About

The Orphan Drug Development Guidebook is a patient focused guidebook that describes the available tools, incentives, resources and practices specific for developing traditional and innovative drugs/therapies for rare disease indications and how to best use them. It can be used by academic, non-profit organizations, small and larger (innovative) biotechs and patient-driven drug developers. The Guidebook currently has two chapters: the chapter dedicated to orphan drug development (ODDG) focused on small molecules or innovative therapies, and a chapter dedicated to drug repurposing (DRG).
More information can also be found in the papers published on the Orphan Drug Development Guidebook, the Drug Repurposing Guidebook and START checklist.

Acknowledgements

The ODDG was written by the IRDiRC ODDG Task Force Steering group: A. H. Jonker, V. Hivert, M. Gabaldo, L. Batista, D. O’Connor, A. Aartsma-Rus, S. Day, K. Sakushima, D. Ardigo

The Drug Repurposing Guidebook was written by the IRDiRC DRG Task Force Steering Group: A.H. Jonker, S. Day, M. Gabaldo, H. Stone, M. de Kort, D. O’Connor, A.M.G. Pasmooij

TheTherapies Scientific Committee members and contributors beyond the IRDiRC ODDG Task Force Steering group were: A. Aiuti, D. Athanasiou, L. Conklin, S. Cheng, R. Conwit, M. Driessens, M. Haffner, E. Hoffman, S. Jethwa, E. Matsuki, A. Mingorance, T. Morel, A. Pariser, C. Pontes, M. Scarpa and R. Yang. We would also like to thank A.-L. Pham-Hung d’Alexandry d’Orengiani for her support.

The members and contributors beyond the DRG Task Force Steering group were: The members and contributors beyond the authors were M. Cavaller Belaubi, L. Falciola, C. Fetro, M. Gogou, F. Guillot, V. Hivert, P.-B. ’t Hoen, S. Hortelano Blanco, O. Iliach, A. Korsunska, R. Muthyala, V. Pirard, K. Sakushima, M. Schmidt and N. Valentine. We would also like to thank G. Zanello for his support.

The IRDiRC ODDG Web App has been created by Ing. Matteo Manzinello and improved upon by GetYos Webdevelopment

The work was supported by the European contracts “SUPPORT-IRDiRC” (N° 305207) and “European Joint Programme on Rare Diseases”(N°825575).

IRDiRC ODDG Task Force workshop – Dublin, December 12-13, 2018

IRDiRC DRG Task Force workshop – 2023

Credits

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Open Source Acknowledgements

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