QJM Advance Access originally published online on February 27, 2006
QJM 2006 99(5):341-345; doi:10.1093/qjmed/hcl033
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Commentaries |
Orphan drugs revisited
From the 1Section of Health Economics and Decision Science, School of Health and Related Research, University of Sheffield,2Department of Economics, University of Sheffield, Sheffield,3Department of Economics and Related Studies, University of York, York, and 4Wessex Institute for Health Research and Development, University of Southampton, Southampton, UK
Address correspondence to Dr C. McCabe, Health Economics and Decision Science, School of Health and Related Research, Regent Court, 30 Regent Street, Sheffield S1 4DA. email: c.mccabe@sheffield.ac.uk
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Introduction |
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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.
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Special status considerations |
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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
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Methodological issues concerning evidence on effectiveness |
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Limited budget impact |
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Equity issues |
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Options for policy recommendations |
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Assigning equity weights
Risk sharing and ‘no cure, no pay’ schemes
Clinical and pharmacogenetic criteria
Funding by research councils
Dedicated funding
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Conclusions |
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