Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, how many times a medicine for a rare disease has received a negative recommendation as a result of lack of data through the single technology appraisal process.
The National Institute for Health and Care Excellence has published two technology appraisals on medicines for rare diseases where they have received a negative recommendation as a result of a lack of data:
- cenegermin for treating neurotrophic keratitis [TA532]; and
- darvadstrocel for treating perianal fistula in Crohn’s disease [TA556].