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Fetro. Rare Dis Orphan Drugs J 2023;2:7 Rare Disease and
DOI: 10.20517/rdodj.2023.06
Orphan Drugs Journal

Perspective Open Access

Connecting academia and industry for innovative
drug repurposing in rare diseases: it is worth a try

Christine Fetro

Foundation for Rare Diseases, Paris 75014, France.
Correspondence to: Dr. Christine Fetro, PharmD, MSc, Foundation for Rare Diseases, Paris 75014, France. E-mail:
christine.fetro@fondation-maladiesrares.com

How to cite this article: Fetro C. Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a
try. Rare Dis Orphan Drugs J 2023;2:7. https://dx.doi.org/10.20517/rdodj.2023.06

Received: 30 Jan 2023 First Decision: 8 Mar 2023 Revised: 22 Mar 2023 Accepted: 24 Mar 2023 Published: 10 Apr 2023

Academic Editors: Daniel Scherman, Marc Dooms Copy Editor: Ying Han Production Editor: Ying Han

Abstract
There are different approaches to drug repurposing (DR) depending on the status of the repurposable
drug/molecule (approved, investigational, withdrawn, shelved), the context, and the stakeholders involved. The
purpose of this perspective paper is to highlight the complexity of academia-industry collaborations in DR for rare
diseases and go beyond stereotypes to consider realistic and mutually reinforcing cooperation among various
stakeholders, including not only academia and industry but also regulators, legal experts, and payers, leading to
benefits for patients with unmet medical needs. Key questions are addressed through the presentation of select DR
case studies. Some ongoing and promising European and international initiatives are introduced and some
recommendations are proposed.

Keywords: Drug repurposing, academia-industry collaboration, rare diseases, sildenafil, alpelisib, propranolol

INTRODUCTION
Drug repurposing (DR) can be defined as the process of identification of new uses for approved or
investigational drugs that are outside the scope of the original medical indication. It is considered to have
significant advantages over developing an entirely new drug: fewer risks, lower costs, and shorter timelines.
Public-specific programs promote DR initiatives while pharmaceutical companies integrate them as a
strategy in the life cycle management of their products. Indeed, DR represents a relevant opportunity to find

© 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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