Orphan drug clinical development

被引：19

作者：

Blin, Olivier ^{[1
,2
,4
]}

Lefebvre, Marie-Noelle ^{[1
,2
,4
]}

Rascol, Olivier ^{[3
]}

Micallef, Joelle ^{[1
,4
]}

机构：

[1] Aix Marseille Univ, AP HM, Serv Pharmacol Clin & Pharmacovigilance, INSERM,Inst Neurosci Syst, F-13005 Marseille, France

[2] INSERM, Orphandev FCRIN, F-13005 Marseille, France

[3] Univ Toulouse UPS, Serv Pharmacol Clin & Neurosci, Ctr Invest Clin CIC 1436, CHU Toulouse,INSERM,NS Pk FCRIN Network,NeuroToul, F-31000 Toulouse, France

[4] CHU Timone, Serv Pharmacol Clin, 254 Rue St Pierre, F-13005 Marseille, France

来源：

THERAPIE | 2020年 / 75卷 / 02期

关键词：

Rare diseases; Orphan drugs; Market authorization; Clinical trial; Patient; SUCCESS RATES;

D O I：

10.1016/j.therap.2020.02.004

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

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. (C) 2020 Published by Elsevier Masson SAS on behalf of Societe francaise de pharmacologie et de therapeutique.

引用

页码：141 / 147

页数：7

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