To ask His Majesty's Government what assessment they have made of the impact of changes to the National Institute for Health and Care Excellence's Methods and Process on patients with rare and ultra-rare diseases.

A number of the changes made by the National Institute for Health and Care Excellence (NICE) to its methods and processes for health technology evaluation are expected to benefit medicines for rare diseases, including the introduction of a severity modifier and greater flexibility in responding to uncertainty. NICE has committed to assessing the impact of the changes on patients with rare diseases.

Due to length of the medicine’s evaluation process and number of rare disease topics using older methods or processes, the analysis of the impact of changes will take some time to assess. Data is being collected which will allow, for example, the percentage of positive NICE recommendations made following old methods and processes compared with new methods and processes for rare diseases, to be assessed. NICE will be in a position to publish the impact of the 2022 manual changes in a report by the end of 2024.