Orphan Drugs
2026-07-12 · Department of Health and Social Care · HL1999 · Official question
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...