[Federal Register Volume 70, Number 162 (Tuesday, August 23, 2005)]
[Notices]
[Pages 49296-49297]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 05-16629]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2005D-0310]


Draft Guidance for Industry on Gene Therapy Clinical Trials--
Observing Participants 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 draft document entitled ``Guidance for Industry: Gene 
Therapy Clinical Trials--Observing Participants for Delayed Adverse 
Events,'' dated August 2005. The draft guidance provides sponsors of 
gene therapy studies with recommendations regarding collection of data 
on delayed adverse events in participants who have been exposed to gene 
therapy products. When finalized, this guidance will supplement the 
recommendations 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 October 2000, for study participant long-term 
followup. However, the recommendations in the Retroviral Vector 
guidance regarding the length of followup will be superseded by this 
Gene Therapy Clinical Trials guidance.

DATES: Submit written or electronic comments on the draft guidance by 
November 21, 2005, to ensure their

[[Page 49297]]

adequate consideration in preparation of the final guidance. General 
comments on agency guidance documents are welcome at any time.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Office of Communication, Training, and Manufacturers 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 The draft guidance may 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to the draft guidance document.
    Submit written comments on the draft guidance to the Division of 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 draft document entitled 
``Guidance for Industry: Gene Therapy Clinical Trials--Observing 
Participants for Delayed Adverse Events'' dated August 2005. This draft 
guidance provides to sponsors of gene therapy studies recommendations 
on: (1) Methods to assess the risk of gene-therapy-related delayed 
adverse events following exposure to gene therapy products, (2) 
guidance for determining the likelihood that long-term followup 
observations on study participants will provide scientifically 
meaningful information, and (3) specific advice regarding the duration 
and design of long-term followup observations.
    This draft guidance, when finalized, will supplement the 
recommendations 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 October 2000, for study participant long-term 
followup. However, the recommendations in the Retroviral Vector 
guidance regarding the length of followup will be superseded by this 
Gene Therapy Clinical Trials guidance.
    The draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent 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 requirement of the applicable statutes 
and regulations.

II. Paperwork Reduction Act of 1995

    This guidance contains information collection provisions that 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 
information collection provisions in this guidance for the 
investigational new drug application (IND) regulations (21 CFR part 
312) have been approved under OMB control number 0910-0014; and the 
good laboratory practice (GLP) regulations (21 CFR part 58) have been 
approved under OMB control number 0910-0119.

III. Comments

    The draft guidance is being distributed for comment purposes only 
and is not intended for implementation at this time. Interested persons 
may submit to the Division of Dockets Management (see ADDRESSES) 
written or electronic comments regarding the draft 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 the brackets in 
the heading of this document. A copy of the draft guidance and received 
comments are available for public examination 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 draft guidance 
at either http://www.fda.gov/cber/guidelines.htm or http://www.fda.gov/ohrms/dockets/default.htm.

    Dated: August 12, 2005.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 05-16629 Filed 8-22-05; 8:45 am]
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