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GUIDANCE DOCUMENT

Long Term Follow-up After Administration of Human Gene Therapy Products Draft Guidance for Industry July 2018

Draft

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Long Term Follow-up After Administration of Human Gene Therapy Products

Docket Number:
FDA-2018-D-2173
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

We, FDA, are providing you, a sponsor who is developing a human gene therapy (GT) product, recommendations regarding the design of long term follow-up observational studies (LTFU observations) for the collection of data on delayed adverse events following administration of a GT product. Often, GT products are designed to achieve therapeutic effect through permanent or long-acting changes in the human body. As a result of long term exposure to an investigational GT product, study subjects may be at increased risk of undesirable and unpredictable outcomes which may present as delayed adverse event(s). To understand and mitigate the risk of a delayed adverse event, subjects in gene therapy trials may be monitored for an extended period of time, which is commonly referred to as the “long term follow-up” (LTFU) period (of a clinical study). LTFU observations are extended assessments that continue some of the scheduled observations of a clinical trial past the active follow-up period, and are an integral portion of the study of some investigational GT products. LTFU observations are important to monitor long term safety of GT products. For GT products that present long term risks to subjects, LTFU/surveillance plan(s) should also be put in place post-licensure for monitoring of delayed adverse events (for details we refer you to section VI. of this document). Not all GT products will require LTFU observations; a risk assessment is performed by a sponsor based on several factors as outlined in this guidance.


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:

Division of Dockets Management (HFA- 305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2018-D-2173.

Questions?

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
ocod@fda.hhs.gov
(800) 835-4709
(240) 402-8010