Question to the Department of Health and Social Care:

To ask His Majesty's Government when the National Institute for Health and Care Excellence will publish its report on the impact of its 2022 manual changes as set out in the England Rare Diseases Action Plan 2024.

In the 2022 England Rare Diseases Action Plan, action 13 committed to capitalising on the changes made to the National Institute for Health and Care Excellence’s (NICE) 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. An update was provided in the 2024 action plan, pointing to ongoing work to review the impact of the severity modifier and the highly specialised technology criteria.

NICE has now concluded its review of the implementation of the severity modifier and its report was presented to the NICE board in September 2024. NICE’s approval rate overall has increased compared with its previous methods and the severity modifier has contributed to an increase in positive decisions for both cancer and non-cancer medicines. The severity modifier has been applied to diseases including non-end-of-life cancers as well as non-cancer conditions that have dramatic and far-reaching impacts on patients, such as cystic fibrosis and chronic hepatitis D. A copy of the report is attached.

The 2025 England Rare Diseases Action Plan was published on 28 February 2025 providing further updates on progress on this action.