[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this notice finalizes the draft guidance of the same title dated July 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]
 BILLING CODE 4164-01-P