data Quality: Ensuring the data source’s quality is crucial; otherwise, the benefits of using RWD become moot. Key considerations include the source data’s accuracy, validity, reliability, and provenance. Pre-study protocols must address questions about data quality, population size, data capture details, data availability, interoperability issues, and more.

[5] Methodological Rigor: Traditional principles of evidence strength, such as randomization and blinding, still apply when using RWD. The guidelines indicate that sponsors should clarify how data quality checks, data extraction, and data handling will be performed and validated.

[6] Digital Health Technologies: Digital platforms, including wearables and sensors, are recognized as valid RWD sources. These technologies must be validated for their intended use.

[7] Ethical and Legal Aspects: The guidelines stress the importance of understanding privacy and security policies associated with database use, including data transfer and storage restrictions.

[8] Inspection: MHRA will continue its risk-based Good Clinical Practice (GCP) inspections. The integrity of the reported data will be a focus during these inspections, which may also involve reviewing the systems and processes used for RWD data management.

[9] Sponsor Responsibilities: Sponsors must clearly outline methods for data selection, extraction, transfer, and handling in the study protocol. Validity of the RWD should be formally documented and approved before study initiation.

Future Outlook:

Overall, the guidelines strive to provide a structured approach for incorporating RWD into clinical research, ensuring data integrity, methodological rigor, and compliance with ethical and legal standards. By doing so, they aim to pave the way for more effective and rapid healthcare solutions.

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