Notice2021-12166
Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft Guidance for Industry; Availability
Primary source
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Published
June 10, 2021
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Core Patient-Reported Outcomes in Cancer Clinical Trials." This draft guidance provides recommendations to sponsors regarding the collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related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 The draft guidance recommendations supplement previous guidance on use of PRO measures in clinical trials by providing additional considerations specific to the cancer clinical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 The draft guidance is intended to facilitate generation of high-quality data on a core set of patient-reported symptom and functional impacts that are important contributors to a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.
Full Text
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<title>Federal Register, Volume 86 Issue 110 (Thursday, June 10, 2021)</title>
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[Federal Register Volume 86, Number 110 (Thursday, June 10, 2021)]
[Notices]
[Pages 30944-30945]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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Core Patient-Reported Outcomes in Cancer Clinical Trials; Draft
Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Core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 This draft
guidance provides recommendations to sponsors regarding the collection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: <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-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 <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
[[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:
<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 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 <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>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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