1.
Science
; 355(6330): 1131-1133, 2017 Mar 17.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28302813
2.
QJM
; 107(3): 241-5, 2014 Mar.
Artigo
em Inglês
| MEDLINE
| ID: mdl-24453281
RESUMO
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.