[Federal Register Volume 78, Number 127 (Tuesday, July 2, 2013)]
[Notices]
[Pages 39736-39737]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-15797]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-0576]


Draft Guidance for Industry: Considerations for the Design of 
Early-Phase Clinical Trials of 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availability of a draft guidance document entitled ``Guidance for 
Industry: Considerations for the Design of Early-Phase Clinical Trials 
of Cellular and Gene Therapy Products'' dated July 2013. The draft 
guidance document provides sponsors of Investigational New Drug 
Applications (INDs) for cellular therapy (CT) and gene therapy (GT) 
products (referred to collectively as CGT products) with 
recommendations to assist in designing early-phase clinical trials of 
CGT products.

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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by November 22, 2013.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Office of Communication, Outreach and Development (HFM-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 The draft guidance may also be obtained by 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SUPPLEMENTARY INFORMATION section for electronic access to the draft 
guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov.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: Melissa Reisman, Center for Biologics 
Evaluation and Research (HFM-17), Food and Drug Administration, 1401 
Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-6210.

[[Page 39737]]


SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance document 
entitled ``Guidance for Industry: Considerations for the Design of 
Early-Phase Clinical Trials of Cellular and Gene Therapy Products,'' 
dated July 2013. The draft guidance document provides sponsors of INDs 
for CGT products with recommendations to assist in designing early-
phase clinical trials of CGT products. The scope of this guidance is 
limited to products for which the Office of Cellular, Tissue and Gene 
Therapies/FDA has regulatory authority. CGT products within the scope 
of this guidance meet the definition of ``biological product'' in 
section 351(i) of the Public Health Service (PHS) Act (42 U.S.C. 
262(i)). 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), to products regulated as 
medical devices under the Federal Food, Drug, and Cosmetic Act (the 
FD&C Act), or to the therapeutic biological products for which the 
Center for Drug Evaluation and Research (CDER) 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 draft 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 draft 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 draft guidance 
encourages prospective sponsors to meet with FDA review staff regarding 
their IND submission and offers references for additional guidance on 
submitting an IND.
    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 have been approved under 
OMB control number 0910-0014.

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 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 draft guidance 
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: June 25, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-15797 Filed 7-1-13; 8:45 am]
BILLING CODE 4160-01-P