[Federal Register Volume 88, Number 132 (Wednesday, July 12, 2023)]
[Notices]
[Pages 44363-44365]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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for Drug Product Development; Draft Guidance for Industry; Availability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the availability of a revised draft guidance for industry entitled 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 This draft guidance describes FDA's 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This guidance revises the draft guidance of the same name issued on 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DATES: Submit either electronic or written comments on the draft 
guidance by September 11, 2023, to ensure that the Agency considers 
your comment on this draft guidance before it begins work on the final 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:

     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-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 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; 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

[[Page 44365]]

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 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: 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