FDA Reauthorization Act Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs; Guidance for Industry; Availability, 28113-28115 [2021-11022]

Download as PDF jbell on DSKJLSW7X2PROD with NOTICES Federal Register / Vol. 86, No. 99 / Tuesday, May 25, 2021 / Notices listed drug (§ 314.161 (21 CFR 314.161)). FDA may not approve an ANDA that does not refer to a listed drug. MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, is the subject of NDA 019228, held by Abraxis Pharmaceutical Products, and initially approved on May 5, 1987. MANGANESE SULFATE is indicated for use as a supplement to intravenous solutions given for total parenteral nutrition. Administration helps to maintain manganese serum levels and to prevent depletion of endogenous stores and subsequent deficiency symptoms. MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, is currently listed in the ‘‘Discontinued Drug Product List’’ section of the Orange Book. Fresenius Kabi USA, LLC, submitted a citizen petition dated October 4, 2020 (Docket No. FDA–2020–P–2048), under 21 CFR 10.30, requesting that the Agency determine whether MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, was withdrawn from sale for reasons of safety or effectiveness. After considering the citizen petition and reviewing Agency records and based on the information we have at this time, FDA has determined under § 314.161 that MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, was not withdrawn for reasons of safety or effectiveness. The petitioner has identified no data or other information suggesting that MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, was withdrawn for reasons of safety or effectiveness. We have carefully reviewed our files for records concerning the withdrawal of MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, from sale. We have also independently evaluated relevant literature and data for possible postmarketing adverse events. We have found no information that would indicate that this drug product was withdrawn from sale for reasons of safety or effectiveness. Accordingly, the Agency will continue to list MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, in the ‘‘Discontinued Drug Product List’’ section of the Orange Book. The ‘‘Discontinued Drug Product List’’ delineates, among other items, drug products that have been discontinued from marketing for reasons other than safety or effectiveness. ANDAs that refer to MANGANESE SULFATE, injectable, Eq 0.1 mg manganese/mL, may be approved by the Agency as long as they meet all other legal and regulatory requirements for the approval of ANDAs. If FDA VerDate Sep<11>2014 18:09 May 24, 2021 Jkt 253001 determines that labeling for this drug product should be revised to meet current standards, the Agency will advise ANDA applicants to submit such labeling. Dated: May 18, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–11021 Filed 5–24–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2019–D–4751] FDA Reauthorization Act Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled ‘‘FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs.’’ This 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, 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). Early pediatric evaluation of certain molecularly targeted oncology drugs as required by the FD&C Act is expected to accelerate the creation of an informed pediatric development plan and ultimately the development of promising drugs for pediatric patients. This guidance finalizes the draft guidance entitled ‘‘FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs’’ issued on December 13, 2019, and finalizes certain material related to implementation of FDARA that was included in the draft guidance entitled ‘‘Pediatric Study Plans for Oncology Drugs: Questions and Answers’’ issued on January 16, 2020. Accordingly, FDA does not intend to finalize the draft guidance entitled ‘‘Pediatric Study Plans for Oncology SUMMARY: PO 00000 Frm 00061 Fmt 4703 Sfmt 4703 28113 Drugs: Questions and Answers,’’ which is now withdrawn. DATES: The announcement of the guidance is published in the Federal Register on May 25, 2021. 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–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 E:\FR\FM\25MYN1.SGM 25MYN1 jbell on DSKJLSW7X2PROD with NOTICES 28114 Federal Register / Vol. 86, No. 99 / Tuesday, May 25, 2021 / Notices 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-201509-18/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 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. The guidance may also be obtained by mail by calling CBER at VerDate Sep<11>2014 18:09 May 24, 2021 Jkt 253001 1–800–835–4709 or 240–402–8010. See the SUPPLEMENTARY INFORMATION section for electronic access to the 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, Gregory.Reaman@fda.hhs.gov; 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, Stephen.Ripley@fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a guidance for industry entitled ‘‘FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs.’’ This 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, in accordance with the provisions of section 505B of the FD&C Act (21 U.S.C. 355c). Section 505B of the FD&C Act (also referred to as the Pediatric Research Equity Act or PREA) was amended by FDARA. The amendments to section 505B require pediatric evaluation of certain molecularly targeted oncology drugs, with the goal of helping address the needs of pediatric patients with cancer. FDARA amended section 505B of the FD&C Act to require—for original applications submitted on or after August 18, 2020—pediatric investigations of certain targeted cancer drugs with new active ingredients, based on molecular mechanism of action rather than clinical indication. Specifically, if an original NDA or BLA is submitted on or after August 18, 2020, 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 on the molecularly targeted pediatric cancer investigation required under section 505B(a)(3) of the FD&C Act must be submitted with the marketing application, unless the requirement is waived or deferred (sections 505B(a)(1)(B) and (a)(3)(C) of the FD&C Act). PO 00000 Frm 00062 Fmt 4703 Sfmt 4703 This 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, information regarding the molecular target lists, recommendations on the content of the initial pediatric study plan and description of recommended study(ies), additional considerations for rare cancers, information pertaining to oncology drug combination regimens, and considerations regarding planned waivers and deferrals. In addition, the guidance includes information regarding global implications of, and the importance of international collaboration regarding, pediatric oncology studies. This guidance finalizes the draft guidance entitled ‘‘FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs,’’ issued on December 13, 2019 (see 84 FR 68174). This guidance also finalizes certain recommendations related to implementation of FDARA section 504 that was included in the draft guidance entitled ‘‘Pediatric Study Plans for Oncology Drugs: Transitional Information Until Full Implementation of FDARA Section 504 Questions and Answers,’’ issued on January 16, 2020 (85 FR 2746). FDA considered comments received on both of these draft guidances as this guidance was finalized. Changes from the draft to the final guidance include the following: Describing additional safeguards for children in clinical investigations (see 21 CFR part 50, subpart D), providing information sources that have been used in the development of the molecular target lists, describing information that should be included in a request for a meeting regarding early advice on pediatric development for oncology projects subject to the amended provisions, and providing additional information and clarification regarding planned waivers and deferrals. Recommendations that were finalized in this guidance from the draft guidance entitled ‘‘Pediatric Study Plans for Oncology Drugs: Transitional Information Until Full Implementation of FDARA Section 504 Questions and Answers’’ include the following: Clarifying that a supplemental application does not trigger the requirement to submit reports on the molecularly targeted pediatric cancer E:\FR\FM\25MYN1.SGM 25MYN1 jbell on DSKJLSW7X2PROD with NOTICES Federal Register / Vol. 86, No. 99 / Tuesday, May 25, 2021 / Notices investigation and describing considerations for initial pediatric study plans for oncology drug combination regimens. 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 ‘‘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. Dated: May 18, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. 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 parts 50 and 56 have been approved under OMB control number 0910–0130; 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 number 0910–0014; the collections of information in 21 CFR part 601 have been approved under OMB control number 0910–0338; the collections of information in FDA’s draft guidance for industry entitled ‘‘Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products’’ have been approved under OMB control number 0910–0429; and the collections of information pertaining to submission of a biologics license application under section 351(k) of the Public Health Service Act and the draft guidance for industry entitled ‘‘Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products’’ have been approved under OMB control number 0910–0719. AGENCY: III. Electronic Access Persons with access to the internet may obtain the guidance at either https://www.fda.gov/drugs/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, or https:// www.regulations.gov. VerDate Sep<11>2014 18:09 May 24, 2021 Jkt 253001 [FR Doc. 2021–11022 Filed 5–24–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2019–D–0621] Bispecific Antibody Development Programs; Guidance for Industry; Availability Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled ‘‘Bispecific Antibody Development Programs.’’ This guidance provides recommendations to assist industry and other parties involved in the development of bispecific antibodies. The guidance focuses on general regulatory and scientific considerations for bispecific antibodies, but not on development of a particular bispecific antibody. This guidance finalizes the draft guidance of the same title issued on April 19, 2019. DATES: The announcement of the guidance is published in the Federal Register on May 25, 2021. ADDRESSES: You may submit either electronic or written comments on Agency guidances at any time as follows: SUMMARY: 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 PO 00000 Frm 00063 Fmt 4703 Sfmt 4703 28115 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–0621 for ‘‘Bispecific Antibody Development Programs.’’ 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 E:\FR\FM\25MYN1.SGM 25MYN1

Agencies

[Federal Register Volume 86, Number 99 (Tuesday, May 25, 2021)]
[Notices]
[Pages 28113-28115]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-11022]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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


FDA Reauthorization Act Implementation Guidance for Pediatric 
Studies of Molecularly Targeted Oncology Drugs; 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 final guidance for industry entitled ``FDARA 
Implementation Guidance for Pediatric Studies of Molecularly Targeted 
Oncology Drugs.'' This 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, 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). Early pediatric evaluation of certain molecularly 
targeted oncology drugs as required by the FD&C Act is expected to 
accelerate the creation of an informed pediatric development plan and 
ultimately the development of promising drugs for pediatric patients. 
This guidance finalizes the draft guidance entitled ``FDARA 
Implementation Guidance for Pediatric Studies of Molecularly Targeted 
Oncology Drugs'' issued on December 13, 2019, and finalizes certain 
material related to implementation of FDARA that was included in the 
draft guidance entitled ``Pediatric Study Plans for Oncology Drugs: 
Questions and Answers'' issued on January 16, 2020. Accordingly, FDA 
does not intend to finalize the draft guidance entitled ``Pediatric 
Study Plans for Oncology Drugs: Questions and Answers,'' which is now 
withdrawn.

DATES: The announcement of the guidance is published in the Federal 
Register on May 25, 2021.

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-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

[[Page 28114]]

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 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. The 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 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, 
[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 20993-0002, 240-
402-7911, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``FDARA Implementation Guidance for Pediatric Studies of 
Molecularly Targeted Oncology Drugs.'' This 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, in accordance with the 
provisions of section 505B of the FD&C Act (21 U.S.C. 355c). Section 
505B of the FD&C Act (also referred to as the Pediatric Research Equity 
Act or PREA) was amended by FDARA.
    The amendments to section 505B require pediatric evaluation of 
certain molecularly targeted oncology drugs, with the goal of helping 
address the needs of pediatric patients with cancer. FDARA amended 
section 505B of the FD&C Act to require--for original applications 
submitted on or after August 18, 2020--pediatric investigations of 
certain targeted cancer drugs with new active ingredients, based on 
molecular mechanism of action rather than clinical indication. 
Specifically, if an original NDA or BLA is submitted on or after August 
18, 2020, 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 on the molecularly targeted pediatric 
cancer investigation required under section 505B(a)(3) of the FD&C Act 
must be submitted with the marketing application, unless the 
requirement is waived or deferred (sections 505B(a)(1)(B) and (a)(3)(C) 
of the FD&C Act).
    This 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, 
information regarding the molecular target lists, recommendations on 
the content of the initial pediatric study plan and description of 
recommended study(ies), additional considerations for rare cancers, 
information pertaining to oncology drug combination regimens, and 
considerations regarding planned waivers and deferrals. In addition, 
the guidance includes information regarding global implications of, and 
the importance of international collaboration regarding, pediatric 
oncology studies.
    This guidance finalizes the draft guidance entitled ``FDARA 
Implementation Guidance for Pediatric Studies of Molecularly Targeted 
Oncology Drugs,'' issued on December 13, 2019 (see 84 FR 68174). This 
guidance also finalizes certain recommendations related to 
implementation of FDARA section 504 that was included in the draft 
guidance entitled ``Pediatric Study Plans for Oncology Drugs: 
Transitional Information Until Full Implementation of FDARA Section 504 
Questions and Answers,'' issued on January 16, 2020 (85 FR 2746). FDA 
considered comments received on both of these draft guidances as this 
guidance was finalized. Changes from the draft to the final guidance 
include the following: Describing additional safeguards for children in 
clinical investigations (see 21 CFR part 50, subpart D), providing 
information sources that have been used in the development of the 
molecular target lists, describing information that should be included 
in a request for a meeting regarding early advice on pediatric 
development for oncology projects subject to the amended provisions, 
and providing additional information and clarification regarding 
planned waivers and deferrals. Recommendations that were finalized in 
this guidance from the draft guidance entitled ``Pediatric Study Plans 
for Oncology Drugs: Transitional Information Until Full Implementation 
of FDARA Section 504 Questions and Answers'' include the following: 
Clarifying that a supplemental application does not trigger the 
requirement to submit reports on the molecularly targeted pediatric 
cancer

[[Page 28115]]

investigation and describing considerations for initial pediatric study 
plans for oncology drug combination regimens. 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 ``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

    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 parts 50 and 56 have been approved under OMB 
control number 0910-0130; 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 number 0910-0014; the collections of information in 21 CFR 
part 601 have been approved under OMB control number 0910-0338; the 
collections of information in FDA's draft guidance for industry 
entitled ``Formal Meetings Between the FDA and Sponsors or Applicants 
of PDUFA Products'' have been approved under OMB control number 0910-
0429; and the collections of information pertaining to submission of a 
biologics license application under section 351(k) of the Public Health 
Service Act and the draft guidance for industry entitled ``Formal 
Meetings Between the FDA and Sponsors or Applicants of BsUFA Products'' 
have been approved under OMB control number 0910-0719.

III. Electronic Access

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

    Dated: May 18, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-11022 Filed 5-24-21; 8:45 am]
BILLING CODE 4164-01-P

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