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Zanello et al. Rare Dis Orphan Drugs J 2023;2:9 https://dx.doi.org/10.20517/rdodj.2023.04 Page 11 of 15

It also clarifies the scope of repurposing: “It includes new therapeutic uses for existing medicines, different
formulations of the same medicine, and/or creating new combinations of medicines or medicines with medical
devices. Repurposing of medicines is part of the routine research portfolio of both the pharmaceutical industry
and academic institutions in the search for solutions for those conditions with unmet medical needs including
aspects related to sustainability and patient access”. This is very much aligned with the findings from this
Task Force, although few considerations have been given so far to a combination of medicines or medicines
with medical devices and should be included in further steps.

The second aspect that should be disentangled is the shadow put on repurposing based on pricing
considerations. The authors of this paper believe that this discussion is not specific to repurposing and
relates to considerations around value-based assessment versus willingness to pay, and as such, this should
not hamper the opportunities offered by repurposing.

Thirdly, it came very strongly from the interviews that neither the benefits brought by repurposing nor the
pitfalls that come with it should be underestimated. Despite some aspects of the therapeutic development
path being easier than a development starting from scratch, careful consideration should be given to all
[23]
items identified in the START checklist of the Orphan Drug Development Guidebook : (a) mapping of the
stakeholders; (b) analysis of the available knowledge already generated on the disease, the product and the
targeted patient population; (c) resources planning in order to anticipate the gaps, the additional data to be
generated, the regulatory and market access requirements; and (d) the target patient value profile to always
keep the unmet needs of patients in focus.

What is needed for sustainable approaches in drug repurposing in terms of funding orientation?
Over the years, IRDiRC funding member organizations have often been referring to the Consortium
recommendations when setting up their work program and calls for funding. Notably, the Horizon Europe
work program from the European Commission has proposed HORIZON-HLTH-2022-DISEASE-06-04-
two-stage: Development of new effective therapies for rare diseases & HORIZON-HLTH-2021-DISEASE-04-
02: Building a European innovation platform for the repurposing of medicinal products which are fully
[21]
[26]
aligned with the roadmap of the IRDiRC Therapies Scientific Committee . The latter provides provision
for two consortia [19,20] that are aiming to contribute to the goal presented in this article.

Funding schemes are of utmost importance to allow for a true collaborative partnership to be formed [27-29] .
In turn, public-private partnerships are crucial to help cross the so-called valley of death of therapeutic
development, which exists for all types of diseases and therapeutic approaches, and which is maybe even
more visible in the case of repurposing approaches in the rare disease space.

Funding is not only needed to help progress individual repurposing approaches; there is also a need for
funding of pre-competitive activities in order to build infrastructures, foster common knowledge generation
(e.g., validate rare disease clinical trial endpoints) and enable sharing of existing and evolving data and
information. One avenue for additional investment that came out from the interviews is the lack of accurate
figures of prevalence for all rare diseases and in all geographic regions. These figures are not only crucial in
the development space of medicines, but also for diagnosis, care and socio-economic planning.

What is needed for sustainable approaches in drug repurposing in terms of implementation?
Access to data generated by previous researchers and developers has been clearly described as a major
difficulty during the interviews. The publication of failed or non-conclusive clinical trials would be utterly
helpful in the case of repurposing and should be encouraged, as well as the sharing of information amongst