To enable the eco-system of research and drug development in rare diseases to achieve IRDiRC goal 2, a quantum change is needed in the way drugs are developed. To enable this change, new practices should become standard elements of the development roadmap. However, in order to fully integrate these new elements within a defined development plan generating better data quality AND shorter development timelines AND lower development costs/ better R&D efficiency, the entire development roadmap should be re-engineered.
The “Orphan Drug Development Guidebook” project aims at creating a simple guidebook for academic and industrial drug developers describing the available tools and initiatives specific for rare disease development and how to best use them.
The guidebook will include fact-sheet descriptions of the new development tools, a series of standard cases of drug development defining “how” and “when” to use these tools and engage in these initiatives, and a series of practical check-lists of things to consider at each.
Task Force Members
The Orphan Drug Development Guidebook Task Force members include:
- Annemieke Aartsma-Rus, Leiden University Medical Center, The Netherlands
- Alessandro Aiuti, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), San Raffaele Scientific Institute, Italy
- Diego Ardigo, Chiesi Pharmaceuticals, Italy (Chair of the Therapies Scientific Committee)
- Simon Day, Clinical Trials Consulting & Training, United Kingdom
- Michela Gabaldo, Fondazione Telethon, Italy
- Marlene Haffner, Haffner Associates, LLC, USA
- Virginie Hivert, EURORDIS-Rare Diseases Europe, France (Vice-Chair of the Therapies Scientific Committee)
- Sangeeta Jethwa, Roche, Switzerland
- Eri Matsuki, Japan Agency for Medical Research and Development, Japan
- Ana Mingorance, Dracaena consulting, Loulou Foundation, Dravet Syndrome Foundation, Norway/Spain
- Thomas Morel, KU Leuven, Belgium
- Daniel O’Connor, MHRA, United Kingdom
- Anne Pariser, NIH NCATS, Office of Rare Diseases Research, USA
- Caridad Pontes, Catalan Health Service, Spain
- Ken Sakushima, Pharmaceuticals and Medical Devices Agency, Japan
- Maurizio Scarpa, Center for Rare Diseases HSK, Germany
- Richard Yang, Reflection Biotechnologies Limited, Hong Kong
Plans and timeline
- June 2018: Internal drafting of material
- October 2018: First teleconference of the Task Force
- November 2018: Second teleconference of the Task Force
- December 2018: Workshop with external experts, Dublin, Ireland
- June 2019: Report and main outputs available
Comments and suggestions
To submit your comments and suggestions on this Task Force, please contact us by completing this form.