[Federal Register Volume 77, Number 230 (Thursday, November 29, 2012)]
[Notices]
[Page 71194]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-28882]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-D-1038]


Draft Guidance for Industry: Preclinical Assessment of 
Investigational Cellular and Gene Therapy Products; 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: 
Preclinical Assessment of Investigational Cellular and Gene Therapy 
Products,'' dated November 2012. The draft guidance document provides 
sponsors and individuals that design and implement preclinical studies 
with recommendations on the substance and scope of preclinical 
information needed to support clinical trials for investigational 
products regulated by the Center for Biologics Research and Evaluation 
(CBER), Office of Cellular, Tissue, and Gene Therapies (OCTGT). The 
product areas covered by this guidance are cellular therapy, gene 
therapy, therapeutic vaccination, and xenotransplantation. The guidance 
is intended to clarify current expectations regarding the preclinical 
information that supports an investigational new drug application (IND) 
and a biologics license application (BLA) for these product areas.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by February 27, 2013.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Office of Communication, Outreach and Development (HFM-
40), 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 The draft guidance 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access to the draft guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Tami Belouin, 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: Preclinical Assessment of Investigational 
Cellular and Gene Therapy Products,'' dated November 2012. The draft 
guidance document provides sponsors and individuals that design and 
implement preclinical studies with recommendations on the substance and 
scope of preclinical information needed to support clinical trials for 
investigational products regulated by OCTGT. The product areas covered 
by this guidance are cellular therapy, gene therapy, therapeutic 
vaccination, and xenotransplantation. The guidance is intended to 
clarify current expectations regarding the preclinical information that 
supports an IND and a BLA for these product areas.
    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 draft 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 21 CFR part 312 has been approved under 
0910-0014; the collections of information in 21 CFR part 601 has been 
approved under 0910-0338; and the collections of information in 21 CFR 
part 58 has been approved under 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 either written comments regarding this document to the 
Division of Dockets Management (see ADDRESSES) or electronic comments 
to http://www.regulations.gov. It is only necessary to send one set of 
comments. Identify comments with the docket number found in brackets in 
the heading of this document. Received comments may be seen in the 
Division of Dockets Management between 9 a.m. and 4 p.m., Monday 
through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the draft guidance 
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: November 26, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-28882 Filed 11-28-12; 8:45 am]
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