UK regulator publishes guidance on use of real-world data to support clinical trials

Two new guidance documents, the first in a series to published by the MHRA, outline considerations when planning a randomised clinical trial using real-world data.

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The UK’s Medicines and Healthcare products Regulatory Authority (MHRA) has published new guidance on the use of real-world data (RWD) for randomised clinical trials.

The two guidance documents were drafted with the Commission on Human Medicines Real-World Data ad hoc group following consultation with industry. They provide considerations when planning a randomised clinical trial using RWD, with the intention of submitting this trial to gain a regulatory approval.

The MHRA stated that using RWD – information generated during routine healthcare, including electronic patient health records, and disease and patient registries – to improve recruitment and aid regulatory decision-making could accelerate the development and approval of life-changing new medicines, making them available to patients more quickly.

While RWD is frequently used to monitor the safety of medicines and medical devices after they have gained approval, it is currently rarely used to demonstrate the effectiveness of an intervention before it is approved.

The new guidance is intended to be the first in a series addressing issues around using real-world evidence in support of a regulatory submission. It considers aspects related to clinical trial authorisation, clinical trial design (including choice of endpoints and safety data requirements), and requirements in terms of database quality and inspection.

The two guidance documents are:

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

An introduction to the MHRA’s RWD guideline series, outlining 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

Guidance on considerations 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.

Commenting on the publication, Dr June Raine, MHRA Chief Executive, stated: “When used in this innovative way, real-world data has the potential to make a huge difference when it comes to bringing medicines through clinical trials to patients.

“With fewer or even no trial-specific visits, consenting trial participants do not have to travel long distances to get to their appointments. And with fewer logistical hurdles, real-world data could make it more feasible for trial sponsors to repurpose existing medicines for new conditions.

“Because of this, and the growing need to find more cost-effective ways of conducting clinical trials, our new series of guidelines focuses on how to use real-world evidence to aid regulatory approval, helping to bring medicines to the patients who need them, sooner.”