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Orphan drugs and the NHS: Should we value rarity

Claxton, K. (orcid.org/0000-0003-2002-4694), McCabe, C. and Tsuchiya, A. (2005) Orphan drugs and the NHS: Should we value rarity. BMJ. pp. 1016-1019. ISSN 1756-1833

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Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients. Several countries have put measures in place to safeguard research and development of orphan drugs, but few get close to meeting the cost effectiveness criteria for funding by healthcare providers. We examine the justifications for special status for rare diseases and ask whether the cost effectiveness of drugs for rare or very rare diseases should be treated differently from that of other drugs and interventions.

Item Type: Article
Copyright, Publisher and Additional Information: © 2005 BMJ Publishing Group Ltd
Institution: The University of York
Academic Units: The University of York > Economics and Related Studies (York)
Depositing User: Sherpa Assistant
Date Deposited: 23 Jan 2006
Last Modified: 21 Aug 2016 00:04
Published Version: http://dx.doi.org/10.1136/bmj.331.7523.1016
Status: Published
Refereed: Yes
URI: http://eprints.whiterose.ac.uk/id/eprint/948

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