doi: 10.1093/qjmed/hcu016.
Epub 2014 Jan 22.
Limits on use of health economic assessments for rare diseases
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- PMID: 24453281
- DOI: 10.1093/qjmed/hcu016
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Limits on use of health economic assessments for rare diseases
QJM.
2014 Mar.
Abstract
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.
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