Value Health. 2014 Dec;17(8):757-61. doi: 10.1016/j.jval.2014.08.2672. Epub 2014 Oct 18.
Toward a functional definition of a "rare disease" for regulatory authorities and funding agencies.
RESULTS AND CONCLUSIONS:
Copyright © 2014 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.
drug development; drug reimbursement; orphan drugs; rare disease
- [PubMed - in process]