Orphan drugs revisited

Abstract

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. Hughes et al . state that a key issue is ‘whether the rarity and gravity of the condition represents a rational basis for applying a different value to health gain …’1 The defining characteristic of an orphan drug is that it treats a rare disease. However, the justification for special funding frequently rests upon the ‘gravity’ of the condition. To examine whether orphan drug legislation accurately represents societal preferences, it would be necessary to ask whether society was willing to pay more for treatments for rare severe disorders than for more prevalent severe disorders. No study has done this. Hughes et al . recount another frequently cited argument for special treatment: ‘ensuring access to treatment where no other treatment exists.’ Like ‘gravity’, this is not a defining characteristic of an orphan drug, but it is a frequently cited argument for their special status in licensing and reimbursement.1 Not being unique to orphan drugs, it cannot be a justification for their special status. Further, this argument contains an implicit preference for biological disease modification over health gain. In the developed world, ‘no other treatment’ is a substantial misrepresentation of reality; patients are not simply left with no medical treatment at …