Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry; Availability, 33271-33272 [2015-14261]

Download as PDF Federal Register / Vol. 80, No. 112 / Thursday, June 11, 2015 / Notices DEPARTMENT OF HEALTH AND HUMAN SERVICES 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 https://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: Food and Drug Administration I. Background entitled ‘‘Class II Special Controls Guidance Document: Labeling for Natural Rubber Latex Condoms’’ to OMB for review and clearance under 44 U.S.C. 3507. An Agency may not conduct or sponsor, and a person is not required to respond to, a collection of information unless it displays a currently valid OMB control number. OMB has now approved the information collection and has assigned OMB control number 0910–0633. The approval expires on May 31, 2018. A copy of the supporting statement for this information collection is available on the Internet at https://www.reginfo.gov/ public/do/PRAMain. Dated: June 5, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–14285 Filed 6–10–15; 8:45 am] BILLING CODE 4164–01–P [Docket No. FDA–2013–D–0576] Considerations for the Design of EarlyPhase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice. 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 mstockstill on DSK4VPTVN1PROD with NOTICES SUMMARY: VerDate Sep<11>2014 17:06 Jun 10, 2015 Jkt 235001 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 tissuebased 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. PO 00000 Frm 00044 Fmt 4703 Sfmt 4703 33271 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 E:\FR\FM\11JNN1.SGM 11JNN1 33272 Federal Register / Vol. 80, No. 112 / Thursday, June 11, 2015 / Notices 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 https://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 https:// www.regulations.gov. IV. Electronic Access Persons with access to the Internet may obtain the guidance at either https://www.fda.gov/ BiologicsBloodVaccines/ GuidanceCompliance RegulatoryInformation/Guidances/ default.htm or https:// 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 Authority: Section 330 of the Public Health Service Act, as amended (42 U.S.C. 254b, as amended). DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Services Administration Health Center Program Health Resources and Services Administration, HHS. ACTION: Notice of class deviation from competition requirements for the Health Center Program. AGENCY: In accordance with the Awarding Agency Grants Administration Manual (AAGAM) Chapter 2.04.103, the Bureau of Primary Health Care (BPHC) has been granted a class deviation from the exceptions to maximum competition requirements contained in the AAGAM Chapter 2.04.104A–5 to provide additional funding without competition to the 115 Health Center Program awardees whose budget period ends October 31, 2015, for up to 4 months. The extension allows BPHC to eliminate the November 1 budget period start date by redistributing these grants to established starting dates later in the fiscal year, thereby allowing awardees comparable opportunity to prepare and submit applications while allowing BPHC to remain compliant with internal process timelines. SUPPLEMENTARY INFORMATION: Intended Recipient of the Award: Health Center Program awardees with a project period end date of October 31, 2015. Amount of Non-Competitive Awards: $44,481,501. Period of Supplemental Funding: November 1, 2015, to a maximum of February 28, 2016. SUMMARY: CFDA Number: 93.224. Justification: Targeting the nation’s neediest populations and geographic areas, the Health Center Program currently funds nearly 1,300 health centers that operate approximately 9,000 service delivery sites in every state, the District of Columbia, Puerto Rico, the Virgin Islands, and the Pacific Basin. In 2013, more than 21 million patients, including medically underserved and uninsured patients, received comprehensive, culturally competent, quality primary health care services through the Health Center Program awardees. Due to the vast size of the Health Center Program, the active grants are distributed across eight budget periods that begin on the first of the month, November through June. BPHC uses the information awardees report annually via the Uniform Data System (UDS) to objectively determine the patient and service area requirements that new and continuing applications must address. The requirements are available for applicant use in June. The deviation allows BPHC to redistribute the awardees with November 1 start dates to budget period start dates later in the fiscal year, thus allowing these awardees comparable opportunity to prepare and submit applications while allowing BPHC to remain compliant with internal process timelines. By September 15, 2015, $44,481,501 will be awarded to these 115 awardees to continue approved activities for up to 4 months. Awardees will report progress and financial obligations made during their budget period extension through routine reports. TABLE 1—RECIPIENT AWARDEES Grant No. mstockstill on DSK4VPTVN1PROD with NOTICES H80CS00001 H80CS00002 H80CS00003 H80CS00006 ....................... ....................... ....................... ....................... H80CS00007 H80CS00008 H80CS00009 H80CS00011 H80CS00013 H80CS00016 H80CS00017 H80CS00018 H80CS00019 H80CS00020 H80CS00022 H80CS00023 H80CS00024 H80CS00026 ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... ....................... VerDate Sep<11>2014 17:06 Jun 10, 2015 Name CITY OF SPRINGFIELD ................................................... CITY OF MANCHESTER ................................................. COMMUNITY HEALTHLINK, INC .................................... BOSTON HEALTH CARE FOR THE HOMELESS PROGRAM, INC., THE. CARE FOR THE HOMELESS .......................................... MUNICIPALITY OF SAN JUAN ........................................ CITY OF NEWARK, NEW JERSEY, INC ......................... MONTEFIORE MEDICAL CENTER ................................. UNDER 21, INC ................................................................ PUBLIC HEALTH MANAGEMENT CORPORATION ....... HEALTH CARE FOR THE HOMELESS, INC .................. DAILY PLANET, INC ........................................................ NORTH BROWARD HOSPITAL DISTRICT ..................... BIRMINGHAM HEALTH CARE, INC ................................ SAINT JOSEPHS MERCY CARE SVCS ......................... COUNTY OF HAMILTON ................................................. COUNTY OF PINELLAS .................................................. CAMILLUS HEALTH CONCERN, INC ............................. Jkt 235001 PO 00000 Frm 00045 New budget period start State Fmt 4703 Sfmt 4703 Award amount MA NH MA MA February .............................. March .................................. March .................................. January ................................ $333,353 224,147 316,608 505,654 NY PR NJ NY NY PA MD VA FL AL GA TN FL FL January ................................ March .................................. January ................................ January ................................ March .................................. January ................................ January ................................ February .............................. February .............................. January ................................ January ................................ March .................................. March .................................. January ................................ 734,361 226,508 411,022 401,335 209,692 710,886 606,970 490,501 437,971 713,355 614,459 364,024 193,752 515,685 E:\FR\FM\11JNN1.SGM 11JNN1

Agencies

[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]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 https://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 https://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 https://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the guidance at 
either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or https://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
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