How is the development of drugs for an individual or small group of patients different from traditional drug development? Is there a guidebook available?
Hi Marlos, FDA had published some guidance on N-of-1 therapy:
“Draft Guidance On Clinical Recommendations For “N of 1” Gene Therapies”
Zie link:
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@MBouma On your profile I see that you work at Radboud Therapy Accelerator for Rare Diseases. Do you have experience in the field of developing therapies for small groups of patients? Could you give some additional information about this topic?
Hi Frank and Marga @MBouma
Are you familiar with the N=1 Collaborative? If yes, can you give guidance how this initiative could be of added value for innovators in the Netherlands?
Maybe @Joanna from the Therapy Accelerator could help?
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Hi Marloes,
There is indeed a guidebook on drug development for small populations: The Orphan Drug Development Guidebook (ODDG), from IRDiRC.
See https://orphandrugguide.org
There is also a similar guidebook for drug repurposing:
https://irdirc.org/drug-repurposing-guidebook/
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Hi Benien,
I do know the initiative, but I cannot directly give guidance.
I know that Anneliene Jonker from University of Twente has written an article that might be of help:
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap
Thank you Joanna for the link, that is very helpful