[Federal Register Volume 86, Number 110 (Thursday, June 10, 2021)]
[Notices]
[Pages 30944-30945]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-12166]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-2303]


Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft 
Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Core 
Patient-Reported Outcomes in Cancer Clinical Trials.'' This draft 
guidance provides recommendations to sponsors regarding the collection 
of a core set of patient-reported clinical outcomes in cancer clinical 
trials and related considerations for instrument selection and trial 
design. This guidance focuses on patient-reported outcome (PRO) 
measures and is specific to registration trials for anti-cancer 
therapies intended to demonstrate an effect on survival, tumor 
response, or delay in the progression of a malignancy. The draft 
guidance recommendations supplement previous guidance on use of PRO 
measures in clinical trials by providing additional considerations 
specific to the cancer clinical trial setting. The draft guidance is 
intended to facilitate generation of high-quality data on a core set of 
patient-reported symptom and functional impacts that are important 
contributors to a patient's health-related quality of life.

DATES: Submit either electronic or written comments on the draft 
guidance by August 9, 2021 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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: 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.
    <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-2020-D-2303 for ``Core Patient-Reported Outcomes in Cancer Clinical 
Trials.'' 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.
    <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 https://www.regulations.gov. 
Submit

[[Page 30945]]

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 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 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. 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: Vishal Bhatnagar, Oncology Center of 
Excellence, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 22, Rm. 2113, Silver Spring, MD 20993-0002, 240-402-3696; or 
Janice Kim, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2329, Silver 
Spring, MD 20993-0002, 301-796-9628; 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 ``Core Patient-Reported Outcomes in Cancer Clinical Trials.'' 
This draft guidance provides recommendations to sponsors regarding the 
collection of a core set of PROs in cancer clinical trials and related 
considerations for instrument selection and trial design. Although this 
draft guidance focuses on PRO measures, some of the recommendations may 
be relevant to other clinical outcome assessments (i.e., clinician-
reported outcome, observer-reported outcome, performance outcome) in 
cancer clinical trials. The draft guidance is specific to registration 
trials for anti-cancer therapies intended to demonstrate an effect on 
survival, tumor response, or delay in the progression of a malignancy.
    Cancer clinical trials typically employ standardized efficacy 
assessment using overall survival and tumor measures, and safety 
assessments provided by clinician reporting of adverse events. FDA 
acknowledges the potential added value of incorporating PRO measurement 
of symptoms and functional impacts into the benefit/risk assessment in 
appropriately designed trials; however, heterogeneity in PRO assessment 
strategies has lessened the regulatory utility of PRO data from cancer 
trials. Systematic assessment of a core set of PROs can facilitate high 
quality data on patient-reported symptoms and functional impacts. FDA 
has previously described a core set of PROs that may be important 
contributors to a patient's health-related quality of life and that may 
be sensitive to the effect of the disease and treatment under study.\1\
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    \1\ Kluetz, P.G., A. Slagle, E.J. Papadopoulos, et al., 2016, 
``Focusing on Core Patient-Reported Outcomes in Cancer Clinical 
Trials: Symptomatic Adverse Events, Physical Function, and Disease-
Related Symptoms,'' Clinical Cancer Research, 22(7):1553-1558.
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    FDA is issuing this draft guidance to provide FDA's current 
thinking on the core PROs, considerations for instrument selection to 
measure the core PROs, trial design considerations such as assessment 
frequency, and labeling considerations. The core PROs recommended in 
the draft guidance are disease-related symptoms, symptomatic adverse 
events, overall side effect impact summary measure, physical function, 
and role function.
    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 ``Core 
Patient-Reported Outcomes in Cancer Clinical Trials.'' 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; and the 
collections of information in 21 CFR part 601 have been approved under 
0910-0338.

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: June 4, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-12166 Filed 6-9-21; 8:45 am]
BILLING CODE 4164-01-P