Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry; Availability, 33271-33272 [2015-14261]
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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
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Natural Rubber Latex Condoms’’ to
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conduct or sponsor, and a person is not
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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