- 31 Dec 2021
- NEWS
Executive Summary
The UK Medicines and Healthcare products Regulatory Agency has finalized the first part of a series of new guidance documents addressing issues around using real-world data in support of regulatory submissions. 
Source: AlamyUSE OF RWD CAN MAKE CLINICAL TRIALS MORE COST-EFFECTIVE
The UK medicines regulator has finalized much-awaited guidance explaining how drug sponsors can conduct randomized controlled trials (RCT) primarily using real-world data (RWD) sources to support a regulatory decision relating to a medicinal product.
Its use could make it more feasible for sponsors to repurpose their existing medicines for new conditions and help bring life-changing new medicines more quickly to those who need them, the Medicines and Healthcare products Regulatory Agency said.
UK industry body, the ABPI, said it was important now to look at how to help put the guidance into action in an appropriate manner.
The RCT guideline offers advice on clinical trial authorization for sponsors wanting to run such a trial wholly or in part in the UK. It also offers guidance on clinical trial design, including choice of endpoints and safety data requirements. The guideline does not cover other types of studies that could be run using RWD such as observational studies, or clinical trials using RWD as a control arm.
This is the first in a planned series of guidance documents being developed by the MHRA on how to use RWD to support regulatory approval. To this end, the UK agency also has issued a separate guideline to introduce its RWD guideline series and to provide general points to consider when evaluating whether a RWD source is of sufficient quality for the intended use.
“It is good to see regulators’ guidance bring innovation into clinical trials while maintaining the principles of data quality and transparency.” – Steve Hoare, UK ABPI
The UK pharmaceutical industry association, the ABPI, said greater use of RWD could improve assessment of medical innovations and make sure they bring maximum benefit to patients. “These are priorities for industry,” said Steve Hoare, the ABPI’s Quality, Regulatory Science & Safety Policy Director.
“It is good to see regulators’ guidance bring innovation into clinical trials while maintaining the principles of data quality and transparency. We must now look at how to help put this guidance into action for the UK’s real-world data, so that the evidence generated in trials continues to be as robust as possible,” Hoare told the Pink Sheet.
Stakeholder Feedback Integrated
The two MHRA guidelines, initially grouped in a single document, were issued for stakeholder consultation earlier this year and drew responses from a range of stakeholders, including pharmaceutical companies, academic groups, patient organizations/charities, contract research organizations and healthcare providers/regulatory organizations. (Also see “UK’s First Real-World Evidence Guideline Targets Randomized Trials” – Pink Sheet, 30 Oct, 2020.)
Based on stakeholder feedback, the MHRA has made several changes to the document. For example, it split the guideline into two documents – one on RWD data quality, and the other on RCTs – to address concerns about the lack of clarity about its scope.
In addition, some stakeholders sought clarity on whether trials using RWD that were conducted outside of the UK would be acceptable to the MHRA. The UK regulator said the answer to this question was “’yes,’ but the comments led to the realization that the discussion related to clinical trial approval (which is UK specific) had been intertwined with the general points on trial design, leading to a lack of clarity.” In the revised guideline, therefore, these two aspects have been clearly separated.
Efforts Elsewhere
Regulators in other regions are also making efforts to support the uptake of RWD in regulatory decision-making.
The two UK guidelines were issued on 16 December. Also in December, the US Food and Drug Administration issued draft guidance on “Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.”
Australia’s Therapeutic Goods Administration has also indicated plans to develop guidance on this topic in 2022.