[Federal Register Volume 84, Number 240 (Friday, December 13, 2019)]
[Notices]
[Pages 68174-68175]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-26877]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-4751]


Food and Drug Administration Reauthorization Act Implementation 
Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs; 
Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``FDARA 
Implementation Guidance for Pediatric Studies of Molecularly Targeted 
Oncology Drugs.'' This draft guidance addresses early planning for 
pediatric evaluation of certain molecularly targeted oncology drugs, 
including biological products, for which original new drug applications 
(NDAs) and biologics license applications (BLAs) are expected to be 
submitted to FDA on or after August 18, 2020, in accordance with the 
provisions of the Federal Food, Drug, and Cosmetic Act (FD&C Act) as 
amended by the FDA Reauthorization Act of 2017 (FDARA). This guidance 
addresses the implementation of amendments made by FDARA to the FD&C 
Act regarding molecularly targeted oncology drugs.

DATES: Submit either electronic or written comments on the draft 
guidance by February 11, 2020 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2019-D-4751 for ``FDARA Implementation Guidance for Pediatric 
Studies of Molecularly Targeted Oncology Drugs.'' 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.
     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

[[Page 68175]]

``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.gpo.gov/fdsys/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.
    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 Office of Communication, Outreach and Development, 
Center for Biologics Evaluation and Research (CBER), Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver 
Spring, MD 20993-0002; Division of Drug Information, Center for Drug 
Evaluation and Research (CDER), Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993-0002. Send one self-addressed adhesive label to assist that 
office in processing your requests. The draft guidance may also be 
obtained by mail by calling CBER at 1-800-835-4709 or 240-402-8010. See 
the SUPPLEMENTARY INFORMATION section for electronic access to the 
draft guidance document.

FOR FURTHER INFORMATION CONTACT: Gregory Reaman, Oncology Center of 
Excellence, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 22, Rm. 2202, Silver Spring, MD 20993-0002, 301-796-0785; or 
Stephen Ripley, Center for Biologics Evaluation and Research, Food and 
Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, 
Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``FDARA Implementation Guidance for Pediatric Studies of 
Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the 
FD&C Act.'' This draft guidance addresses early planning for pediatric 
evaluation of certain molecularly targeted oncology drugs (including 
biological products) for which original NDAs and BLAs are expected to 
be submitted to FDA on or after August 18, 2020, in accordance with the 
provisions of section 505B of the FD&C Act. Section 505B of the FD&C 
Act (21 U.S.C. 355c) (also referred to as the Pediatric Research Equity 
Act or PREA (Pub. L. 108-155)), was amended by FDARA.
    The amendments provide a new mechanism to expedite the evaluation 
of certain novel drugs with the potential to address an unmet medical 
need of pediatric patients with cancer. Specifically, FDARA amended the 
requirement for pediatric investigations of certain new targeted cancer 
drugs to be based on molecular mechanism of action rather than clinical 
indication. For original NDAs and BLAs submitted on or after August 18, 
2020, if the application is for a new active ingredient, and the drug 
or biological product that is the subject of the application is 
intended for treatment of an adult cancer and directed at a molecular 
target FDA determines to be substantially relevant to the growth or 
progression of a pediatric cancer, reports of molecularly targeted 
pediatric cancer investigations must be submitted with the marketing 
application, unless the required investigations are waived or deferred 
(section 505B(a)(1)(B) of the FD&C Act).
    This draft guidance provides recommendations on regulatory 
considerations related to the amendments to section 505B of the FD&C 
Act, including information on molecular targets, factors FDA intends to 
consider in the determination of whether a molecular target is 
substantially relevant to the growth or progression of a pediatric 
cancer, the molecular target lists, content of the initial pediatric 
study plan and description of recommended studies, additional 
considerations for rare cancers, and considerations for planned waivers 
and deferrals. In addition, the draft guidance includes information 
regarding global implications and international collaboration.
    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 ``FDARA 
Implementation Guidance for Pediatric Studies of Molecularly Targeted 
Oncology Drugs.'' 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

    This draft guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521). The 
collections of information in 21 CFR part 314 have been approved under 
OMB control number 0910-0001. The collections of information in 21 CFR 
part 312 have been approved under OMB control numbers 0910-0014. The 
collections of information in 21 CFR part 601 have been approved under 
OMB control number 0910-0338.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, or 
https://www.regulations.gov.

    Dated: December 9, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-26877 Filed 12-12-19; 8:45 am]
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