Guidance for Industry: Gene Therapy Clinical Trials
Observing Subjects for Delayed Adverse Events
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Biologics Evaluation and Research
November 2006
https://www.ngvbcc.org/pdf/gtclin.pdf;jsessionid
"This guidance provides to you, 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. We, FDA, are providing: (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.1 When a gene therapy clinical trial presents long-term risks to human subjects, a gene therapy clinical trial must provide for long-term follow-up observations in order to mitigate those risks. Without such long-term follow-up observations, the study would expose the subjects to an unreasonable and significant risk of illness or injury (21 Code of Federal Regulations (CFR) 312.42(b)(1)(i) and (b)(2)(i))."
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[BioNTech (Pfizer) SEC filing] [Moderna SEC filing]:
"Currently, mRNA is considered a gene therapy product by the FDA.”
Observing Subjects for Delayed Adverse Events
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Biologics Evaluation and Research
November 2006
https://www.ngvbcc.org/pdf/gtclin.pdf;jsessionid
"This guidance provides to you, 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. We, FDA, are providing: (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.1 When a gene therapy clinical trial presents long-term risks to human subjects, a gene therapy clinical trial must provide for long-term follow-up observations in order to mitigate those risks. Without such long-term follow-up observations, the study would expose the subjects to an unreasonable and significant risk of illness or injury (21 Code of Federal Regulations (CFR) 312.42(b)(1)(i) and (b)(2)(i))."
-
[BioNTech (Pfizer) SEC filing] [Moderna SEC filing]:
"Currently, mRNA is considered a gene therapy product by the FDA.”
Study C4591022, entitled “Pfizer-BioNTech COVID-19 Vaccine Exposure during Pregnancy: A Non-Interventional Post-Approval Safety Study of Pregnancy and Infant Outcomes in the Organization of Teratology Information Specialists
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