Rare Diseases: Drugs
(asked on 16th October 2023) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, how many single technology appraisals for medicines for rare diseases conducted by the National Institute for Health and Care Excellence resulted in (a) a positive recommendation, (b) an optimised recommendation, (c) a recommendation for managed access, (d) a negative recommendation and (e) termination in each financial year since 2018-19.
The following table shows a breakdown of technologies with designated orphan status appraised through the National Institute for Health and Care Excellence resulted single technology appraisal process by financial year from 2018/19:
Financial Year | 2018/19 | 2019/20 | 2020/21 | 2021/22 | 2022/23 | Total |
Recommended | 2 | 1 | 6 | 11 | 5 | 25 |
Recommended (Cancer Drugs Fund) | 2 | 2 | 4 | 0 | 0 | 8 |
Optimised | 3 | 9 | 3 | 3 | 8 | 26 |
Optimised (Cancer Drugs Fund) | 1 | 1 | 0 | 1 | 0 | 3 |
Research Only | 0 | 0 | 0 | 0 | 0 | 0 |
Not Recommended | 2 | 0 | 0 | 1 | 2 | 5 |
Total | 10 | 13 | 13 | 16 | 15 | 67 |
Terminated | 1 | 5 | 5 | 10 | 2 | 23 |
Medicines with an orphan designation are for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare, namely affecting not more than five in 10,000 people in Great Britain, or where the medicine is unlikely to generate sufficient profit to justify research and development costs.