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US FDA’s New ‘N of 1’ Guidance Essentially A Down Payment On Critical Advice

 
• By Sarah Karlin-Smith

Draft guidance will help sponsor-investigators working on individualized gene therapies. The focus on basic administrative and procedural aspects of interaction with the FDA leaves many policy and commercialization questions about how these personalized investigational products should be studied unanswered.

Genetic engineering, GMO and Gene manipulation concept. Hand is inserting sequence of DNA. 3D illustration of DNA.

The US Food and Drug Administration’s new draft guidance on investigational new drug application submissions for individualized gene therapies focuses largely on helping sponsors initiate early interaction with the agency and does not address regulatory requirements for clinical trials or commercialization pathways, making the new document a relatively small down payment on FDA’s more than year and a half old promise to quickly put together policy on how to handle applications for personalized medicine.

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