Graft-Versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment; Draft Guidance for Industry; Availability, 67301-67303 [2023-21524]
Download as PDF
Federal Register / Vol. 88, No. 188 / Friday, September 29, 2023 / Notices
OMB control number. To be assured
consideration, comments and
recommendations must be submitted in
any one of the following ways:
1. Electronically. You may send your
comments electronically to https://
www.regulations.gov. Follow the
instructions for ‘‘Comment or
Submission’’ or ‘‘More Search Options’’
to find the information collection
document(s) that are accepting
comments.
2. By regular mail. You may mail
written comments to the following
address: CMS, Office of Strategic
Operations and Regulatory Affairs,
Division of Regulations Development,
Attention: Document Identifier/OMB
Control Number: ll, Room C4–26–05,
7500 Security Boulevard, Baltimore,
Maryland 21244–1850.
To obtain copies of a supporting
statement and any related forms for the
proposed collection(s) summarized in
this notice, please access the CMS PRA
website by copying and pasting the
following web address into your web
browser: https://www.cms.gov/
Regulations-and-Guidance/Legislation/
PaperworkReductionActof1995/PRAListing.
FOR FURTHER INFORMATION CONTACT:
William N. Parham at (410) 786–4669.
SUPPLEMENTARY INFORMATION:
lotter on DSK11XQN23PROD with NOTICES1
Contents
This notice sets out a summary of the
use and burden associated with the
following information collections. More
detailed information can be found in
each collection’s supporting statement
and associated materials (see
ADDRESSES).
CMS–1561/1561A Health Insurance
Benefit Agreement
Under the PRA (44 U.S.C. 3501–
3520), federal agencies must obtain
approval from the Office of Management
and Budget (OMB) for each collection of
information they conduct or sponsor.
The term ‘‘collection of information’’ is
defined in 44 U.S.C. 3502(3) and 5 CFR
1320.3(c) and includes agency requests
or requirements that members of the
public submit reports, keep records, or
provide information to a third party.
Section 3506(c)(2)(A) of the PRA
requires federal agencies to publish a
60-day notice in the Federal Register
concerning each proposed collection of
information, including each proposed
extension or reinstatement of an existing
collection of information, before
submitting the collection to OMB for
approval. To comply with this
requirement, CMS is publishing this
notice.
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Information Collection
67301
Notice; exemption period
extension.
ACTION:
1. Type of Information Collection
Request: Reinstatement without change
of a previously approved collection;
Title of Information Collection: Health
Insurance Benefit Agreement; Use: The
CMS–1561 form applies to specific
types of health care providers and
opioid treatment programs and the
CMS–1561A form applies to rural health
clinics (RHCs). The CMS–1561 and
CMS–1561A forms are health insurance
benefits agreements that are essential for
the Centers for Medicare and Medicaid
Services (CMS) to ensure that applicants
to the Medicare program have made a
binding commitment to comply with all
applicable Federal requirements. The
CMS–1561/1561A forms are essential in
that they allow CMS to ensure that
applicants are in compliance with the
requirements. Applicants will be
required to sign the completed form and
provide operational information to CMS
to assure that they continue to meet the
requirements after approval. The
collection is made only once, when the
provider or RHC submits their
application for participation in
Medicare by signing the completed
CMS–1561 or CMS–1561A form (as
applicable). Form Number: CMS–1561/
1561A (OMB control number: 0938–
0832); Frequency: Once only; Affected
Public: Private sector—(Business or
other for-profits and Not-for-profit
institutions); Number of Respondents:
2,050; Total Annual Responses: 2,050;
Total Annual Hours: 2,050. (For policy
questions regarding this collection
contact Caroline Gallaher at 410–786–
8705).
Dated: September 25, 2023
William N. Parham, III,
Director, Paperwork Reduction Staff, Office
of Strategic Operations and Regulatory
Affairs.
The Centers for Medicare &
Medicaid Services (CMS) announce the
extension of the Clinical Laboratory
Improvement Amendments of 1988
(CLIA) exemption period for the State of
Washington. The exemption period is
extended for 6 months, that is until
April 2, 2024.
DATES: The exemption granted by this
notice is effective from October 2, 2023
to April 2, 2024.
FOR FURTHER INFORMATION CONTACT:
Mary Hasan, (410) 786–6480.
SUPPLEMENTARY INFORMATION: In the
‘‘Medicare, Medicaid, and CLIA
Programs; Clinical Laboratory
Improvement Amendments of 1988
Exemption of Laboratories Licensed by
the State of Washington’’ notice that
appeared in the September 30, 2019
Federal Register (84 FR 51591), we
announced that laboratories located in
and licensed by the State of Washington
that possess a valid license under the
Medical Test Site law, Chapter 70.42 of
the Revised Code of Washington, are
exempt from the requirements of the
Clinical Laboratory Improvement
Amendments of 1988 (CLIA) for a
period of 4 years. This period expires on
October 2, 2023. Pending re-approval of
Washington State’s CLIA exemption
period, we are extending Washington
State’s current CLIA exemption period
for 6 months, that is until April 2, 2024.
The Administrator of CMS, Chiquita
Brooks-LaSure, having reviewed and
approved this document, authorizes
Chyana Woodard, who is the Federal
Register Liaison, to electronically sign
this document for purposes of
publication in the Federal Register.
SUMMARY:
[FR Doc. 2023–21334 Filed 9–28–23; 8:45 am]
Chyana Woodard,
Federal Register Liaison, Centers for Medicare
& Medicaid Services.
BILLING CODE 4120–01–P
[FR Doc. 2023–21460 Filed 9–28–23; 8:45 am]
BILLING CODE 4120–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Centers for Medicare & Medicaid
Services
Clinical Laboratory Improvement
Amendments of 1988 Exemption of
Laboratories Licensed by the State of
Washington; Exemption Period
Extension
Centers for Medicare &
Medicaid Services (CMS), Department
of Health and Human Services (HHS).
AGENCY:
Frm 00077
Fmt 4703
Food and Drug Administration
[Docket No. FDA–2023–D–3900]
[CMS–3383–N2]
PO 00000
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Sfmt 4703
Graft-Versus-Host Diseases:
Developing Drugs, Biological
Products, and Certain Devices for
Prevention or Treatment; Draft
Guidance for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
E:\FR\FM\29SEN1.SGM
Notice of availability.
29SEN1
67302
Federal Register / Vol. 88, No. 188 / Friday, September 29, 2023 / Notices
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘Graftversus-Host Diseases: Developing Drugs,
Biological Products, and Certain Devices
for Prevention or Treatment.’’ The
purpose of this guidance is to assist
sponsors in the clinical development of
drugs, biological products, and certain
devices for the prevention or treatment
of acute graft-versus-host disease
(aGVHD) or chronic graft-vs-host disease
(cGVHD). Specifically, this guidance
addresses FDA’s current thinking
regarding the overall clinical
development program and critical
design elements for early and late phase
trials for the intended populations. This
guidance focuses on clinical trial
design, statistical analysis, or other
issues specific to aGVHD or cGVHD,
and it does not contain a discussion of
the general principles regarding
statistical analysis, clinical trial design,
or drug development. Additionally, this
guidance is not intended to provide
advice on the technical aspects of
therapeutic or cell-processing devices.
SUMMARY:
Submit either electronic or
written comments on the draft guidance
by November 28, 2023 to ensure that the
Agency considers your comment on this
draft guidance before it begins work on
the final version of the guidance.
DATES:
You may submit comments
on any guidance at any time as follows:
ADDRESSES:
lotter on DSK11XQN23PROD with NOTICES1
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
instructions for submitting comments.
Comments submitted electronically,
including attachments, to https://
www.regulations.gov will be posted to
the docket unchanged. Because your
comment will be made public, you are
solely responsible for ensuring that your
comment does not include any
confidential information that you or a
third party may not wish to be posted,
such as medical information, your or
anyone else’s Social Security number, or
confidential business information, such
as a manufacturing process. Please note
that if you include your name, contact
information, or other information that
identifies you in the body of your
comments, that information will be
posted on https://www.regulations.gov.
• If you want to submit a comment
with confidential information that you
do not wish to be made available to the
public, submit the comment as a
written/paper submission and in the
VerDate Sep<11>2014
21:46 Sep 28, 2023
Jkt 259001
manner detailed (see ‘‘Written/Paper
Submissions’’ and ‘‘Instructions’’).
Written/Paper Submissions
Submit written/paper submissions as
follows:
• Mail/Hand Delivery/Courier (for
written/paper submissions): Dockets
Management Staff (HFA–305), Food and
Drug Administration, 5630 Fishers
Lane, Rm. 1061, Rockville, MD 20852.
• For written/paper comments
submitted to the Dockets Management
Staff, FDA will post your comment, as
well as any attachments, except for
information submitted, marked and
identified, as confidential, if submitted
as detailed in ‘‘Instructions.’’
Instructions: All submissions received
must include the Docket No. FDA–
2023–D–3900 for ‘‘Graft-versus-Host
Diseases: Developing Drugs and
Biological Products for Prevention or
Treatment.’’ Received comments will be
placed in the docket and, except for
those submitted as ‘‘Confidential
Submissions,’’ publicly viewable at
https://www.regulations.gov or at the
Dockets Management Staff between 9
a.m. and 4 p.m., Monday through
Friday, 240–402–7500.
• Confidential Submissions—To
submit a comment with confidential
information that you do not wish to be
made publicly available, submit your
comments only as a written/paper
submission. You should submit two
copies total. One copy will include the
information you claim to be confidential
with a heading or cover note that states
‘‘THIS DOCUMENT CONTAINS
CONFIDENTIAL INFORMATION.’’ The
Agency will review this copy, including
the claimed confidential information, in
its consideration of comments. The
second copy, which will have the
claimed confidential information
redacted/blacked out, will be available
for public viewing and posted on
https://www.regulations.gov. Submit
both copies to the Dockets Management
Staff. If you do not wish your name and
contact information to be made publicly
available, you can provide this
information on the cover sheet and not
in the body of your comments and you
must identify this information as
‘‘confidential.’’ Any information marked
as ‘‘confidential’’ will not be disclosed
except in accordance with 21 CFR 10.20
and other applicable disclosure law. For
more information about FDA’s posting
of comments to public dockets, see 80
FR 56469, September 18, 2015, or access
the information at: https://
www.govinfo.gov/content/pkg/FR-201509-18/pdf/2015-23389.pdf.
Docket: For access to the docket to
read background documents or the
PO 00000
Frm 00078
Fmt 4703
Sfmt 4703
electronic and written/paper comments
received, go to https://
www.regulations.gov and insert the
docket number, found in brackets in the
heading of this document, into the
‘‘Search’’ box and follow the prompts
and/or go to the Dockets Management
Staff, 5630 Fishers Lane, Rm. 1061,
Rockville, MD 20852, 240–402–7500.
You may submit comments on any
guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single
copies of the draft guidance to the
Division of Drug Information, Center for
Drug Evaluation and Research, Food
and Drug Administration, 10001 New
Hampshire Ave., Hillandale Building,
4th Floor, Silver Spring, MD 20993–
0002; 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; or to the Office of Policy, Center
for Devices and Radiological Health,
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 66, Rm.
5431, Silver Spring, MD 20993–0002.
Send one self-addressed adhesive label
to assist that office in processing your
requests. See the SUPPLEMENTARY
INFORMATION section for electronic
access to the draft guidance document.
FOR FURTHER INFORMATION CONTACT:
Robert Le, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 2124, Silver Spring,
MD 20993, 240–402–8320, or Anne
Taylor, Center for Biologics Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 71, Rm. 7256, Silver Spring,
MD 20993, 240–402–5683.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft guidance for industry entitled
‘‘Graft-versus-Host Diseases: Developing
Drugs, Biological Products, and Certain
Devices for Prevention or Treatment.’’
The purpose of this guidance is to assist
sponsors in the clinical development of
drugs, biological products, and certain
devices for the prevention or treatment
of aGVHD or cGVHD. Specifically, this
guidance addresses FDA’s current
thinking regarding the overall clinical
development program and critical
design elements for early and late phase
trials for the intended populations.
aGVHD and cGVHD are clinical
syndromes that may arise after
allogeneic hematopoietic stem cell
transplantation as a result of
E:\FR\FM\29SEN1.SGM
29SEN1
Federal Register / Vol. 88, No. 188 / Friday, September 29, 2023 / Notices
lotter on DSK11XQN23PROD with NOTICES1
immunocompetent donor cells
recognizing and reacting to disparity
with major or minor histocompatibility
antigens on recipient tissues. The
classical approach to prevention of
GVHD involves pharmacological or
physical methods to delete alloreactive
T cells in the immediate peritransplant
setting with or without additional drugs
to prevent activation of naive T cells.
Should aGVHD or cGVHD occur despite
these measures, treatment has depended
largely on drugs that impair T cells.
Further basic science investigations
have elucidated the molecular
mechanisms behind the clinical
manifestations of aGVHD and cGVHD,
including cytokines, the innate immune
system, and components of the adaptive
immune system other than T cells.
These scientific advances have provided
opportunities for development of
biomarkers to identify the specific
immune dysfunction present in an
individual patient and for development
of drugs to modulate the immune
system with precision rather than to just
suppress the immune system globally.
Given the complexity of the clinical
manifestation of aGVHD and cGVHD
and the potential for a paradigm shift in
the management of GVHD, this guidance
provides recommendations regarding
the design and conduct of clinical trials
and the types of supporting data that
could facilitate efficient development of
drugs and/or certain devices for the
prevention or treatment of aGVHD or
cGVHD. This guidance also provides
recommendations on what should be
included in the marketing application to
facilitate review.
This draft guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
The draft guidance, when finalized, will
represent the current thinking of FDA
on ‘‘Graft-versus-Host Diseases:
Developing Drugs, Biological Products,
and Certain Devices for Prevention or
Treatment.’’ It does not establish any
rights for any person and is not binding
on FDA or the public. You can use an
alternative approach if it satisfies the
requirements of the applicable statutes
and regulations.
II. Paperwork Reduction Act of 1995
While this guidance contains no
collection of information, it does refer to
previously approved FDA collections of
information. The previously approved
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–
3521). The collections of information in
21 CFR part 312 have been approved
under OMB control number 0910–0014;
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21:46 Sep 28, 2023
Jkt 259001
the collections of information in 21 CFR
part 314 have been approved under
OMB control number 0910–0001; the
collections of information in 21 CFR
part 601 have been approved under
OMB control number 0910–0338; the
collections of information in 21 CFR
part 812 have been approved under
OMB control number 0910–0078; and
the collections of information in 21 CFR
parts 50 and 56 have been approved
under OMB control number 0910–0130.
III. Electronic Access
67303
by using the search function. The OMB
control number for this information
collection is 0910–0256. Also include
the FDA docket number found in
brackets in the heading of this
document.
FOR FURTHER INFORMATION CONTACT:
Domini Bean, Office of Operations,
Food and Drug Administration, Three
White Flint North, 10A–12M, 11601
Landsdown St., North Bethesda, MD
20852, 301–796–5733, PRAStaff@
fda.hhs.gov.
In
compliance with 44 U.S.C. 3507, FDA
has submitted the following proposed
collection of information to OMB for
review and clearance.
Persons with access to the internet
may obtain the draft guidance at https://
www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, https://
www.fda.gov/regulatory-information/
search-fda-guidance-documents, or
https://www.regulations.gov.
SUPPLEMENTARY INFORMATION:
Dated: September 26, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
This information collection supports
FDA regulations, and associated Agency
forms and guidance, pertaining to infant
formula requirements. Statutory
provisions for infant formula under the
Federal Food, Drug, and Cosmetic Act
(FD&C Act) were enacted to protect the
health of infants and include specific
current good manufacturing practice,
labeling (disclosure), and a number of
reporting and recordkeeping
requirements. Section 412 of the FD&C
Act (21 U.S.C. 350a) requires
manufacturers of infant formula to
establish and document the adherence
to quality control procedures, notify
FDA when a batch of infant formula that
has left the manufacturers’ control may
be adulterated or misbranded, and keep
records of infant formula distribution.
Notification requirements are also
included in the regulations regarding
the quantitative formulation of the
infant formula; a description of any
reformulation or change in processing;
assurances that the formula will not be
marketed until regulatory requirements
are met as demonstrated by specific
testing; and assurances that
manufacturing processes comply with
the regulations. The regulations are
found in 21 CFR part 106: Infant
Formula Requirements Pertaining to
Current Good Manufacturing Practice,
Quality Control Procedures, Quality
Factors, Records and Reports, and
Notifications; and part 107 (21 CFR part
107): Infant Formula.
In the Federal Register of October 6,
2022 (87 FR 60689), we provided notice
communicating updates to the
information collection and invited
public comment on the proposed
[FR Doc. 2023–21524 Filed 9–28–23; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2022–D–0814]
Agency Information Collection
Activities; Submission for Office of
Management and Budget Review;
Comment Request; Infant Formula
Recalls
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA, the Agency, or
we) is announcing that a proposed
collection of information has been
submitted to the Office of Management
and Budget (OMB) for review and
clearance under the Paperwork
Reduction Act of 1995.
DATES: Submit written comments
(including recommendations) on the
collection of information by October 30,
2023.
ADDRESSES: To ensure that comments on
the information collection are received,
OMB recommends that written
comments be submitted to https://
www.reginfo.gov/public/do/PRAMain.
Find this particular information
collection by selecting ‘‘Currently under
Review—Open for Public Comments’’ or
SUMMARY:
PO 00000
Frm 00079
Fmt 4703
Sfmt 4703
Infant Formula Requirements Under
the Federal Food, Drug, and Cosmetic
Act—21 CFR Parts 106 and 107
OMB Control Number 0910–0256—
Revision
E:\FR\FM\29SEN1.SGM
29SEN1
Agencies
[Federal Register Volume 88, Number 188 (Friday, September 29, 2023)]
[Notices]
[Pages 67301-67303]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-21524]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-D-3900]
Graft-Versus-Host Diseases: Developing Drugs, Biological
Products, and Certain Devices for Prevention or Treatment; Draft
Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
-----------------------------------------------------------------------
[[Page 67302]]
SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft guidance for industry entitled ``Graft-
versus-Host Diseases: Developing Drugs, Biological Products, and
Certain Devices for Prevention or Treatment.'' The purpose of this
guidance is to assist sponsors in the clinical development of drugs,
biological products, and certain devices for the prevention or
treatment of acute graft-versus-host disease (aGVHD) or chronic graft-
vs-host disease (cGVHD). Specifically, this guidance addresses FDA's
current thinking regarding the overall clinical development program and
critical design elements for early and late phase trials for the
intended populations. This guidance focuses on clinical trial design,
statistical analysis, or other issues specific to aGVHD or cGVHD, and
it does not contain a discussion of the general principles regarding
statistical analysis, clinical trial design, or drug development.
Additionally, this guidance is not intended to provide advice on the
technical aspects of therapeutic or cell-processing devices.
DATES: Submit either electronic or written comments on the draft
guidance by November 28, 2023 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final
version of the guidance.
ADDRESSES: You may submit comments on any guidance at any time as
follows:
Electronic Submissions
Submit electronic comments in the following way:
Federal eRulemaking Portal: https://www.regulations.gov.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to https://www.regulations.gov
will be posted to the docket unchanged. Because your comment will be
made public, you are solely responsible for ensuring that your comment
does not include any confidential information that you or a third party
may not wish to be posted, such as medical information, your or anyone
else's Social Security number, or confidential business information,
such as a manufacturing process. Please note that if you include your
name, contact information, or other information that identifies you in
the body of your comments, that information will be posted on https://www.regulations.gov.
If you want to submit a comment with confidential
information that you do not wish to be made available to the public,
submit the comment as a written/paper submission and in the manner
detailed (see ``Written/Paper Submissions'' and ``Instructions'').
Written/Paper Submissions
Submit written/paper submissions as follows:
Mail/Hand Delivery/Courier (for written/paper
submissions): Dockets Management Staff (HFA-305), Food and Drug
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Dockets
Management Staff, FDA will post your comment, as well as any
attachments, except for information submitted, marked and identified,
as confidential, if submitted as detailed in ``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-2023-D-3900 for ``Graft-versus-Host Diseases: Developing Drugs and
Biological Products for Prevention or Treatment.'' Received comments
will be placed in the docket and, except for those submitted as
``Confidential Submissions,'' publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday, 240-402-7500.
Confidential Submissions--To submit a comment with
confidential information that you do not wish to be made publicly
available, submit your comments only as a written/paper submission. You
should submit two copies total. One copy will include the information
you claim to be confidential with a heading or cover note that states
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will
review this copy, including the claimed confidential information, in
its consideration of comments. The second copy, which will have the
claimed confidential information redacted/blacked out, will be
available for public viewing and posted on https://www.regulations.gov.
Submit both copies to the Dockets Management Staff. If you do not wish
your name and contact information to be made publicly available, you
can provide this information on the cover sheet and not in the body of
your comments and you must identify this information as
``confidential.'' Any information marked as ``confidential'' will not
be disclosed except in accordance with 21 CFR 10.20 and other
applicable disclosure law. For more information about FDA's posting of
comments to public dockets, see 80 FR 56469, September 18, 2015, or
access the information at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in
the heading of this document, into the ``Search'' box and follow the
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852, 240-402-7500.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single copies of the draft guidance to
the Division of Drug Information, Center for Drug Evaluation and
Research, Food and Drug Administration, 10001 New Hampshire Ave.,
Hillandale Building, 4th Floor, Silver Spring, MD 20993-0002; 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; or to
the Office of Policy, Center for Devices and Radiological Health, Food
and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 5431,
Silver Spring, MD 20993-0002. Send one self-addressed adhesive label to
assist that office in processing your requests. See the SUPPLEMENTARY
INFORMATION section for electronic access to the draft guidance
document.
FOR FURTHER INFORMATION CONTACT: Robert Le, Center for Drug Evaluation
and Research, Food and Drug Administration, 10903 New Hampshire Ave.,
Bldg. 22, Rm. 2124, Silver Spring, MD 20993, 240-402-8320, or Anne
Taylor, Center for Biologics Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7256, Silver
Spring, MD 20993, 240-402-5683.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Graft-versus-Host Diseases: Developing Drugs, Biological
Products, and Certain Devices for Prevention or Treatment.'' The
purpose of this guidance is to assist sponsors in the clinical
development of drugs, biological products, and certain devices for the
prevention or treatment of aGVHD or cGVHD. Specifically, this guidance
addresses FDA's current thinking regarding the overall clinical
development program and critical design elements for early and late
phase trials for the intended populations.
aGVHD and cGVHD are clinical syndromes that may arise after
allogeneic hematopoietic stem cell transplantation as a result of
[[Page 67303]]
immunocompetent donor cells recognizing and reacting to disparity with
major or minor histocompatibility antigens on recipient tissues. The
classical approach to prevention of GVHD involves pharmacological or
physical methods to delete alloreactive T cells in the immediate
peritransplant setting with or without additional drugs to prevent
activation of naive T cells. Should aGVHD or cGVHD occur despite these
measures, treatment has depended largely on drugs that impair T cells.
Further basic science investigations have elucidated the molecular
mechanisms behind the clinical manifestations of aGVHD and cGVHD,
including cytokines, the innate immune system, and components of the
adaptive immune system other than T cells. These scientific advances
have provided opportunities for development of biomarkers to identify
the specific immune dysfunction present in an individual patient and
for development of drugs to modulate the immune system with precision
rather than to just suppress the immune system globally.
Given the complexity of the clinical manifestation of aGVHD and
cGVHD and the potential for a paradigm shift in the management of GVHD,
this guidance provides recommendations regarding the design and conduct
of clinical trials and the types of supporting data that could
facilitate efficient development of drugs and/or certain devices for
the prevention or treatment of aGVHD or cGVHD. This guidance also
provides recommendations on what should be included in the marketing
application to facilitate review.
This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent the current thinking of FDA on ``Graft-
versus-Host Diseases: Developing Drugs, Biological Products, and
Certain Devices for Prevention or Treatment.'' It does not establish
any rights for any person and is not binding on FDA or the public. You
can use an alternative approach if it satisfies the requirements of the
applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
While this guidance contains no collection of information, it does
refer to previously approved FDA collections of information. The
previously approved 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. 3501-3521). The collections of information in 21
CFR part 312 have been approved under OMB control number 0910-0014; the
collections of information in 21 CFR part 314 have been approved under
OMB control number 0910-0001; the collections of information in 21 CFR
part 601 have been approved under OMB control number 0910-0338; the
collections of information in 21 CFR part 812 have been approved under
OMB control number 0910-0078; and the collections of information in 21
CFR parts 50 and 56 have been approved under OMB control number 0910-
0130.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.
Dated: September 26, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-21524 Filed 9-28-23; 8:45 am]
BILLING CODE 4164-01-P