Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether the National Institute for Health and Care Excellence has made an assessment of the potential merits of undertaking modular updates on topics relevant to rare diseases since the publication of its updated process and methods for health technology evaluation on 31 January 2022.

The National Institute for Health and Care Excellence (NICE) has no current plans to undertake a modular update of its methods and processes related specifically to rare diseases. NICE specifically considered treatments for rare diseases during its methods review, and many of the changes introduced in NICE’s updated health technology evaluation manual in January 2022 will benefit treatments for rare diseases. These include the severity modifier, flexibility in uncertainty considerations and the emphasis on a comprehensive evidence base including real-world, qualitative, surrogate, and expert evidence. These updates mitigate the barriers faced by rare disease technologies while maintaining an evidence-based, robust, and proportionate evaluation approach.