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Added value of a collaborative approach in drug repurposing
From the interviews that we have conducted, there are three main patterns that emerge when it comes to
funding a repurposing approach.

Firstly, when the project is initiated by a pharmaceutical company, the funding is primarily ensured by the
company’s own funding, with the addition of private funding such as capital risks investors on occasion.

Secondly, when the project is initiated by an academic team (researchers and/or clinicians), the project is
mostly fuelled by public funding (e.g., European Union/National funding, E-Rare calls, United States federal
grants, NIH grants). In that case, it can be complemented by funding from patient organizations and other
not-for-profit entities, such as foundations. Additional support can be sought from pharmaceutical
companies to supply the drug in clinical trials at no cost, for instance.

The third avenue which breaks the silos existing in the two previously described situations, and that the
authors would encourage, is a public-private partnership, as we know that often the discovery phase is done
by the public sector while the development phase is performed by the private sector. To make such a public-
private approach successful, the expectations of each party and the respective contributions have to be
clarified at the beginning of the collaboration, including the signature of contracts and legal agreements.

To make the link with the above item (“As for every therapeutic development, patients should be
considered as equal partners”), the involvement and, to a larger extent, a true patient-led approach seem to
be critical to ensure a multi-stakeholder repurposing program. The development of a framework for
funding and risk in collaborative projects when there are non-commercial opportunities (patent expired)
and where private partners would put the responsibility to patients above profit-motive could address some
of the needs for sustainable approaches in drug repurposing. As an example, the two Consortia REPO4EU
and REMEDI4ALL [19,20] selected following the call by the European Commission under the Horizon Europe
program might bring a piece of the answer here.
[21]

Learning lessons from failures and building on past findings
The trigger for a repurposing approach can be anything from scientific discovery, data mining or artificial
intelligence (AI), clinical observation, off-label use or extensive literature evidence.

It was reported almost unanimously that the collection of scientific evidence in order to support a
repurposing approach is a major obstacle. Early detection of the potential for repurposing can represent an
advantage and sometimes a competitive one. However, gathering knowledge from past non-conclusive
clinical studies in order to inform the new development is not always that easy (e.g., lack of responsiveness
of previous investigators, failed clinical trials not published). When it has happened well, it has been highly
beneficial.

According to several of the interviewees, a lot of emphases have to be put on the publication of failed
clinical trials, the importance of collaboration between the research teams (e.g., the possibility of contacting
the initial investigators), and the importance of data sharing which is often identified as one of the main
challenges (e.g., non-clinical development information for the product that might support dosing strategies
for the repurposed indication).