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
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.
|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:||12 Feb 2017 01:20|