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“Draft Guidance On Clinical Recommendations For “N of 1” Gene Therapies”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
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Thank you Joanna for the link, that is very helpful
Dear all,
I am new to the forum and would like to inform that one of the co-authors of this publication is Annemieke Aartsma-Rus. A few years back she has founded the Dutch Center for RNA Therapeutics which aims to develop ASO therapies for very rare genetic mutations in patients (N=1) with a brain or eye disorder. LUMC, Radboudumc and ErasmusMC are equal partners in the DCRT, soon to be joined by UMCU and UMCG. The DCRT is also working closely with international initiatives such as the already mentioned N=1 Collaborative and 1Mutation1Medicine. Please check our website for more information about our work or contact us if you need advice or help with N=1 cases or therapy development.
Best regards,
Pauline de Graaf
DCRT project manager
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