Rare Diseases: Drugs

(asked on 16th January 2024) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment her Department has made of the potential impact of the (a) methods and (b) processes used by the National Institute for Health and Care Excellence on access to medicines for patients with rarer diseases since January 2022; and whether this assessment will be reflected in the next Rare Diseases Action Plan for England.


Answered by
Andrew Stephenson Portrait
Andrew Stephenson
Minister of State (Department of Health and Social Care)
This question was answered on 23rd January 2024

The National Institute for Health and Care Excellence (NICE) can recommend most rare disease medicines that it appraises through the technology appraisal programme for some or all eligible patients. 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 seek to mitigate the barriers faced by rare disease technologies while maintaining an evidence-based, robust and proportionate evaluation approach.

The 2022 England Rare Diseases Action Plan was published following the NICE methods and processes review. The 2022 Action Plan includes actions to capitalise on the changes made to NICE’s methods and processes to ensure that NICE continues to support the rapid adoption of effective new treatments for National Health Service patients with rare diseases. The impact of these changes is being assessed and an update will be provided in the 2024 England Rare Diseases Action Plan.

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