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Treating rare diseases: the challenge of orphan drugs

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Orphan drugs are drugs used to treat rare diseases. In April 2025, NICE adopted new criteria for evaluating drugs for 'ultra rare' diseases.

Publication type:
policy brief
Publication Date:
2025-07
Language:
en
Publication URL:
https://doi.org/10.58248/RR83
Institute(s):
Parliamentary Office of Science and Technology of the UK Parliament (POST)
Country:
United Kingdom of Great Britain & Northern Ireland