Enrichment designed clinical trials are not unique to gene therapy modality! It started way back in oncology targeted therapy approvals and were also approved using accelerated pathway for unmet medical need indications!
Original Message:
Sent: 09-Feb-2024 16:37
From: Stephanie Markey
Subject: Enrichment Designs
thanks for the reply. I was reading through the biologics guidance documents this summer to gain a better understanding of these products. I have not worked in regulatory on a gene-therapy drug myself or specifically used the guidance about enrichment strategies in a trial yet. It seems like a lot of the topics proposed in the guidance could be incorporated into an AI plan for selecting patients.
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Stephanie Markey
Associate Director
Fraser CO
United States
Original Message:
Sent: 07-Feb-2024 15:38
From: Andrew Hadd
Subject: Enrichment Designs
Stephanie,
I know you didn't receive a reply on the thread and request for enrichment study designs, but would you be willing to share what else you learned or the approach you took? Cheers, Andy Hadd
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Andrew Hadd
Director of Regulatory Affairs
Natera
Austin TX
United States
Original Message:
Sent: 22-Jun-2023 19:28
From: Stephanie Markey
Subject: Enrichment Designs
FDA mentions the use of enrichment designs in gene therapy neurodegenerative trials. Has anyone used an enrichment design or can provide more information on this topic?
It is from the FDA guidance: Human Gene Therapy for Neurodegenerative Diseases, https://www.fda.gov/media/144886/download
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Stephanie Markey
Associate Director
Fraser CO
United States
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