title: Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act
creator: Lutz, Thomas
creator: Lampert, Anette
creator: Hoffmann, Georg F.
creator: Ries, Markus
subject: ddc-610
subject: 610 Medical sciences Medicine
description: Background: Rare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity. As a whole group, rare rheumatologic diseases afflict millions of people demanding for effective therapies. Therefore, we analyzed the impact of the US Orphan Drug Act on the development of anti-rheumatic orphan drugs.   Methods: Analysis of the FDA database for orphan drug designations.   Results: In the last three decades, out of 77 orphan drug designations, 14 orphan drug approvals were granted by the FDA for the treatment of rare rheumatologic disorders, i.e. juvenile idiopathic arthritis (N = 5), cryopyrin-associated periodic syndromes (N = 3), uveitis (N = 3), familial Mediterranean fever (N = 1), anti-neutrophil cytoplasmic antibody-associated vasculitis (N = 1), and xerostomia and keratoconjunctivitis sicca in Sjögren’s syndrome (N = 1). Mean time (standard deviation) from designation to approval was 3.9 (2.81) [range 1 … 12] years. Number of FDA-approved small molecules (N = 6, 43 %) and biologics (N = 8, 57 %) was comparable. Almost every fifth (19 %) orphan drug designation was withdrawn. Despite the rarity of conditions, 13/14 pivotal studies were randomized controlled trials.   Conclusions: Orphan drug development is challenging: thirty years of US orphan drug act supported the development and FDA approval of 14 orphan drug programs with anti-rheumatic compounds for six rheumatologic diseases.
publisher: BioMed Central
date: 2016
type: Article
type: info:eu-repo/semantics/article
type: NonPeerReviewed
format: application/pdf
identifier: https://archiv.ub.uni-heidelberg.de/volltextserverhttps://archiv.ub.uni-heidelberg.de/volltextserver/20703/1/13023_2016_Article_443.pdf
identifier: DOI:
identifier: urn:nbn:de:bsz:16-heidok-207031
identifier:   Lutz, Thomas ; Lampert, Anette ; Hoffmann, Georg F. ; Ries, Markus  (2016) Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act.  Orphanet Journal of Rare Diseases, 11 (60).  pp. 1-12.  ISSN 1750-1172     
relation: https://archiv.ub.uni-heidelberg.de/volltextserver/20703/
rights: info:eu-repo/semantics/openAccess
rights: Please see front page of the work (Sorry, Dublin Core plugin does not recognise license id)
language: eng