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Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le mardi 31 mars 2020
Doi : 10.1016/j.therap.2020.02.006
Received : 15 September 2019 ;  accepted : 15 October 2019
Drug repurposing in rare diseases: Myths and reality

Christine Fetro a, , Daniel Scherman a, b, c, d
a French Foundation for rare diseases, 75014 Paris, France 
b Chemical and biological technologies for health unit (UTCBS), CNRS UMR8258, 75006 Paris, France 
c UTCBS, Inserm U1267, 75006 Paris, France 
d Faculté de pharmacie, université Paris Descartes, université de Paris, 75006 Paris, France 

Corresponding author. French Foundation for rare diseases, 96, rue Didot, 75014 Paris, France.French Foundation for rare diseases96, rue DidotParis75014France

While nearly 8000 rare diseases have been identified, only 5 percent have licensed treatments. As most of these diseases are life threatening, it underscores the urgent need for new drugs. Drug repurposing (also called drug repositioning) consists in identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication. It represents an opportunity for rare diseases and patients with unmet needs. It is an alternative option in drug development and is often presented as being a viable, risk-managed strategy for pharmaceutical companies developing orphan drugs. Drug repurposing is presented as offering various advantages over developing an entirely new drug for a given indication: fewer risks, lower costs and shorter timelines. However, matters are not as simple as this. There are notable successes for drug repurposing. Nevertheless, repurposing does not always succeed. The repurposed drug may fail to demonstrate a benefits-harms balance in clinical trials. Moreover, there are legal and regulatory issues which are specific barriers to drug repurposing and which have to be carefully analyzed before any development of repurposed drugs. The objective of this article is to identify major challenges and opportunities of drug repurposing in rare diseases and to separate fact from fiction.

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Keywords : Drug repurposing, Drug repositioning, Drug development risks, Rare diseases

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