On 13 January 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) launched its pilot Real-World Evidence (RWE) Scientific Dialogue Programme, which will run throughout 2025.
The programme is part of the MHRA's wider Data Strategy for 2024-2027 following the Pro-innovation Regulations of Technologies review in 2023, which identified the critical role of regulators in enabling innovation-led growth. Harnessing the potential of data, digital tools and evidence generation is key to enabling innovation at multiple points in the life-cycle of medical products.
In parallel with the adopted regulatory guidelines, the pilot seeks to facilitate and refine evidence generation to benefit both regulatory and health technology assessment (HTA) evaluations in the UK.
The objectives of the pilot programme are fourfold:
- Consistent with the established standards for evaluating the safety, quality and efficacy/effectiveness and promoting public health and protect patient safety, the pilot sets out the MHRA's expectations for the methodologies for generating robust evidence to address specific regulatory and HTA questions.
- Complementary to the existing scientific advice procedures, a safe harbour environment is created to enable commercially-sensitive discussions to take place between applicants and the MHRA on evidence-generation strategies.
- It seeks to increase clarity of the regulatory and HTA expectations for data sources, datasets, methodologies, and outcome measurements for general RWE.
- The learning from the pilot programme will be disseminated to inform the future collaborations between MHRA, National Institute for Health and Care Excellence (NICE) and the industry stakeholders.
For the purpose of the pilot programme, real-world data (RWD) is defined as data relating to patient health status and RWE is defined as evidence derived from the analysis of RWD.
Whilst the pilot is agnostic of the product types (chemical drugs, biologicals, and advanced therapies) covering evidence generation derived from interventional and non-interventional studies for evaluating effectiveness and safety, the pilot is limited to a number of applications that represent a diversity of disease areas, product-types and stages of the authorisation.
Accordingly, priority is given to applications focusing on: pressing public health challenges, areas of significant unmet clinical need, preventative medicine, genomic data, biomarkers or precision medicine approach, and innovative methodologies or study design that advance that field. Applications which are not selected can continue to benefit from scientific advice procedures.
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