[Federal Register Volume 89, Number 202 (Friday, October 18, 2024)]
[Notices]
[Pages 83884-83885]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-24111]


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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; 
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 final guidance for industry entitled ``Core 
Patient-Reported Outcomes in Cancer Clinical Trials.'' This final 
guidance provides recommendations to sponsors regarding the collection 
of a core set of patient-reported clinical outcomes (herein referred to 
as core patient-reported outcomes) in cancer clinical trials and 
related considerations for instrument selection and trial design. This 
final 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.

DATES: The announcement of the guidance is published in the Federal 
Register on October 18, 2024.

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:
    <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 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. Send one self-
addressed adhesive label to assist that office in processing your 
requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the 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 
James Myers, Center for Biologics Evaluation and Research, Food and 
Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, 
Silver Spring, MD 20993-0002, 240-402-7910.

SUPPLEMENTARY INFORMATION:

[[Page 83885]]

I. Background

    FDA is announcing the availability of a final guidance for industry 
entitled ``Core Patient-Reported Outcomes in Cancer Clinical Trials.'' 
This final 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. The final 
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 final 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.
    Although this final 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 final 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 
assessments using overall survival and tumor measures, and safety 
assessments provided by clinician reporting of adverse events. FDA 
acknowledges the 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. 
In published literature, FDA authors have 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.
    FDA is issuing this final 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 guidance are disease-related symptoms, symptomatic adverse events, 
overall side effect impact summary measure, physical function, and role 
function.
    In the Federal Register of June 10, 2021 (86 FR 30944), FDA 
announced the availability of the draft guidance of the same title 
dated June 2021. FDA considered comments received on the draft guidance 
as the guidance was finalized. Changes from the draft to the final 
guidance include recommendations to consult FDA when selecting adverse 
events for reporting, edits to include hematological malignancies, and 
minor, editorial changes to improve clarity.
    This final guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). This final guidance 
represents 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. The 
previously approved 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 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 guidance at 
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: October 11, 2024.
Eric Flamm,
Acting Associate Commissioner for Policy.
[FR Doc. 2024-24111 Filed 10-17-24; 8:45 am]
BILLING CODE 4164-01-P