[Federal Register Volume 71, Number 228 (Tuesday, November 28, 2006)]
[Notices]
[Pages 68821-68822]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E6-20129]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2005D-0310]


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

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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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notice finalizes the draft guidance entitled ``Guidance for Industry: 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).

[[Page 68822]]

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.
[FR Doc. E6-20129 Filed 11-27-06; 8:45 am]
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