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FDA Guidance for Industry: Gene Therapy Clinical Trials

This guidance provides sponsors of gene therapy studies, recommendations regarding the design of studies to include the collection of data on delayed adverse events in subjects who have been exposed to investigational gene therapy products. The FDA, provides (1) recommended methods to assess the risk of gene therapy-related delayed adverse events following exposure to investigational gene therapy products, (2) recommended methods to determine the likelihood that long-term follow-up observations on study subjects will provide scientifically meaningful information, and (3) specific advice regarding the duration and design of long-term follow-up observations.

Published: 2006
Format: PDF
Length: 25 pages
Type: White paper
Language: English
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