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Page 14 of 16 Fetro. Rare Dis Orphan Drugs J 2023;2:7 https://dx.doi.org/10.20517/rdodj.2023.06
CONCLUSION
DR represents a real opportunity and a cheaper and faster way to find and develop new treatments for rare
diseases. But DR is also associated with misconceptions and specific barriers (IP, regulatory, market access,
and lack of incentives). It makes no sense to stereotype public-private partnerships and set one community
against the other. Without research, there will not be any innovation; without regulatory and commercial
development, there will not be any drug on the market. Although being far away from each other in terms
of culture, constraints, and objectives, academia and industry can join forces and build off each other’s
strengths to advance research and development of repurposed drugs for patients with rare diseases. This
approach has already proved successful in several instances, but its potential is far from being fully
exploited. Some fruitful collaborations have resulted in success stories that are widely publicized, but they
do not fully reflect reality. The success rates of academia-industry collaboration vary depending on various
factors (IP rights of the drug candidate, business strategy, Proof of Concept robustness, regulatory
challenges, commercial aspects, and type of pharmaceutical company). Academic drug (re)discovery is a
vital component, but the lack of knowledge of industry standards can make academics uncomfortable.
Educational programs and guidelines could help them better understand industrial constraints and
anticipate and adjust accordingly. There is also a need for new and sustainable DR business models
involving not only academia and industry but also patient associations, regulators and payers to equitably
address these questions. Some current European and international initiatives such as the pilot project from
EMA and STAMP expert group, the call for proposal “Building a European innovation platform for the
repurposing of medicinal products” within Horizon Europe or the IRDiRC Task Force on DR guidebook
should encourage and stimulate new opportunities.

DECLARATIONS
Authors’ contributions
The author contributed solely to the article.

Availability of data and materials
Not applicable.

Financial support and sponsorship
None.

Conflicts of interest
The author declared that there are no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Author(s) 2023.

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