[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:
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-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.
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]
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