Document ID: FDA-2012-D-1038-0017
Agency: fda
Document Type: Notice
Title: Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products; Availability
Posted Date: 2013-11-25T05:00Z

[Federal Register Volume 78, Number 227 (Monday, November 25, 2013)]
[Notices]
[Page 70307]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-28173]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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

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 document entitled ``Guidance for Industry: 
Preclinical Assessment of Investigational Cellular and Gene Therapy 
Products'' dated November 2013. The 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 reviewed 
by the Office of Cellular, Tissue and Gene Therapies (OCTGT). The 
product areas covered by this guidance are cellular therapy, gene 
therapy, therapeutic vaccination, xenotransplantation, and certain 
biologic-device combination products, which OCTGT reviews. The guidance 
clarifies current expectations regarding the preclinical information 
that would support an investigational new drug application (IND) and a 
biologics license application (BLA) for these products. The guidance 
announced in this notice finalizes the draft guidance of the same title 
dated November 2012, and supersedes the recommendations in Section VIII 
in the guidance entitled ``Guidance for Industry: Guidance for Human 
Somatic Cell Therapy and Gene Therapy'' dated March 1998.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of the guidance to 
the Office of Communication, Outreach and Development (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 electronic comments on the 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 document entitled 
``Guidance for Industry: Preclinical Assessment of Investigational 
Cellular and Gene Therapy Products'' dated November 2013. The 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 reviewed by OCTGT. The product areas covered 
by this guidance include cellular therapy, gene therapy, therapeutic 
vaccination, xenotransplantation, and certain biologic-device 
combination products. The guidance is intended to clarify current 
expectations regarding the preclinical information that supports an IND 
and a BLA for these products.
    In the Federal Register of November 29, 2012 (77 FR 71194), FDA 
announced the availability of the draft guidance of the same title 
dated November 2012. FDA received numerous comments on the draft 
guidance and those comments were considered as the guidance was 
finalized. In response to these comments, several sections were 
reorganized and editorial changes throughout the document were made to 
improve clarity. The guidance announced in this notice finalizes the 
draft guidance dated November 2012.
    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 
requirement 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 21 CFR part 312 have been approved under 
OMB control number 0910-0014; the collections of information in 21 CFR 
part 601 have been approved under OMB control number 0910-0338; the 
collections of information in 21 CFR part 58 have been approved under 
OMB control number 0910-0119.

III. Comments

    Interested persons may submit electronic comments regarding this 
document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). 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 guidance at 
either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: November 19, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-28173 Filed 11-22-13; 8:45 am]
BILLING CODE 4160-01-P