Orphan drugs for rare diseases: grounds for special status

Drug Development Research, 05/03/2012

Authors provide an overview of how the special status of orphan drugs can be put into practice with respect to registration, pricing and reimbursement. Adopting this special status in practice is possible, but not straightforward. Hence authors propose a number of policy recommendations, such as risk sharing arrangements, to take account of the special status of orphan drugs.

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