[Federal Register Volume 88, Number 29 (Monday, February 13, 2023)]
[Notices]
[Pages 9296-9298]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-02962]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2022-D-0112]


Considerations for Long-Term Clinical Neurodevelopmental Safety 
Studies in Neonatal Product Development; Draft Guidance for Industry; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled 
``Considerations for Long-Term Clinical Neurodevelopmental Safety 
Studies in Neonatal Product Development.'' This guidance is intended to 
provide a framework for considering whether and what type of long-term 
neurologic, sensory, and/or developmental evaluations could be useful 
in supporting a determination of safety of a regulated product for use 
in neonates, and which domains of assessment may be most pertinent. 
Although short-term safety evaluations may be acceptable for adults or 
other populations, such short-term evaluations may not identify 
important adverse events in the neonatal population, as latent effects 
may follow early-life exposures and drug treatment during the neonatal 
period coincides with a time of critical growth and physiologic 
development. Consideration of these potential long-term neurologic, 
sensory, and development effects in the neonatal population early in a 
drug development program will help ensure a safer product.

DATES: Submit either electronic or written comments on the draft 
guidance by April 14, 2023 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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'').

[[Page 9297]]

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-2022-D-0112 for ``Considerations for Long-Term Clinical 
Neurodevelopmental Safety Studies in Neonatal Product Development; 
Draft Guidance for Industry.'' 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 
An Massaro, Office of Pediatric Therapeutics, Office of Clinical Policy 
and Programs, Office of the Commissioner, Food and Drug Administration, 
10903 New Hampshire Avenue, Bldg. 32, 5th Floor, Silver Spring, MD 
20993-0002, 301-467-8507; Gerri Baer, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
32, Silver Spring, MD 20993-0002, 240-402-2865; Stephen Ripley, Center 
for Biologics Evaluation and Research, Food and Drug Administration, 
10903 New Hampshire Ave., Bldg. 71, Room 3128, Silver Spring, MD 20993-
0002, 240-402-7911; Vasum Peiris, Center for Devices and Radiological 
Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
66, Silver Spring, MD 20993-0002, 301-796-6089. 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: An Massaro, Office of Pediatric 
Therapeutics, Office of Clinical Policy and Programs, Office of the 
Commissioner, Food and Drug Administration, 10903 New Hampshire Avenue, 
Bldg. 32, 5th Floor, Silver Spring, MD 20993-0002, 301-467-8507; Gerri 
Baer, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 32, Silver Spring, MD 
20993-0002, 240-402-2865; Stephen Ripley, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002, 240-
402-7911; and Vasum Peiris, Center for Devices and Radiological Health, 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, 
Silver Spring, MD 20993-0002, 301-796-6089.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Considerations for Long-Term Clinical Neurodevelopmental 
Safety Studies in Neonatal Product Development; Draft Guidance for 
Industry.''
    Treatment with drugs, biological products, or devices, medical 
products (referred to as ``medical products'') during the neonatal 
period coincides with a time of critical growth and physiologic 
development. Although short-term safety evaluations may be acceptable 
for adults or other populations, such short-term evaluations may not 
identify important adverse events in the neonatal population, as latent 
effects may follow early-life exposures. Historically, most medical 
products used to treat neonates and young infants were not approved for 
use in these populations for the relevant indications, and thus long-
term impacts were infrequently systematically evaluated.
    Clinical investigators and sponsors of neonatal studies should 
consider and assess both the potential short- and long-term effects of 
an investigational therapy, whether novel or developed for a different 
indication. Prospectively designed long-term follow-up is helpful to 
understand medical product safety in growing and developing neonates.
    Neonates should have the same access as other populations to drugs 
and biologics that have been adequately evaluated for optimal dosing, 
efficacy, and safety. There are unique conditions that occur in term or 
preterm neonates that will not have analogous development programs in 
older populations. As products are developed for unique neonatal 
conditions, it may be useful for novel development programs and first-
in-human studies to occur in neonates, and these development programs 
should demonstrate long-term neurologic, sensory, and developmental 
safety. This guidance will discuss general, patient-specific and 
product-specific considerations ranging from neurodevelopmental 
screening through a comprehensive neurodevelopmental evaluation. It 
will also address what to measure in a risk assessment, when, and for 
how long. 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 
``Considerations for Long-Term Clinical Neurodevelopmental Safety 
Studies in Neonatal Product Development; Draft Guidance for Industry.'' 
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

[[Page 9298]]

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 for submission of investigational new drug applications, 
21 CFR part 312, have been approved under 0910-0014. The collections of 
information for submission of new drug applications, 21 CFR part 314, 
have been approved under 0910-0001. The collections of information for 
submission of biologic license applications, 21 CFR part 601, have been 
approved under 0910-0338. The collections of information for submission 
of premarket approval applications, 21 CFR part 807, subpart E; 
investigational device exemptions, 21 CFR part 812; premarket 
notifications, 21 CFR part 814, subparts A through E; humanitarian 
device exemptions, 21 CFR part 814, subpart H; and De Novo 
classification requests, 21 CFR part 860, subpart D, have been approved 
under OMB control numbers 0910-0120, 0910-0078, 0910-0231, 0910-0332, 
and 0910-0844, respectively.

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/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: February 7, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-02962 Filed 2-10-23; 8:45 am]
BILLING CODE 4164-01-P


