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

收藏

页码：141 / 147

页数：7

相关论文

共 23 条

[11] The ultimate model organism: progress in experimental medicine [J].

Littman, BH ;

Williams, SA .

NATURE REVIEWS DRUG DISCOVERY, 2005, 4 (08) :631-638

[12]

Lu B, 2012, CLIN INVESTIG, V2, P663

[13] Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs [J].

Maeda, Kojiro ;

Kaneko, Masayuki ;

Narukawa, Mamoru ;

Arato, Teruyo .

ORPHANET JOURNAL OF RARE DISEASES, 2017, 12

[14] Post-approval Studies for Rare Disease Treatments and Orphan Drugs [J].

Maier, William C. ;

Christensen, Ronald A. ;

Anderson, Patricia .

RARE DISEASES EPIDEMIOLOGY: UPDATE AND OVERVIEW, 2ND EDITION, 2017, 1031 :197-205

[15] Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases [J].

Micallef, Joelle ;

Blin, Olivier .

THERAPIE, 2020, 75 (02) :133-139

[16] Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology [J].

Micallef, Joelle ;

Boutouyrie, Pierre ;

Blin, Olivier .

FUNDAMENTAL & CLINICAL PHARMACOLOGY, 2017, 31 (06) :685-694

[17] Effect of ascorbic acid in patients with Charcot-Marie-Tooth disease type 1A: a multicentre, randornised, double-blind, placebo-control led trial [J].

Micallef, Joelle ;

Attarian, Shahram ;

Dubourg, Odile ;

Gonnaud, Pierre-Marie ;

Hogrel, Jean-Yves ;

Stojkovic, Tanya ;

Bernard, Rafaelle ;

Jouve, Elisabeth ;

Pitel, Severine ;

Vacherot, Francois ;

Remec, Jean-Francois ;

Jomir, Laurent ;

Azabou, Eric ;

Al-Moussawi, Mahmoud ;

Lefebvre, Marie-Noelle ;

Attolini, Laurence ;

Yaici, Sadek ;

Tanesse, Daniel ;

Fontes, Michel ;

Pouget, Jean ;

Blin, Olivier .

LANCET NEUROLOGY, 2009, 8 (12) :1103-1110

[18] Applicability and added value of novel methods to improve drug development in rare diseases [J].

Mitroiu, Marian ;

Oude Rengerink, Katrien ;

Pontes, Caridad ;

Sancho, Aranzazu ;

Vives, Roser ;

Pesiou, Stella ;

Manuel Fontanet, Juan ;

Torres, Ferran ;

Nikolakopoulos, Stavros ;

Pateras, Konstantinos ;

Rosenkranz, Gerd ;

Posch, Martin ;

Urach, Susanne ;

Ristl, Robin ;

Koch, Armin ;

Loukia, Spineli ;

van der Lee, Johanna H. ;

Roes, Kit C. B. .

ORPHANET JOURNAL OF RARE DISEASES, 2018, 13

[19] Estimated Costs of Pivotal Trials for Novel Therapeutic Agents Approved by the US Food and Drug Administration, 2015-2016 [J].

Moore, Thomas J. ;

Zhang, Hanzhe ;

Anderson, Gerard ;

Alexander, G. Caleb .

JAMA INTERNAL MEDICINE, 2018, 178 (11) :1451-1457

[20] Impact of a five-dimensional framework on R&D productivity at AstraZeneca [J].

Morgan, Paul ;

Brown, Dean G. ;

Lennard, Simon ;

Anderton, Mark J. ;

Barrett, J. Carl ;

Eriksson, Ulf ;

Fidock, Mark ;

Hamren, Bengt ;

Johnson, Anthony ;

March, Ruth E. ;

Matcham, James ;

Mettetal, Jerome ;

Nicholls, David J. ;

Platz, Stefan ;

Rees, Steve ;

Snowden, Michael A. ;

Pangalos, Menelas N. .

NATURE REVIEWS DRUG DISCOVERY, 2018, 17 (03) :167-+