Notice2023-02962
Considerations for Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development; Draft Guidance for Industry; Availability
Primary source
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Published
February 13, 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Considerations for Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development."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.
Full Text
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<title>Federal Register, Volume 88 Issue 29 (Monday, February 13, 2023)</title>
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[Federal Register Volume 88, Number 29 (Monday, February 13, 2023)]
[Notices]
[Pages 9296-9298]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-02962]
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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Studies in Neonatal Product Development; Draft Guidance for Industry;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.
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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the availability of a draft guidance for industry entitled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:
<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'').
[[Page 9297]]
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-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 <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
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 <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/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: 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
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