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Opportunity cost of funding drugs for rare diseases: the cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria.

Authors
  • Coyle, Doug1
  • Cheung, Matthew C2
  • Evans, Gerald A3
  • 1 Department of Epidemiology and Community Medicine, University of Ottawa, Ottawa, Canada (DC) , (Canada)
  • 2 Division of Hematology/Oncology, Sunnybrook Health Sciences Centre, Odette Cancer Center, University of Toronto, Toronto, ON, Canada (MCC) , (Canada)
  • 3 Division of Infectious Diseases, Department of Medicine, Kingston General Hospital and Queen's University, Kingston, ON, Canada (GAE). , (Canada)
Type
Published Article
Journal
Medical decision making : an international journal of the Society for Medical Decision Making
Publication Date
Nov 01, 2014
Volume
34
Issue
8
Pages
1016–1029
Identifiers
DOI: 10.1177/0272989X14539731
PMID: 24990825
Source
Medline
Keywords
License
Unknown

Abstract

This study demonstrates the feasibility of conducting economic evaluations in the context of rare diseases. Eculizumab may provide substantive benefits to patients with PNH in terms of life expectancy and quality of life but at a high incremental cost and a substantial opportunity cost. Decision makers should fully consider the opportunity costs before making positive reimbursement decisions.

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