Notice2023-14691
Inborn Errors of Metabolism That Use Dietary Management: Considerations for Optimizing and Standardizing Diet in Clinical Trials for Drug Product Development; Draft Guidance for Industry; Availability
Primary source
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Published
July 12, 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a revised draft guidance for industry entitled "Inborn Errors of Metabolism That Use Dietary Management: Considerations for Optimizing and Standardizing Diet in Clinical Trials for Drug Product Development." This draft guidance describes FDA's current recommendations for optimizing and standardizing dietary management in clinical trials for the development of drug products intended to treat inborn errors of metabolism (IEM) when dietary management is a key component of patients' metabolic control. Optimizing and standardizing dietary management in these patients before they enter clinical trials and during clinical trials is essential to providing an accurate evaluation of the efficacy of new drug products. This guidance revises the draft guidance of the same name issued on July 24, 2018.
Full Text
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<title>Federal Register, Volume 88 Issue 132 (Wednesday, July 12, 2023)</title>
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[Federal Register Volume 88, Number 132 (Wednesday, July 12, 2023)]
[Notices]
[Pages 44363-44365]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-14691]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-2647]
Inborn Errors of Metabolism That Use Dietary Management:
Considerations for Optimizing and Standardizing Diet in Clinical Trials
for Drug Product Development; Draft Guidance for Industry; Availability
AGENCY: Food and Drug Administration, Department of Health and Human
Services (HHS).
ACTION: Notice of availability.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a revised draft guidance for industry entitled
``Inborn Errors of Metabolism That Use Dietary Management:
Considerations for Optimizing and Standardizing Diet in Clinical Trials
for Drug Product Development.'' This draft guidance describes FDA's
current recommendations for optimizing and standardizing dietary
management in clinical trials for the development of drug products
intended to treat inborn errors of metabolism (IEM) when dietary
management is a key component of patients' metabolic control.
Optimizing
[[Page 44364]]
and standardizing dietary management in these patients before they
enter clinical trials and during clinical trials is essential to
providing an accurate evaluation of the efficacy of new drug products.
This guidance revises the draft guidance of the same name issued on
July 24, 2018.
DATES: Submit either electronic or written comments on the draft
guidance by September 11, 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-2018-D-2647 for ``Inborn Errors of Metabolism That Use Dietary
Management: Considerations for Optimizing and Standardizing Diet in
Clinical Trials for Drug Product Development.'' 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; or the
Office of Communication, Outreach, and Development, Center for
Biologics Evaluation and Research, 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 that office in
processing your requests. See the SUPPLEMENTARY INFORMATION section for
electronic access to the draft guidance document.
FOR FURTHER INFORMATION CONTACT: Robert Temple, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6304, Silver Spring, MD 20993, 301-796-
1153; or Diane Maloney, 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 revised draft guidance for
industry entitled ``Inborn Errors of Metabolism That Use Dietary
Management: Considerations for Optimizing and Standardizing Diet in
Clinical Trials for Drug Product Development.'' This draft guidance
describes FDA's current recommendations for optimizing and
standardizing dietary management in clinical trials for the development
of drug products intended to treat IEM when dietary management is a key
component of patients' metabolic control. Optimizing and standardizing
dietary management in these patients before they enter into clinical
trials and during clinical trials is essential to providing an accurate
evaluation of the efficacy of new drug products.
This draft guidance revises the draft guidance of the same name
issued on July 24, 2018 (83 FR 35006). FDA considered comments received
on the draft guidance and is reissuing the guidance in draft form.
Revisions from the previous draft include: (1) clarification that drug
products, including both small molecules and biological products,
should be studied in conjunction with dietary management for conditions
where dietary management is the current standard of clinical care; (2)
clarification that metabolic control may be evaluated by biochemical
analytes and clinical assessment as substantiated by current clinical
standards of care; (3) clarification that differences in baseline
dietary management standards among patients from different countries
should be considered and clearly stated in the protocol; and (4)
clarification that the
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most informative design is a randomized, double-blind clinical trial
that includes a concurrent control group (approved drug or placebo).
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 ``Inborn
Errors of Metabolism That Use Dietary Management: Considerations for
Optimizing and Standardizing Diet in Clinical Trials for Drug Product
Development.'' 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. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. The previously approved collections of
information are subject to review by OMB under the PRA. The collections
of information in 21 CFR part 312 have been approved under OMB Control
No. 0910-0014.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
either <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: July 6, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-14691 Filed 7-11-23; 8:45 am]
BILLING CODE 4164-01-P
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