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

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to drug repurposing (DR) depending on the status of the repurposable drug/molecule (approved, investigational, withdrawn, shelved), the context, and the stakeholders involved.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.