[Federal Register Volume 87, Number 143 (Wednesday, July 27, 2022)]
[Notices]
[Pages 45110-45111]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-16076]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-3132]


General Clinical Pharmacology Considerations for Neonatal Studies 
for Drugs and Biological Products; 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 or Agency) is announcing 
the availability of a final guidance for industry entitled ``General 
Clinical Pharmacology Considerations for Neonatal Studies for Drugs and 
Biological Products.'' This guidance is intended to assist sponsors of 
investigational new drug applications (INDs) and applicants of new drug 
applications (NDAs), biologics license applications (BLAs), and 
supplements to such applications who are planning to conduct clinical 
studies in neonatal populations. This guidance finalizes the draft 
guidance of the same title issued on August 1, 2019.

DATES: The announcement of the guidance is published in the Federal 
Register on July 27, 2022.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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-2019-D-3132 for ``General Clinical Pharmacology Considerations for 
Neonatal Studies for Drugs and Biological Products.'' 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 this 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 to 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 guidance document.

FOR FURTHER INFORMATION CONTACT: Elimika Pfuma Fletcher, Center for 
Drug Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 2162, Silver Spring, MD 20993, 301-796-
3473, [email protected] or Stephen Ripley, Center for 
Biologics Evaluation and Research, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993, 240-
402-7911, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a final guidance for industry 
``General Clinical Pharmacology Considerations for Neonatal Studies for 
Drugs and Biological Products.'' This guidance is intended to assist 
sponsors of INDs and applicants of NDAs, BLAs, and supplements to such 
applications who are planning to conduct clinical studies in neonatal 
populations.
    In 2012, the Best Pharmaceuticals for Children Act (Pub. L. 107-
109) (BPCA)

[[Page 45111]]

and the Pediatric Research Equity Act (Pub. L. 108-155) were made 
permanent under Title V of the Food and Drug Administration Safety and 
Innovation Act (Pub. L. 112-144) (FDASIA). FDASIA requires that all 
BPCA requests for pediatric drug studies include a rationale for not 
including neonatal studies if none are requested.
    Given that most drugs used in neonatal intensive care units (NICUs) 
are used in an off-label capacity, it is important that drug 
information be obtained in neonates to address gaps in neonatal 
labeling. In addition, therapies need to be developed for conditions 
unique to neonates. New approaches to the study of drugs in neonates 
should consider the diversity of the patient population and underlying 
conditions that are cared for in NICUs. Therefore, this guidance 
addresses subgroup classifications of neonates; general 
pharmacokinetic, pharmacodynamic, and pharmacogenomic considerations 
for clinical pharmacology studies in neonates; and clinical 
pharmacology considerations for planned studies in neonates.
    This guidance finalizes the draft guidance entitled ``General 
Clinical Pharmacology Considerations for Neonatal Studies for Drugs and 
Biological Products'' issued on August 1, 2019 (84 FR 37653). FDA 
considered comments received on the draft guidance as the guidance was 
finalized. Changes from the draft to the final guidance include the 
addition of a section addressing immunogenicity, additional text 
regarding consideration of the total volume administered to neonates, 
and additional clarity regarding the use of microsampling methodology. 
In addition, editorial changes were made to improve clarity.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``General Clinical Pharmacology 
Considerations for Neonatal Studies for Drugs and Biological 
Products.'' 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 
refers to previously approved FDA collections of information. 
Therefore, clearance by the Office of Management and Budget (OMB) under 
the Paperwork Reduction Act of 1995 (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 related to institutional review boards in 21 CFR part 56 
have been approved under OMB control number 0910-0130. The collections 
of information in 21 CFR part 314 for the submission of new drug 
applications have been approved under OMB control number 0910-0001. The 
collections of information in 21 CFR part 601 for the submission of 
biologics license applications have been approved under OMB control 
number 0910-0338. The collections of information 21 CFR part 312 for 
the submission of investigational new drug applications have been 
approved under OMB control number 0910-0014. The collections of 
information in Sec. Sec.  312.47 and 312.82 for requesting meetings 
with FDA about drug development programs have been approved under OMB 
control number 0910-0429. The collections of information for the 
submission of prescription drug labeling in 21 CFR 201.56 and 21 CFR 
201.57 have been approved under OMB control number 0910-0572.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, https://www.regulations.gov, or https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances.

    Dated: July 21, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-16076 Filed 7-26-22; 8:45 am]
BILLING CODE 4164-01-P