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Zanello et al. Rare Dis Orphan Drugs J 2023;2:9 Rare Disease and
DOI: 10.20517/rdodj.2023.04
Orphan Drugs Journal

Perspective Open Access

Sustainable approaches for drug repurposing in rare
diseases: recommendations from the IRDiRC Task

Force

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Galliano Zanello , Diego Ardigò , Florence Guillot , Anneliene H. Jonker , Oxana Iliach , Hervé Nabarette ,
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Daniel O’Connor , Virginie Hivert 8
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IRDiRC Scientific Secretariat, National Institute of Health and Medical Research(Inserm), Paris 75013, France.
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Rare Diseases, Chiesi Farmaceutici S.p.A, Parma 43122, Italy.
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Agence Nationale de la Recherche, Paris 75013, France.
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TechMed Centre, University of Twente, Enschede 7522 NH, The Netherlands.
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Canadian Organization for Rare Disorders, Toronto, ON M5S 1S4, Canada.
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AFM-Téléthon, Évry-Courcouronnes 91002, France.
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Medicines and Healthcare products Regulatory Agency (MHRA), London E14 4PU, UK.
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EURORDIS - Rare Diseases Europe, Paris 75014, France.
Correspondence to: Dr. Virginie Hivert, EURORDIS - Rare Diseases Europe, Rare Diseases Platform, 96 rue Didot, Paris 75014,
France. E-mail: virginie.hivert@eurordis.org
How to cite this article: Zanello G, Ardigò D, Guillot F, Jonker AH, Iliach O, Nabarette H, O’Connor D, Hivert V. Sustainable
approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force. Rare Dis Orphan Drugs J
2023;2:9. https://dx.doi.org/10.20517/rdodj.2023.04
Received: 19 Jan 2023 First Decision: 7 Mar 2023 Revised: 24 Mar 2023 Accepted: 30 Mar 2023 Published: 25 Apr 2023
Academic Editor: Daniel Scherman Copy Editor: Ying Han Production Editor: Ying Han
Abstract
Drug repurposing represents a real opportunity to address unmet needs and improve the lives of rare disease
patients. It is often presented as a faster, safer and cheaper path for bringing drugs into new indications. However,
several economic, regulatory and scientific barriers can impede the successful repurposing of drugs for rare
diseases. The International Rare Diseases Research Consortium (IRDiRC) set up the Task Force on Sustainable
Models in Drug Repurposing with the objective of identifying key factors for achieving sustainable repurposing
approaches in rare diseases.

In order to help inform expert opinion, the Task Force investigated six cases of medicinal products repurposed into
new rare indications and four cases of ongoing development programs. A questionnaire addressing the major steps
of the repurposing approach was developed by the Task Force and sent to contact points of the organizations. In
addition, interviews were conducted with the relevant organization representatives to conduct a deeper dive into

© The Author(s) 2023. Open Access This article is licensed under a Creative Commons Attribution 4.0
International License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, sharing,
adaptation, distribution and reproduction in any medium or format, for any purpose, even commercially, as
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