[Federal Register Volume 78, Number 227 (Monday, November 25, 2013)]
[Notices]
[Page 70307]
From the Federal Register Online via the Government Publishing 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announced in this notice finalizes the draft guidance of the same title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