Publications
Publications
- March 2014
- QJM: An International Journal of Medicine
Limits on Use of Health Economic Assessments for Rare Diseases
By: Hanna I. Hyry, Ariel Dora Stern, Jonathan CP Roos and Timothy M. Cox
Abstract
Funding of expensive treatments for rare ('orphan') diseases is contentious. These agents fare poorly on 'efficiency' or health economic measures, such as the QALY, 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.
Keywords
Citation
Hyry, Hanna I., Ariel Dora Stern, Jonathan CP Roos, and Timothy M. Cox. "Limits on Use of Health Economic Assessments for Rare Diseases." hcu016. QJM: An International Journal of Medicine 107, no. 3 (March 2014): 241–245.