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UK – MHRA – Randomised Controlled Trials Using Real World Data

RWE 201 – UK – MHRA – Randomised Controlled Trials Using Real World Data


MHRA RWD Guidance:

The MHRA guideline focuses on sponsors planning to conduct randomised, controlled clinical trials (RCTs) primarily using Real-World Data (RWD) for regulatory decisions concerning medicinal products.


The guideline covers:

  1. Clinical trial authorisation in the UK.
  2. Trial design, including endpoint selection and safety requirements.

It excludes discussions on observational studies, and clinical trials using RWD as a control arm.

Definition and Types of RWD Trials

RWD is health data obtained outside clinical studies and can include electronic healthcare records, disease registries, and patient-reported outcomes. In a simple RWD-based RCT, patients are randomized to receive standard clinical care alone or an added intervention. Data quality and study design need to be as rigorous as traditional RCTs for the results to be valid for regulatory decisions.

Protocol Requirements

The protocol should pre-specify objectives, data collection methods, and primary and secondary endpoints. Consent is required before enrolment, and in most real-world settings, patients are not blinded to treatment allocation.

Regulatory Acceptability

From a regulatory standpoint, the source of the data (RWD, hybrid, or traditional) is irrelevant as long as the trial answers the necessary regulatory questions. The need for blinding should be considered, especially if the primary endpoints are not sufficiently objective.

Examples and Practical Considerations

The guideline suggests that RWD-based trials can be effective when dealing with an established EHR database and objective endpoints like all-cause mortality. Scenarios where the intervention is an existing licensed product with a well-known safety profile are particularly suited for RWD trials. It’s critical not to assume the completeness of potential endpoints in EHRs. Sponsors are advised to conduct a feasibility study to assess the reliability of the data capture methods.

Data Gaps and Hybrid Trials

If the RWD source does not cover all required endpoints, a hybrid trial can supplement RWD with specific additional data. These could be additional clinical assessments, which might even be carried out remotely.

In summary, the MHRA guideline serves as a comprehensive framework for sponsors interested in leveraging RWD for RCTs aimed at supporting regulatory decisions for medicinal products. While RWD-based trials offer advantages in reducing patient and healthcare burdens, they must be designed and executed with rigor comparable to traditional RCTs to be deemed acceptable for regulatory purposes.

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UK – MHRA – Randomised Controlled Trials Using Real World Data2023-10-14T08:31:42+00:00

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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UK – MHRA – Use of RWD to Support Regulatory Decisions2023-10-14T08:25:36+00:00

UK | MHRA Guidance on the Use of Real-World Data in Clinical Studies to Support Regulatory Decisions

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UK | MHRA Guidance on the Use of Real-World Data in Clinical Studies to Support Regulatory Decisions2022-08-07T17:00:17+00:00
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