[Federal Register Volume 85, Number 167 (Thursday, August 27, 2020)]
[Notices]
[Pages 53007-53008]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-18894]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-0938]


Evaluating Cancer Drugs in Patients With Central Nervous System 
Metastases; Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Evaluating 
Cancer Drugs in Patients with Central Nervous System Metastases.'' This 
draft guidance document provides recommendations regarding the design 
of clinical trials of drugs and biological products regulated by the 
Center for Drug Evaluation and Research (CDER) and the Center for 
Biologics Evaluation and Research (CBER) that are intended to support 
product labeling describing the antitumor activity in patients with 
central nervous system (CNS) metastases from solid tumors originating 
outside the CNS. The draft guidance includes study design 
recommendations regarding the patient population, available therapy, 
prior therapies, assessment of CNS disease, study endpoints, and 
leptomeningeal disease.

DATES: Submit either electronic or written comments on the draft 
guidance by October 26, 2020, to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2020-D-0938 for ``Evaluating Cancer Drugs in Patients with Central 
Nervous System Metastases.'' 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 
``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

[[Page 53008]]

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.
    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 
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: Shanthi Marur, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2369, Silver Spring, MD 20993-0002, 240-
402-6373; 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 ``Evaluating Cancer Drugs in Patients with Central Nervous 
System Metastases.'' This draft guidance provides recommendations for 
sponsors designing clinical trials of drugs and biological products 
regulated by CDER and CBER that are intended to support product 
labeling describing the antitumor activity in patients with central 
nervous system (CNS) metastases from solid tumors originating outside 
the CNS. Specifically, the draft guidance includes recommendations 
regarding the patient population, available therapy, prior therapies, 
assessment of CNS disease, study endpoints, and leptomeningeal disease. 
The draft guidance describes that CNS metastases should be evaluated in 
the context of the entire disease burden and discusses how treatment 
effects may be described in drug labeling. The recommendations pertain 
to clinical trials for systemic anticancer drugs where patients with 
CNS metastases are included in the study population. These 
recommendations are also applicable to trials conducted exclusively in 
patients with CNS metastases.
    CNS metastases are associated with significant morbidity and 
mortality and development of therapeutic products for patients with CNS 
metastases is needed. FDA has participated in efforts to facilitate 
drug development for patients with CNS metastases including a March 
2019 ``Workshop on Product Development for CNS Metastases''. 
Stakeholders at this meeting stated there is a need for further FDA 
guidance on specific topics including identifying optimal study 
endpoints. Study design challenges for CNS metastases include 
uncertainty regarding optimal endpoints, lack of standardized response 
assessments, understanding how CNS metastases are evaluated in the 
context of the entire burden of metastatic disease to characterize a 
drug's potential benefit (e.g., timing of CNS radiographic assessments 
relative to other sites of metastases), and interpreting radiographic 
response in the setting of recent radiation therapy or surgery. This 
draft guidance is intended to provide recommendations on these study 
design challenges.
    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 ``Evaluating 
Cancer Drugs in Patients with Central Nervous System Metastases.'' 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. 3501-3520). 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 
0910-0338; and 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 the draft 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: August 24, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-18894 Filed 8-26-20; 8:45 am]
BILLING CODE 4164-01-P