The use of prior knowledge is increasingly recognised as an important means of supporting efficient medicinal product development. This Centre of Excellence in Regulatory Science and Innovation for Advanced Therapies (CERSI-AT) white paper outlines the Medicines and Healthcare products Regulatory Agency’s (MHRA’s) approach to the use of prior knowledge in regulatory submissions and how it may be leveraged to support more proportionate, risk-based evidence generation.
Prior knowledge may be derived from a range of sources, including published literature, public datasets, regulatory precedent and experience gained through research, development and manufacturing activities. Focusing on advanced therapy medicinal products (ATMPs), the paper examines the evolving regulatory landscape in the UK, EU and US and highlights opportunities and considerations for applying prior knowledge throughout development.
The paper presents two practical case studies based on real-world developer interactions with the MHRA, illustrating how prior knowledge may be applied in regulatory submissions. The examples demonstrate how prior knowledge can support platform-based development strategies and scientifically justified bridging approaches, helping to focus evidence generation and reduce unnecessary duplication of studies.
The paper also highlights key considerations for developers, including the importance of a robust scientific rationale, a risk-based approach to addressing residual uncertainties, and early engagement with regulators.
Read the white paper
