Paper I
2020 February (Supplementary) (2010 Scheme) · 40 marks · 120 min

Question

Orphan drugs

Q190.3 marksShort Answers

Answer

Orphan drugs are drugs developed for the diagnosis, prevention, or treatment of rare diseases (typically affecting a very small proportion of the population), for which pharmaceutical development would otherwise be commercially unviable without special regulatory incentives (e.g., tax credits, market exclusivity, expedited approval). Examples: Imatinib (originally for chronic myeloid leukaemia), Eculizumab (paroxysmal nocturnal haemoglobinuria).

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