Orphan drugs revisited : [comment]

Hughes et al.1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive. The aims of this paper are to correct some inaccuracies in the original paper, develop some of the key issues, and to draw some conclusions regarding the question ‘Do drugs for exceptionally rare disease deserve special status for funding?’ For ease, our paper adopts the same structure as the original.