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IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases

The International Rare Diseases Research Consortium (IRDiRC) has developed a new guidebook to invigorate drug repurposing for rare diseases.
  1. Anneliene Hechtelt Jonker

    1. IRDiRC, Paris, France; University of Twente, Enschede, the Netherlands.

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  2. Simon Day

    1. IRDiRC, Paris, France; CTCT Ltd, North Marston, United Kingdom.

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  3. Michela Gabaldo

    1. IRDiRC, Paris, France; Evotec, Verona, Italy.

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  4. Heather Stone

    1. IRDiRC, Paris, France; US Food and Drug Administration, Silver Spring, MD, United States.

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  5. Martin de Kort

    1. IRDiRC, Paris, France; EATRIS ERIC, Amsterdam, the Netherlands.

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  6. Daniel J. O’Connor

    1. IRDiRC, Paris, France; The Association of the British Pharmaceutical Industry, London, United Kingdom.

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  7. Anna Maria Gerdina Pasmooij

    1. IRDiRC, Paris, France; Dutch Medicines Evaluation Board, Utrecht, The Netherlands.

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Drug repurposing has often been suggested as a key solution for developing more therapies for the estimated 6,000–8,000 rare diseases1. This strategy can be attractive because of its potential to develop therapies in a more efficient and cost-effective way compared to de novo drug discovery approaches, by building on existing knowledge about key characteristics of the candidates for drug repurposing such as their safety profile and pharmacokinetic properties in humans, as well as clinical experience2.

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Nature Reviews Drug Discovery 22, 937-938 (2023)

doi: https://doi.org/10.1038/d41573-023-00168-9

Acknowledgements

This article was written on behalf of the IRDiRC DRG Task Force and Therapies Scientific Committee. The members and contributors beyond the authors were Maria Cavaller Belaubi, Luca Falciola, Christine Fetro, Maria Gogou, Florence Guillot, Virginie Hivert, Peter-Bram ’t Hoen, Sonsoles Hortelano Blanco, Oxana Iliach, Ania Korsunska, Ramaiah Muthyala, Vinciane Pirard, Ken Sakushima, Marco Schmidt and Nivedita Valentine. We would also like to thank Galliano Zanello for his support. The work was supported by the European contract “European Joint Programme on Rare Diseases”(N°825575).

References

  1. Dawkins, H. J. S. et al. Progress in Rare Diseases Research 2010–2016: An IRDiRC Perspective. Clin. Transl. Sci. 11, 11–20 (2018).

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  2. Fang, H. et al. FDALabel for drug repurposing studies and beyond. Nat. Biotechnol. 38, 1378–1379 (2020).

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  3. Austin, C. P., Mount, B. A. & Colvis, C. M. Envisioning an actionable research agenda to facilitate repurposing of off-patent drugs. Nat. Rev. Drug Discov. 20, 723–724 (2021).

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  4. Hechtelt Jonker, A. et al. Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook. Nat. Rev. Drug Discov. 19, 495–496 (2020).

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Supplementary Information

  1. 41573_2023_CM26185128_MOESM1_ESM

Competing Interests

The authors declare no competing interests. The views expressed in this article are the personal views of the author(s) and may not be understood as being made on behalf of their employer(s)/organization(s).

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