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Gene Therapy Clinical Trials - Observing Subjects for Delayed Adverse Events Guidance for Industry November 2006

Docket Number:
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

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)). 

Submit Comments

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: 2005D-0310.


Contact Point
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
(800) 835-4709
(240) 402-8010