Orphan drug clinical development.

Clinical Trials as Topic Cost-Benefit Analysis Drug Approval Humans Orphan Drug Production / economics Rare Diseases / drug therapy
Clinical trial Market authorization Orphan drugs Patient Rare diseases

Journal

Therapie
ISSN: 1958-5578
Titre abrégé: Therapie
Pays: France
ID NLM: 0420544

Informations de publication

Date de publication:
Apr 2020
Historique:
received: 16 09 2019
accepted: 15 10 2019
pubmed: 6 4 2020
medline: 20 2 2021
entrez: 6 4 2020
Statut: ppublish

Résumé

Clinical development for orphan drugs is extremely demanding but fascinating. There is no single aspect that is really specific to it but instead it gathers most of the hurdles: design, outcomes, recruitment, ethics, cost, probability and predictability for success. To overcome these difficulties, there has to be a great collaboration between academic centers, small and large pharma companies, patients' representatives as well as health authorities to provide support and innovative approaches. The ultimate goal is to give access to patients with unmet medical needs to drugs with a favorable benefit-risk ratio. We review and discuss here the pillars for a successful clinical development for orphan drugs.

Identifiants

DOI: 10.1016/j.therap.2020.02.004 PMID: 32247678
pubmed: 32247678
pii: S0040-5957(20)30009-3
doi: 10.1016/j.therap.2020.02.004
pii:
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

141-147

Informations de copyright

Copyright © 2020. Published by Elsevier Masson SAS.

Auteurs

Olivier Blin (O)

Service de pharmacologie clinique et pharmacovigilance, Aix Marseille Univ, AP-HM, INSERM, Inst Neurosci Syst, 13005 Marseille, France; Orphandev-FCRIN, INSERM, 13005 Marseille, France. Electronic address: olivier.blin@ap-hm.fr.

Marie-Noelle Lefebvre (MN)

Service de pharmacologie clinique et pharmacovigilance, Aix Marseille Univ, AP-HM, INSERM, Inst Neurosci Syst, 13005 Marseille, France; Orphandev-FCRIN, INSERM, 13005 Marseille, France.

Olivier Rascol (O)

Services de pharmacologie clinique et neurosciences, centre d'investigation clinique CIC 1436, NS-Park/FCRIN network, NeuroToul COEN center, université de Toulouse UPS, CHU de Toulouse, INSERM, 31000 Toulouse, France.

Joëlle Micallef (J)

Service de pharmacologie clinique et pharmacovigilance, Aix Marseille Univ, AP-HM, INSERM, Inst Neurosci Syst, 13005 Marseille, France.

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Classifications MeSH

questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Orphan drug clinical development.
questionsmedicales.fr › Organisations et économie des soins de santé › Économie › Coûts et analyse des coûts › Analyse coût-bénéfice › Orphan drug clinical development.
questionsmedicales.fr › Sciences sociales › Sociologie › Agrément de médicaments › Orphan drug clinical development.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Orphan drug clinical development.
questionsmedicales.fr › Technologie, industrie et agriculture › Industrie › Secteur secondaire › Industrie pharmaceutique › Médicament orphelin › Orphan drug clinical development.
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Caractéristiques de la maladie › Maladies rares › Orphan drug clinical development.