E6-20129. Guidance for Industry: Gene Therapy Clinical Trials-Observing Subjects for Delayed Adverse Events; Availability

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA) is announcing the availability of a document entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Subjects for Delayed Adverse Events,” dated November 2006. The guidance document provides sponsors of gene therapy studies with recommendations regarding collection of data on delayed adverse events in subjects who have been exposed to investigational gene therapy products. The guidance announced in this notice finalizes the draft guidance entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Participants for Delayed Adverse Events,” dated August 2005, and supplements the recommendations for study subject long-term follow-up in the “Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors” (Retroviral Vector guidance), dated November 2006.

DATES:

Submit written or electronic comments on agency guidances at any time.

ADDRESSES:

Submit written requests for single copies of the guidance to the Office of Communication, Training, and Manufacturers Assistance (HFM-40), Center for Biologics Evaluation and Research (CBER), Food and Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448. Send one self-addressed adhesive label to assist the office in processing your requests. The guidance may also be obtained by mail by calling CBER at 1-800-835-4709 or 301-827-1800. See the SUPPLEMENTARY INFORMATION section for electronic access to the guidance document.

Submit written comments on the guidance to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Submit electronic comments to http://www.fda.gov/​dockets/​ecomments.

FOR FURTHER INFORMATION CONTACT:

Brenda R. Friend, Center for Biologics Evaluation and Research (HFM-17), Food and Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-6210.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a document entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Subjects for Delayed Adverse Events,” dated November 2006. This guidance provides to sponsors of gene therapy studies recommendations on the following: (1) Methods to assess the risk of gene-therapy-related delayed adverse events following exposure to investigational gene therapy products, (2) guidance for determining 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.

In the Federal Register of August 23, 2005 (70 FR 49296), FDA announced the availability of the draft guidance entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Participants for Delayed Adverse Events,” dated August 2005. FDA received numerous comments on the draft guidance and those comments were considered as the guidance was finalized. A summary of changes includes the following: (1) Clarification on topics not included in the guidance; (2) revised recommendations for preclinical study design to assess vector biodistribution and persistence; and (3) revised recommendations for data collection and data reporting in trials involving integrated vectors (e.g., retroviral vectors). The guidance announced in this notice finalizes the draft guidance entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Participants for Delayed Adverse Events,” dated August 2005. This guidance also supplements the recommendations in the Retroviral Vector guidance, dated November 2006, for study subject long-term follow-up.

The guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents FDA's current thinking on this topic. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

This guidance refers to previously approved collections of information found in FDA regulations. These collections of information are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The collections of information in the Investigational New Drug Application (IND) regulations (21 CFR part 312) have been approved under OMB control number 0910-0014; the Good Laboratory Practice regulations (21 CFR part 58) have been approved under OMB control number 0910-0119.

III. Comments

Interested persons maysubmit to the Division of Dockets Management (see ADDRESSES) written or electronic comments regarding the guidance. Submit a single copy of electronic comments or two paper copies of any mailed comments, except that individuals may submit one paper copy. Comments are to be identified with the docket number found in brackets in the heading of this document. A copy of the guidance and received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.

IV. Electronic Access

Persons with access to the Internet may obtain the guidance at either http://www.fda.gov/​cber/​guidelines.htm or http://www.fda.gov/​ohrms/​dockets/​default.htm.

Dated: November 20, 2006.

Jeffrey Shuren,

Assistant Commissioner for Policy.