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Notice2022-22616

Tissue Agnostic Drug Development in Oncology; Draft Guidance for Industry; Availability

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Published
October 18, 2022

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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Tissue Agnostic Drug Development in Oncology." For the purpose of this guidance, the term "tissue agnostic oncology drug" refers to a drug that targets a specific molecular alteration(s) (a kind of biomarker) across multiple cancer types as defined, for example by organ, tissue, or tumor type. This draft guidance describes the development of tissue agnostic drugs, scientific considerations in determining when tissue agnostic oncology drug development may be appropriate, and, if appropriate, issues to be addressed during such development. Tissue agnostic drug development may expedite or enable the development of new therapies for patients with rare cancer types.

Full Text

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<title>Federal Register, Volume 87 Issue 200 (Tuesday, October 18, 2022)</title>
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[Federal Register Volume 87, Number 200 (Tuesday, October 18, 2022)]
[Notices]
[Pages 63079-63080]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-22616]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2022-D-0286]


Tissue Agnostic Drug Development in Oncology; Draft Guidance for 
Industry; Availability

AGENCY: Food and Drug Administration, Department of Health and Human 
Services (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the availability of a draft guidance for industry entitled ``Tissue 
Agnostic Drug Development in Oncology.'' For the purpose of this 
guidance, the term ``tissue agnostic oncology drug'' refers to a drug 
that targets a specific molecular alteration(s) (a kind of biomarker) 
across multiple cancer types as defined, for example by organ, tissue, 
or tumor type. This draft guidance describes the development of tissue 
agnostic drugs, scientific considerations in determining when tissue 
agnostic oncology drug development may be appropriate, and, if 
appropriate, issues to be addressed during such development. Tissue 
agnostic drug development may expedite or enable the development of new 
therapies for patients with rare cancer types.

DATES: Submit either electronic or written comments on the draft 
guidance by December 19, 2022 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-2022-D-0286 for ``Tissue Agnostic Drug Development in Oncology.'' 
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

[[Page 63080]]

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 this 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. 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: Steven Lemery, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2374, Silver Spring, MD 20993-0002, 301-
796-2276; 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 ``Tissue Agnostic Drug Development in Oncology.'' This draft 
guidance provides recommendations to sponsors regarding considerations 
for tissue agnostic drug development in oncology. For the purpose of 
this guidance, the term ``tissue agnostic oncology drug'' refers to a 
drug that targets a specific molecular alteration(s) (a kind of 
biomarker) across multiple cancer types as defined, for example by 
organ, tissue, or tumor type. A tissue agnostic oncology drug can 
therefore be used to treat multiple types of cancer (e.g., colorectal, 
thyroid, and breast cancers) with the targeted molecular alteration 
(e.g., either the same targeted molecular alteration or targeted 
molecular alterations affecting a single pathway). The guidance 
discusses the need in tissue agnostic drug development to generalize 
treatment effects based on data observed in some cancer types to other 
cancer types with the same targeted molecular alteration, when no 
subjects (or a limited number of subjects) with the other cancer types 
were included in the clinical trial(s). Such an approach may expedite 
or enable the development of new therapies for patients with rare 
cancer types when it may not be feasible to test the drug in an 
adequate number of subjects for every cancer type.
    The draft guidance describes factors that sponsors should consider 
when determining whether a tissue agnostic oncology drug development 
program may be scientifically and clinically appropriate, such as 
biology, subject population, clinical pharmacology, and clinical safety 
and efficacy. In addition, the draft guidance describes issues to be 
addressed in a tissue agnostic drug development program, such as 
nonclinical assessment, subject selection, study designs, statistical 
considerations, endpoints, pediatrics, diagnostic considerations, 
postapproval data and information, and labeling.
    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 ``Tissue 
Agnostic Drug Development in Oncology.'' 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 have been approved under OMB control 
number 0910-0014. 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 601 have been approved under OMB control 
number 0910-0338. The collections of information in 21 CFR 201.56 and 
201.57 have been approved under OMB control number 0910-0572.

III. Electronic Access

    Persons with access to the internet may obtain an electronic 
version of 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: October 11, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-22616 Filed 10-17-22; 8:45 am]
BILLING CODE 4164-01-P


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