[Federal Register Volume 80, Number 112 (Thursday, June 11, 2015)]
[Notices]
[Pages 33271-33272]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-14261]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-D-0576]
Considerations for the Design of Early-Phase Clinical Trials of
Cellular and Gene Therapy Products; Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a document entitled ``Considerations for the Design
of Early-Phase Clinical Trials of Cellular and Gene Therapy Products;
Guidance for Industry.'' The guidance document is to assist sponsors
and investigators in designing early-phase clinical trials for cellular
therapy (CT) and gene therapy (GT) products (referred to collectively
as CGT products). The guidance document provides recommendations
regarding clinical trials in which the primary objectives are the
initial assessments of safety, tolerability, or feasibility of
administration of investigational products. The guidance announced in
this notice finalizes the draft guidance of the same title dated July
2013.
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, Center for
Biologics Evaluation and Research (CBER), Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-
0002. 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 240-402-7800. 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: Valerie Butler, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a document entitled
``Considerations for the Design of Early-Phase Clinical Trials of
Cellular and Gene Therapy Products; Guidance for Industry.'' The
guidance document is to assist sponsors and investigators in designing
early-phase clinical trials for CGT products. The document provides
recommendations regarding clinical trials in which the primary
objectives are the initial assessments of safety, tolerability, or
feasibility of administration of investigational products. The scope of
the guidance is limited to products for which the Office of Cellular,
Tissue, and Gene Therapies/Center for Biologics Evaluation and
Research/FDA has regulatory authority. CGT products within the scope of
the guidance meet the definition of ``biological product'' in section
351(i) of the Public Health Service (PHS) Act (42 U.S.C. 262(i)) and
include CT and GT products that are used as therapeutic vaccines. The
guidance does not apply to those human cells, tissues, and cellular-
and tissue-based products (HCT/Ps) regulated solely under section 361
of the PHS Act (42 U.S.C. 264), or to products regulated as medical
devices under the Federal Food, Drug, and Cosmetic Act, or to the
therapeutic biological products for which the Center for Drug
Evaluation and Research has regulatory responsibility.
The design of early-phase clinical trials of CGT products often
differs from the design of clinical trials for other types of
pharmaceutical products. Differences in trial design are necessitated
by the distinctive features of these products, and also may reflect
previous clinical experience. The guidance document describes features
of CGT products that influence clinical trial design, including product
characteristics, manufacturing considerations, and preclinical
considerations, and suggests other documents for additional
information. Consequently, the guidance document provides
recommendations with respect to these products as to clinical trial
design, including early phase trial objectives, choosing a study
population, using a control group and blinding, dose selection,
treatment plans, monitoring, and follow-up. Finally, the guidance
encourages prospective sponsors to meet with FDA review staff regarding
their investigational new drug application (IND) submission and offers
references for additional guidance on submitting an IND.
In the Federal Register of July 2, 2013 (78 FR 39736), FDA
announced the availability of the draft guidance of the same title
dated July 2013. FDA requested that comments on the guidance be
submitted by November 22, 2013. In the Federal Register of November 20,
2013 (78 FR 69690), FDA extended the comment period for the draft
guidance to May 9, 2014, to provide interested persons additional time
to submit comments and to allow for public discussion of the draft
guidance document at the Cellular, Tissue, and Gene Therapies Advisory
Committee meeting, which was ultimately held on February 25-26, 2014
(78 FR 79699, December 31, 2013).
FDA received a number of comments on the draft guidance and these
comments were considered as the guidance was finalized. In addition,
editorial changes were made to improve clarity. The guidance announced
in this notice finalizes the draft guidance of the same title dated
July 2013.
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 considerations for the design of early-phase
clinical trials of cellular and gene therapy products. 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
[[Page 33272]]
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.
III. Comments
Interested persons may submit either 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: June 5, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-14261 Filed 6-10-15; 8:45 am]
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