Question to the Department of Health and Social Care:

To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 9 September (HL2139), what interim measures are in place to help support treatments for rare diseases to navigate the highly specialised technologies programme while The National Institute for Health and Care Excellence review of its approach to valuing rare disease medicines through its Health Technology Assessment Innovation Laboratory is undertaken.

The National Institute for Health and Care Excellence (NICE) is in the early stages of its Health Technology Assessment Innovation Laboratory project on valuing rare diseases. A detailed project scope will be communicated to stakeholders by December 2026. NICE expects the project to take two years.

NICE’s published methods and processes for health technology evaluation remain in place. NICE has a strong track record of recommending medicines for rare diseases, and since March 2024, NICE has recommended 89% of the rare disease medicines, including advanced therapies, that it has appraised for some or all of the patients who would be eligible under the marketing authorisation.

NICE also operates a highly specialised technologies programme for the evaluation of a small number of medicines for the treatment of very rare diseases.