Written Question·Department of Health and Social Care·UIN HL1999

Orphan Drugs

Tabled: 13 July 2026
Answered: 20 July 2026

Question

To ask His Majesty's Government what assessment they have made of the UK's fall in ranking from fifth to eleventh between 2020 and 2025 in the EFPIA Waiting to Access Innovative Therapies Indicator for orphan medicine availability; and what steps they are

Answer

No specific assessment has been made. The Government recognises how important it is that patients with rare diseases are able to benefit from access to effective new medicines. The National Institute for Health and Care Excellence (NICE) has a strong trac...
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