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- H I Hyry, A D Stern, T M Cox, and J C P Roos.
- Department of Medicine, University of Cambridge, Addenbrooke's Hospital, Hills Road, Cambridge CB2 0QQ, UK. ec432@medschl.cam.ac.uk.
- QJM. 2014 Mar 1; 107 (3): 241-5.
AbstractFunding of expensive treatments for rare (orphan) diseases is contentious. These agents fare poorly on 'efficiency' or health economic measures, such as the quality-adjusted life years, because of high cost and frequently poor gains in quality of life and survival. We show that cost-effectiveness assessments are flawed, and have only a limited role to play in reimbursement decisions for orphan drugs and beyond.
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