By Mark McCarty
The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) has unveiled two guidances for the use of real-world evidence (RWE) in regulatory decision-making. The policy seems limited to pharmaceutical clinical trials, with no indication when it will be expanded to include medical technology.
The MHRA noted that the consultation, which had opened in October 2020, drew some questions as to whether this policy will be extended to medical devices, but the agency said it will consider such an expansion once the policy is firmly in place for pharmaceutical studies. However, the agency also noted that an existing proposal to revise medical technology regulation has to be sorted through before the RWE policy can be proposed for devices.
The scope of the policy is also limited to the use of RWE in the context of conventional randomized clinical trials, which also drove some criticism for failing to include observational studies. The policy addresses the use of randomized, controlled trials (RCT) as a source of RWE, which some observers suggested is a contradiction in terms. The agency promised to review the roles of RWE and real-world data (RWD) in other study types but offered no timelines for such guidance.
The agency also noted that RCTs using a RWD data source are most likely to be used for expansions of the labeled indication for a product that is already on the market, but also for drug repurposing. However, the agency said that “nothing is completely ruled out on principle, including the investigation of new products.”
The policy includes data derived from digital wearable devices along with electronic health records, although the data from a study using these sources must be reliably collected in order to be deemed fit for purpose. Blinding of study subjects might not be necessary in an RWE-based RCT, but this requires that the endpoints be objective.
The agency also noted that the advantages of an RWD-based trial begin to diminish as the role of routine medical care diminishes in a study. While there may be some flexibility regarding reporting of adverse events, this flexibility may be determined principally by the regulatory experience with the product and/or similar pharmaceutical agents, the agency said.
Source: https://www.bioworld.com/articles/514476-uks-mhra-posts-guidelines-for-use-of-real-world-evidence-but-med-tech-in-limbo