80 FR 33271 - Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 112 (June 11, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 112 (June 11, 2015)
| Page Range | 33271-33272 | |
| FR Document | 2015-14261 |
[Federal Register Volume 80, Number 112 (Thursday, June 11, 2015)] [Notices] [Pages 33271-33272] From the Federal Register Online [www.thefederalregister.org] [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 The guidance announced in this notice finalizes the draft guidance of the same title dated July 2013.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 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
![Federal Register / Vol. 80, No. 112 / Thursday, June 11, 2015 / Notices 33271
entitled ‘‘Class II Special Controls and Drug Administration, 10903 New Differences in trial design are
Guidance Document: Labeling for Hampshire Ave., Bldg. 71, Rm. 3128, necessitated by the distinctive features
Natural Rubber Latex Condoms’’ to Silver Spring, MD 20993–0002. Send of these products, and also may reflect
OMB for review and clearance under 44 one self-addressed adhesive label to previous clinical experience. The
U.S.C. 3507. An Agency may not assist the office in processing your guidance document describes features of
conduct or sponsor, and a person is not requests. The guidance may also be CGT products that influence clinical
required to respond to, a collection of obtained by mail by calling CBER at 1– trial design, including product
information unless it displays a 800–835–4709 or 240–402–7800. See characteristics, manufacturing
currently valid OMB control number. the SUPPLEMENTARY INFORMATION section considerations, and preclinical
OMB has now approved the information for electronic access to the guidance considerations, and suggests other
collection and has assigned OMB document. documents for additional information.
control number 0910–0633. The Submit electronic comments on the Consequently, the guidance document
approval expires on May 31, 2018. A guidance to http://www.regulations.gov. provides recommendations with respect
copy of the supporting statement for this Submit written comments to the to these products as to clinical trial
information collection is available on Division of Dockets Management (HFA– design, including early phase trial
the Internet at http://www.reginfo.gov/ 305), Food and Drug Administration, objectives, choosing a study population,
public/do/PRAMain. 5630 Fishers Lane, rm. 1061, Rockville, using a control group and blinding, dose
MD 20852. selection, treatment plans, monitoring,
Dated: June 5, 2015.
FOR FURTHER INFORMATION CONTACT: and follow-up. Finally, the guidance
Leslie Kux,
Valerie Butler, Center for Biologics encourages prospective sponsors to
Associate Commissioner for Policy. meet with FDA review staff regarding
[FR Doc. 2015–14285 Filed 6–10–15; 8:45 am]
Evaluation and Research, Food and
Drug Administration, 10903 New their investigational new drug
BILLING CODE 4164–01–P
Hampshire Ave., Bldg. 71, Rm. 7301, application (IND) submission and offers
Silver Spring, MD 20993–0002, 240– references for additional guidance on
402–7911. submitting an IND.
DEPARTMENT OF HEALTH AND In the Federal Register of July 2, 2013
HUMAN SERVICES SUPPLEMENTARY INFORMATION: (78 FR 39736), FDA announced the
I. Background availability of the draft guidance of the
Food and Drug Administration same title dated July 2013. FDA
FDA is announcing the availability of requested that comments on the
[Docket No. FDA–2013–D–0576]
a document entitled ‘‘Considerations for guidance be submitted by November 22,
Considerations for the Design of Early- the Design of Early-Phase Clinical Trials 2013. In the Federal Register of
Phase Clinical Trials of Cellular and of Cellular and Gene Therapy Products; November 20, 2013 (78 FR 69690), FDA
Gene Therapy Products; Guidance for Guidance for Industry.’’ The guidance extended the comment period for the
Industry; Availability document is to assist sponsors and draft guidance to May 9, 2014, to
investigators in designing early-phase provide interested persons additional
AGENCY: Food and Drug Administration, clinical trials for CGT products. The time to submit comments and to allow
HHS. document provides recommendations for public discussion of the draft
ACTION: Notice. regarding clinical trials in which the guidance document at the Cellular,
primary objectives are the initial Tissue, and Gene Therapies Advisory
SUMMARY: The Food and Drug assessments of safety, tolerability, or
Administration (FDA or Agency) is Committee meeting, which was
feasibility of administration of ultimately held on February 25–26,
announcing the availability of a investigational products. The scope of
document entitled ‘‘Considerations for 2014 (78 FR 79699, December 31, 2013).
the guidance is limited to products for FDA received a number of comments
the Design of Early-Phase Clinical Trials which the Office of Cellular, Tissue, and
of Cellular and Gene Therapy Products; on the draft guidance and these
Gene Therapies/Center for Biologics comments were considered as the
Guidance for Industry.’’ The guidance Evaluation and Research/FDA has
document is to assist sponsors and guidance was finalized. In addition,
regulatory authority. CGT products editorial changes were made to improve
investigators in designing early-phase within the scope of the guidance meet
clinical trials for cellular therapy (CT) clarity. The guidance announced in this
the definition of ‘‘biological product’’ in notice finalizes the draft guidance of the
and gene therapy (GT) products section 351(i) of the Public Health
(referred to collectively as CGT same title dated July 2013.
Service (PHS) Act (42 U.S.C. 262(i)) and The guidance is being issued
products). The guidance document include CT and GT products that are consistent with FDA’s good guidance
provides recommendations regarding used as therapeutic vaccines. The practices regulation (21 CFR 10.115).
clinical trials in which the primary guidance does not apply to those human The guidance represents FDA’s current
objectives are the initial assessments of cells, tissues, and cellular- and tissue- thinking on considerations for the
safety, tolerability, or feasibility of based products (HCT/Ps) regulated design of early-phase clinical trials of
administration of investigational solely under section 361 of the PHS Act cellular and gene therapy products. It
products. The guidance announced in (42 U.S.C. 264), or to products regulated does not create or confer any rights for
this notice finalizes the draft guidance as medical devices under the Federal or on any person and does not operate
of the same title dated July 2013. Food, Drug, and Cosmetic Act, or to the to bind FDA or the public. An
DATES: Submit either electronic or
mstockstill on DSK4VPTVN1PROD with NOTICES
therapeutic biological products for alternative approach may be used if
written comments on Agency guidances which the Center for Drug Evaluation such approach satisfies the
at any time. and Research has regulatory requirements of the applicable statutes
ADDRESSES: Submit written requests for responsibility. and regulations.
single copies of the guidance to the The design of early-phase clinical
Office of Communication, Outreach, and trials of CGT products often differs from II. Paperwork Reduction Act of 1995
Development, Center for Biologics the design of clinical trials for other This guidance refers to previously
Evaluation and Research (CBER), Food types of pharmaceutical products. approved collections of information
VerDate Sep<11>2014 17:06 Jun 10, 2015 Jkt 235001 PO 00000 Frm 00044 Fmt 4703 Sfmt 4703 E:\FR\FM\11JNN1.SGM 11JNN1](https://thefederalregister.org/doc/80_FR_33383/page/1.svg)
![33272 Federal Register / Vol. 80, No. 112 / Thursday, June 11, 2015 / Notices
found in FDA regulations. These DEPARTMENT OF HEALTH AND Authority: Section 330 of the Public Health
collections of information are subject to HUMAN SERVICES Service Act, as amended (42 U.S.C. 254b, as
review by the Office of Management and amended).
Budget (OMB) under the Paperwork Health Resources and Services
Administration Justification: Targeting the nation’s
Reduction Act of 1995 (44 U.S.C. 3501– neediest populations and geographic
3520). The collections of information in Health Center Program areas, the Health Center Program
21 CFR part 312 have been approved currently funds nearly 1,300 health
under OMB control number 0910–0014. AGENCY: Health Resources and Services centers that operate approximately
Administration, HHS. 9,000 service delivery sites in every
III. Comments ACTION: Notice of class deviation from state, the District of Columbia, Puerto
Interested persons may submit either competition requirements for the Health Rico, the Virgin Islands, and the Pacific
electronic comments regarding this Center Program. Basin. In 2013, more than 21 million
document to http://www.regulations.gov SUMMARY: In accordance with the patients, including medically
or written comments to the Division of Awarding Agency Grants underserved and uninsured patients,
Dockets Management (see ADDRESSES). It Administration Manual (AAGAM) received comprehensive, culturally
is only necessary to send one set of Chapter 2.04.103, the Bureau of Primary competent, quality primary health care
comments. Identify comments with the Health Care (BPHC) has been granted a services through the Health Center
docket number found in brackets in the class deviation from the exceptions to Program awardees. Due to the vast size
heading of this document. Received maximum competition requirements of the Health Center Program, the active
contained in the AAGAM Chapter grants are distributed across eight
comments may be seen in the Division
2.04.104A–5 to provide additional budget periods that begin on the first of
of Dockets Management between 9 a.m.
funding without competition to the 115 the month, November through June.
and 4 p.m., Monday through Friday, and
will be posted to the docket at http:// Health Center Program awardees whose BPHC uses the information awardees
www.regulations.gov. budget period ends October 31, 2015, report annually via the Uniform Data
for up to 4 months. The extension System (UDS) to objectively determine
IV. Electronic Access allows BPHC to eliminate the November the patient and service area
1 budget period start date by requirements that new and continuing
Persons with access to the Internet redistributing these grants to established applications must address. The
may obtain the guidance at either starting dates later in the fiscal year, requirements are available for applicant
http://www.fda.gov/ thereby allowing awardees comparable use in June. The deviation allows BPHC
BiologicsBloodVaccines/ opportunity to prepare and submit to redistribute the awardees with
GuidanceCompliance applications while allowing BPHC to November 1 start dates to budget period
RegulatoryInformation/Guidances/ remain compliant with internal process start dates later in the fiscal year, thus
default.htm or http:// timelines. allowing these awardees comparable
www.regulations.gov. SUPPLEMENTARY INFORMATION: opportunity to prepare and submit
Dated: June 5, 2015. Intended Recipient of the Award: applications while allowing BPHC to
Health Center Program awardees with a remain compliant with internal process
Leslie Kux,
project period end date of October 31, timelines. By September 15, 2015,
Associate Commissioner for Policy. 2015. $44,481,501 will be awarded to these
[FR Doc. 2015–14261 Filed 6–10–15; 8:45 am] Amount of Non-Competitive Awards: 115 awardees to continue approved
BILLING CODE 4164–01–P $44,481,501. activities for up to 4 months. Awardees
Period of Supplemental Funding: will report progress and financial
November 1, 2015, to a maximum of obligations made during their budget
February 28, 2016. period extension through routine
CFDA Number: 93.224. reports.
TABLE 1—RECIPIENT AWARDEES
New budget
Grant No. Name State Award amount
period start
H80CS00001 ....................... CITY OF SPRINGFIELD ................................................... MA February .............................. $333,353
H80CS00002 ....................... CITY OF MANCHESTER ................................................. NH March .................................. 224,147
H80CS00003 ....................... COMMUNITY HEALTHLINK, INC .................................... MA March .................................. 316,608
H80CS00006 ....................... BOSTON HEALTH CARE FOR THE HOMELESS PRO- MA January ................................ 505,654
GRAM, INC., THE.
H80CS00007 ....................... CARE FOR THE HOMELESS .......................................... NY January ................................ 734,361
H80CS00008 ....................... MUNICIPALITY OF SAN JUAN ........................................ PR March .................................. 226,508
H80CS00009 ....................... CITY OF NEWARK, NEW JERSEY, INC ......................... NJ January ................................ 411,022
H80CS00011 ....................... MONTEFIORE MEDICAL CENTER ................................. NY January ................................ 401,335
H80CS00013 ....................... UNDER 21, INC ................................................................ NY March .................................. 209,692
H80CS00016 ....................... PUBLIC HEALTH MANAGEMENT CORPORATION ....... PA January ................................ 710,886
mstockstill on DSK4VPTVN1PROD with NOTICES
H80CS00017 ....................... HEALTH CARE FOR THE HOMELESS, INC .................. MD January ................................ 606,970
H80CS00018 ....................... DAILY PLANET, INC ........................................................ VA February .............................. 490,501
H80CS00019 ....................... NORTH BROWARD HOSPITAL DISTRICT ..................... FL February .............................. 437,971
H80CS00020 ....................... BIRMINGHAM HEALTH CARE, INC ................................ AL January ................................ 713,355
H80CS00022 ....................... SAINT JOSEPHS MERCY CARE SVCS ......................... GA January ................................ 614,459
H80CS00023 ....................... COUNTY OF HAMILTON ................................................. TN March .................................. 364,024
H80CS00024 ....................... COUNTY OF PINELLAS .................................................. FL March .................................. 193,752
H80CS00026 ....................... CAMILLUS HEALTH CONCERN, INC ............................. FL January ................................ 515,685
VerDate Sep<11>2014 17:06 Jun 10, 2015 Jkt 235001 PO 00000 Frm 00045 Fmt 4703 Sfmt 4703 E:\FR\FM\11JNN1.SGM 11JNN1](https://thefederalregister.org/doc/80_FR_33383/page/2.svg)
Document Created: 2015-12-15 15:07:29
Document Modified: 2015-12-15 15:07:29
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| Dates | Submit either electronic or written comments on Agency guidances at any time. | |
| 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. | |
| FR Citation | 80 FR 33271 |
2024 Federal Register | Disclaimer | Privacy Policy
USC | CFR | eCFR