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“Draft Guidance On Clinical Recommendations For “N of 1” Gene Therapies”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?