[Federal Register Volume 88, Number 96 (Thursday, May 18, 2023)]
[Notices]
[Pages 31766-31767]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-10611]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2005-D-0460]


Pediatric Drug Development Under the Pediatric Research Equity 
Act and the Best Pharmaceuticals for Children Act: Scientific 
Considerations; Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Pediatric Drug 
Development Under the Pediatric Research Equity Act and the Best 
Pharmaceuticals for Children Act: Scientific Considerations.'' This 
draft guidance addresses selected clinical, scientific, and ethical 
issues involved in developing drugs, including biological products, for 
pediatric use when such drug products are subject to the Pediatric 
Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children 
Act (BPCA). This draft guidance is intended to assist industry in 
obtaining the data and information necessary to support the approval of 
drug products in pediatric populations. This draft guidance does not 
address the clinical development of drug products that are not subject 
to either PREA or the BPCA.

DATES: Submit either electronic or written comments on the draft 
guidance by July 17, 2023 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2005-D-0460 for ``Pediatric Drug Development Under the Pediatric 
Research Equity Act and the Best Pharmaceuticals for Children Act: 
Scientific Considerations.'' 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 Bldg., 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

[[Page 31767]]

INFORMATION section for electronic access to the draft guidance 
document.

FOR FURTHER INFORMATION CONTACT: Rosemary Addy, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6430, Silver Spring, MD 20993-0002, 301-
796-2200, [email protected]; 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 draft guidance for industry 
entitled ``Pediatric Drug Development Under the Pediatric Research 
Equity Act and the Best Pharmaceuticals for Children Act: Scientific 
Considerations.'' This draft guidance addresses selected clinical, 
scientific, and ethical issues involved in developing drugs for 
pediatric use when such drugs are subject to PREA and/or the BPCA. This 
draft guidance, along with the draft guidance entitled ``Pediatric Drug 
Development: Regulatory Considerations--Complying With the Pediatric 
Research Equity Act and Qualifying for Pediatric Exclusivity Under the 
Best Pharmaceuticals for Children Act,'' revises and replaces the draft 
guidance entitled ``How to Comply with the Pediatric Research Equity 
Act.'' \1\ This draft guidance also addresses certain additional topics 
that FDA has not previously addressed in guidance.
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    \1\ This draft guidance also addresses certain topics previously 
addressed in the guidance for industry entitled ``Qualifying for 
Pediatric Exclusivity Under Section 505A of the Federal Food, Drug, 
and Cosmetic Act.'' That guidance was withdrawn August 7, 2013 (78 
FR 48175).
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    The purpose of this draft guidance is to assist industry in 
obtaining the data and information needed to support approval of drug 
products in pediatric populations. Specifically, this draft guidance 
describes considerations regarding data in pediatric patients with 
particular discussion regarding formulation development, nonclinical 
information, clinical pharmacology, and safety information. 
Additionally, the draft guidance discusses pediatric extrapolation, 
timing of pediatric studies, and drug development for the neonatal 
population. This draft guidance does not address the clinical 
development of drugs that are not subject to either PREA or the BPCA.
    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 ``Pediatric 
Drug Development Under the Pediatric Research Equity Act and the Best 
Pharmaceuticals for Children Act: Scientific Considerations.'' 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. The 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 parts 50 and 56 for protection of human 
subjects and institutional review boards, have been approved under OMB 
control number 0910-0130. The collections of information in 21 CFR 210 
and 211 for current good manufacturing practice have been approved 
under OMB control number 0910-0139. The collections of information in 
21 CFR part 312 for investigational new drug applications and 21 CFR 
part 314 for new drug applications and abbreviated new drug 
applications have been approved under OMB control numbers 0910-0014 and 
0910-0001, respectively. The collections of information in 21 CFR parts 
601 and 610 for biologics license applications have been approved under 
OMB control number 0910-0338. The collections of information in 42 
U.S.C. 262(k) and 21 U.S.C. 379g for biosimilar applications have been 
approved under OMB control number 0910-0718. The collections of 
information in 21 CFR 201.56 and 201.57 regarding labeling requirements 
for prescription drugs have been approved under OMB control number 
0910-0572. The collections of information in 21 CFR part 201, subpart C 
regarding over-the-counter products have been approved under OMB 
control number 0910-0340. The collections of information in 21 CFR part 
316 regarding orphan drug product development are approved under OMB 
control number 0910-0167.

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: May 15, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-10611 Filed 5-17-23; 8:45 am]
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