UK’s MHRA launches pilot program to guide industry on use of RWE for regulatory and HTA decisions

The RWE Scientific Dialogue Programme will provide a structured platform for early discussions between stakeholders and the MHRA, focusing on the generation and application of RWE in the development, authorization, and monitoring of medical products.

The Medicines and Healthcare products Regulatory Agency (MHRA), the UK’s medicine regulation body, has announced a new initiative to advance the incorporation of real-world evidence (RWE) into regulatory decision-making processes. The RWE Scientific Dialogue Programme, currently in its pilot phase, will offer pharmaceutical developers a structured framework to seek guidance from the MHRA. Through these discussions, the agency hopes to develop a shared understanding of how RWE can be effectively used to support regulatory submissions as well as health technology assessment (HTA) decision-making processes. The program aligns with the MHRA’s new Data Strategy, which presents a comprehensive plan to harness data, digital technology, and RWE to prioritize patient safety, safeguard public health, and drive innovation in healthcare regulation.

The program has four main aims to support the effective incorporation of RWE into regulatory submissions:

1. MHRA’s expectations for RWE methodologies: Establishes clear requirements for study designs, data quality, and analytical approaches, ensuring that RWE studies are methodologically robust and fit for regulatory evaluation.
2. Early guidance and feedback: Provides a platform for structured dialogue between the MHRA and stakeholders, enabling collaboration to ensure RWE studies meet the necessary regulatory standards.
3. Refining RWE generation for regulatory and HTA: Defines the standards for data sources, datasets, analytical methodologies, and endpoints to ensure the generation of high-quality RWE that supports both regulatory and HTA processes.
4. Information sharing: Promotes shared learning through the development of reflection papers in collaboration with the MHRA, NICE, and industry stakeholders, fostering a unified approach to the use of RWE.

Participation in the program is open to a wide range of medicine developers seeking guidance on pre- and post-authorization evidence generation for claims related to both effectiveness and safety. The program will include applications with evidence generation plans that feature interventional and non-interventional studies, including external control arm studies, pragmatic clinical trials, and pharmacoepidemiology studies such as cohort and case-control designs. These approaches support evidence generation for medicinal products, including drugs, biologics, vaccines, and advanced therapy medicinal products (ATMPs). During the pilot phase, medical devices and medicines under active regulatory review are excluded from consideration.

Priority will be given to applications addressing pressing public health challenges, unmet clinical needs, or preventative medicine. Studies utilizing genomic data, biomarkers, precision medicine, or innovative methodologies that improve representativeness and measure real-world effectiveness will also be prioritized.