In December 2020, we posted about the MHRA’s draft guidance on randomised controlled trials generating real-world evidence (RWE) to support regulatory decisions. As we noted in our previous blog, although real-world data (RWD) are widely used to monitor the performance of medicines and devices in patients after regulatory approval, RWD have been utilised much less frequently to demonstrate the safety and efficacy of a product at the stage of initial authorisation. The MHRA aims to provide sponsors with points to consider when planning to conduct clinical trials using RWD sources, and to provide information on the design of studies seeking to generate evidence suitable for supporting regulatory decisions. It is hoped that a greater use of RWD, and more uniform collection and use, will accelerate the availability of cost-effective treatments and reduce the time and cost currently required to generate relevant data.
Following a public consultation on the draft guidance, the MHRA issued its guidance at the end of last year in the form of two papers:
- An introduction to the RWD guideline series; and
- The first guideline in the series, on planning a prospective randomised controlled trial using RWD sources with the intention of using the trial data to support regulatory decisions.
The intention is for the MHRA to publish further guidelines in the series in due course.