Limits on use of health economic assessments for rare diseases.
Funding 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.
Department of Medicine, University of Cambridge, Addenbrooke's Hospital, Hills Road, Cambridge CB2 0QQ, UK. ec432@medschl.cam.ac.uk.
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D002017:Budgets; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D005062:European Union / Q000191:economics; D005102:Health Expenditures; D006801:Humans; D020380:Needs Assessment / Q000191:economics; D009965:Orphan Drug Production / Q000191:economics*; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D035583:Rare Diseases / Q000188:drug therapy / Q000191:economics*