Notice2023-21524
Graft-Versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment; Draft Guidance for Industry; Availability
Primary source
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Published
September 29, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Graft- versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment."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.
Full Text
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<title>Federal Register, Volume 88 Issue 188 (Friday, September 29, 2023)</title>
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[Federal Register Volume 88, Number 188 (Friday, September 29, 2023)]
[Notices]
[Pages 67301-67303]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-21524]
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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Products, and Certain Devices for Prevention or Treatment; Draft
Guidance for Industry; Availability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.
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[[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the availability of a draft guidance for industry entitled ``Graft-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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday, 240-402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 <a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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</html>Indexed from Federal Register on September 29, 2023.
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