Limits on use of health economic assessments for rare diseases

作者:Hyry H I; Stern A D; Cox T M*; Roos J C P
来源:QJM-An International Journal of Medicine, 2014, 107(3): 241-245.
DOI:10.1093/qjmed/hcu016

摘要

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.

  • 出版日期2014-3