Notice2023-10610
Pediatric Drug Development: Regulatory Considerations-Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act; Draft Guidance for Industry; Availability
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Published
May 18, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled "Pediatric Drug Development: Regulatory Considerations--Complying With the Pediatric Research Equity Act and Qualifying for Pediatric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." This draft guidance, when finalized, is intended to provide recommendations to industry on complying with the pediatric study requirements under the Pediatric Research Equity Act (PREA), and to describe the process for qualifying for pediatric exclusivity and the protections that pediatric exclusivity offers under the Best Pharmaceuticals for Children Act (BPCA). Combining discussion of PREA and the BPCA together in regulatory guidance emphasizes the sponsor's need to consider both laws when developing pediatric drugs and biological products.
Full Text
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<title>Federal Register, Volume 88 Issue 96 (Thursday, May 18, 2023)</title>
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[Federal Register Volume 88, Number 96 (Thursday, May 18, 2023)]
[Notices]
[Pages 31764-31766]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-10610]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2005-D-0460]
Pediatric Drug Development: Regulatory Considerations--Complying
With the Pediatric Research Equity Act and Qualifying for Pediatric
Exclusivity Under the Best Pharmaceuticals for Children Act; Draft
Guidance for Industry; Availability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availability of a draft guidance for industry entitled ``Pediatric Drug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: Regulatory Considerations--Complying With the Pediatric
Research Equity Act and Qualifying for Pediatric Exclusivity Under the
Best Pharmaceuticals for Children Act.'' This draft guidance, when
finalized, is intended to provide recommendations to industry on
complying with the pediatric study requirements under the Pediatric
Research Equity Act (PREA), and to describe the process for qualifying
for pediatric exclusivity and the protections that pediatric
exclusivity offers under the Best Pharmaceuticals for Children Act
(BPCA). Combining discussion of PREA and the BPCA together in
regulatory guidance emphasizes the sponsor's need to consider both laws
when developing pediatric drugs and biological products.
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:
<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'').
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-2005-D-0460 for ``Pediatric Drug Development: Regulatory
Considerations--Complying With the Pediatric Research Equity Act and
Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for
Children Act.'' 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.
[[Page 31765]]
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: 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-
796-1640, <a href="/cdn-cgi/l/email-protection#0f7f6a6b7c6b7d7a687c4f696b6e2167677c21686079"><span class="__cf_email__" data-cfemail="bacadfdec9dec8cfddc9fadcdedb94d2d2c994ddd5cc">[email protected]</span></a>; 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: Regulatory Considerations--
Complying With the Pediatric Research Equity Act and Qualifying for
Pediatric Exclusivity Under the Best Pharmaceuticals for Children
Act.'' This draft guidance is intended to provide recommendations on
how to comply with the pediatric study requirements under sections 505B
of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355c)
(PREA), and to qualify for pediatric exclusivity under section 505A of
the FD&C Act (21 U.S.C. 355a) (BPCA). This guidance also incorporates
recommendations based on FDA's Retrospective Review.\1\
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\1\ This review was conducted pursuant to section 505B(f)(5) of
the FD&C Act and is described in more detail in a report available
at <a href="https://www.fda.gov/media/78050/download">https://www.fda.gov/media/78050/download</a>.
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PREA requires that certain applications (or supplements to
applications) submitted under section 505 of the FD&C Act (21 U.S.C.
355) or under section 351 of the Public Health Service Act (42 U.S.C.
262) (specifically, any application that is subject to PREA) must
either include pediatric assessments or reports on the molecularly
targeted pediatric cancer investigation (as appropriate), or a request
for waiver and/or deferral (see section 505B(a)(1), (a)(4), and (a)(5)
of the FD&C Act). To ensure that sponsors thoroughly consider a
pediatric clinical development program earlier in their overall
clinical development program, PREA requires sponsors to submit an
initial pediatric study plan during the investigational phase of
development (see section 505B(e) of the FD&C Act). PREA also authorizes
FDA to require holders of approved applications for drugs and
biological products, who are not seeking approval for one of the
changes specified, to submit pediatric assessments under certain
circumstances (see section 505B(b) of the FD&C Act).
Under the BPCA, certain applications may qualify for 6 months of
exclusivity if the following conditions are met: (1) FDA determines
that information relating to the use of a drug in the pediatric
population may produce health benefits in that population; (2) FDA
issues a written request (WR) for studies of that drug in pediatric
populations and the applicant agrees to the request; (3) the studies
are completed using appropriate formulations for each age group and
within the requested time; and (4) the reports of the studies are
submitted and accepted by FDA (see section 505A(b)(1) and (c)(1) of the
FD&C Act). In accepting or rejecting the reports, FDA determines
whether the studies fairly respond to the WR, have been reported in
accordance with filing requirements, and otherwise qualify for
pediatric exclusivity (see section 505A(d)(4) of the FD&C Act).
With respect to content, this draft guidance addresses pediatric
assessments, molecularly targeted pediatric cancer investigations,
pediatric study plans, waivers and deferrals (including deferral
extensions), labeling considerations, the noncompliance process, the
relationship of the PREA requirements to pediatric exclusivity, and the
reporting of adverse events for products subject to PREA and the BPCA.
Additionally, the draft guidance includes a description of the
mechanisms FDA uses to obtain pediatric studies, how industry can
obtain a WR and what it includes, how study reports should be submitted
to FDA for filing, the criteria to qualify for pediatric exclusivity,
the nature and scope of pediatric exclusivity, and the information that
should be submitted in support of a request for a pediatric exclusivity
determination.
With respect to its discussion of PREA, this guidance, along with
the draft guidance for industry entitled ``Pediatric Drug Development
Under the Pediatric Research Equity Act and the Best Pharmaceuticals
for Children Act: Scientific Considerations,'' revises and replaces the
draft guidance for industry entitled ``How to Comply With the Pediatric
Research Equity Act'' (2005 draft guidance; 70 FR 53233, September 7,
2005).\2\ In addition to addressing certain PREA-related topics covered
in the 2005 draft guidance, this draft guidance also addresses certain
changes to PREA that have occurred since 2005.
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\2\ This 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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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: Regulatory Considerations--Complying With the
Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity
Under the Best Pharmaceuticals for Children Act.'' 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 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 pertaining to biologics license
applications have been approved under OMB control number 0910-0338. The
collections of information in 42 U.S.C. 262(k) 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
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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 have been approved under OMB control number 0910-
0167.
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/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</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: May 15, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-10610 Filed 5-17-23; 8:45 am]
BILLING CODE 4164-01-P
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