What Makes an Orphan Drug?
Overview
Authors
Affiliations
Debates over orphan drug pricing: a meta-narrative literature review.
Hanchard M Orphanet J Rare Dis. 2025; 20(1):107.
PMID: 40055799 PMC: 11887186. DOI: 10.1186/s13023-025-03634-2.
Vokinger K, Kesselheim A BMJ Open. 2019; 9(10):e028634.
PMID: 31601584 PMC: 6797305. DOI: 10.1136/bmjopen-2018-028634.
Analysis of Health Technology Assessments of Orphan Drugs in Ireland from 2012 to 2017.
Usher C, McCullagh L, Tilson L, Barry M Pharmacoecon Open. 2019; 3(4):583-589.
PMID: 31073976 PMC: 6861405. DOI: 10.1007/s41669-019-0136-1.
[Rare diseases: laying out some challenges].
Cambios Rev Med. 2018; 16(1):8-9.
PMID: 29708234 PMC: 5920522.
Effectiveness, safety and costs of orphan drugs: an evidence-based review.
Onakpoya I, Spencer E, Thompson M, Heneghan C BMJ Open. 2015; 5(6):e007199.
PMID: 26109112 PMC: 4480037. DOI: 10.1136/bmjopen-2014-007199.