Written question asked by Chris Green (Conservative) on Monday, 16 October 2023, in the House of Commons. It was due for an answer on Wednesday, 18 October 2023. It was answered by Will Quince (Conservative) on Friday, 20 October 2023 on behalf of the Department of Health and Social Care.

Rare Diseases: Drugs

Question: 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.

Answer

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.

Secondary information

Type: Written question
Reference: 202779
Session: 2022-23
Related items: Rare Diseases: Drugs
Tuesday, 28 November 2023
Written questions
House of Commons
Subjects: Finance Drugs National Institute for Health and Care Excellence Rare diseases
Contains statistics: Yes
Link: View this Written question on www.parliament.uk

Rare Diseases: Drugs

Secondary information

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