Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of whether current NICE appraisal processes are sufficient responsive to emerging treatments for rare diseases and advanced therapies.

The Government recognises the importance of patients with rare diseases being able to access effective new medicines. The National Institute for Health and Care Excellence (NICE) assesses whether medicines should be funded by the National Health Service. Its process has been proven to be suitable for rare disease medicines, including advanced therapies, where companies work constructively with NICE and NHS England. Since March 2024, NICE has recommended 89% of the rare disease medicines it has appraised for some or all eligible patients.