Notice2022-01377
Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation; Guidance for Industry and Food and Drug Administration Staff, and Other Stakeholders; Availability
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Published
January 26, 2022
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 final guidance entitled "Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation." FDA encourages the collection, analysis, and integration of patient perspectives in the development, evaluation, and surveillance of medical devices, including digital health technologies. Patient-reported outcome (PRO) instruments facilitate the systematic collection of patient perspectives as valid scientific evidence to support the regulatory and healthcare decision-making process. This guidance describes principles that should be considered when using PRO instruments in the evaluation of medical devices and provides recommendations about the importance of ensuring the measures are fit- for-purpose. This guidance is not meant to replace the Patient-Focused Drug Development (PFDD) guidance series. Some of the comments received in the docket may be addressed in PFDD Guidance #3, which is currently in development.
Full Text
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<title>Federal Register, Volume 87 Issue 17 (Wednesday, January 26, 2022)</title>
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[Federal Register Volume 87, Number 17 (Wednesday, January 26, 2022)]
[Notices]
[Pages 4033-4035]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-01377]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2020-D-1564]
Principles for Selecting, Developing, Modifying, and Adapting
Patient-Reported Outcome Instruments for Use in Medical Device
Evaluation; Guidance for Industry and Food and Drug Administration
Staff, and Other Stakeholders; 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 entitled ``Principles for
Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome
Instruments for Use in Medical Device Evaluation.'' FDA encourages the
collection, analysis, and integration of patient perspectives in the
development, evaluation, and surveillance of medical devices, including
digital health technologies. Patient-reported outcome (PRO) instruments
facilitate the systematic
[[Page 4034]]
collection of patient perspectives as valid scientific evidence to
support the regulatory and healthcare decision-making process. This
guidance describes principles that should be considered when using PRO
instruments in the evaluation of medical devices and provides
recommendations about the importance of ensuring the measures are fit-
for-purpose. This guidance is not meant to replace the Patient-Focused
Drug Development (PFDD) guidance series. Some of the comments received
in the docket may be addressed in PFDD Guidance #3, which is currently
in development.
DATES: The announcement of the guidance is published in the Federal
Register on January 26, 2022.
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: <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-1564 for ``Principles for Selecting, Developing, Modifying,
and Adapting Patient-Reported Outcome Instruments for Use in Medical
Device Evaluation.'' 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 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)).
An electronic copy of the guidance document is available for
download from the internet. See the SUPPLEMENTARY INFORMATION section
for information on electronic access to the guidance. Submit written
requests for a single hard copy of the guidance document entitled
``Principles for Selecting, Developing, Modifying, and Adapting
Patient-Reported Outcome Instruments for Use in Medical Device
Evaluation'' to the Office of Policy, Center for Devices and
Radiological Health, Food and Drug Administration, 10903 New Hampshire
Ave., Bldg. 66, Rm. 5431, Silver Spring, MD 20993-0002; or to 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 request.
FOR FURTHER INFORMATION CONTACT: Michelle Tarver, Center for Devices
and Radiological Health, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 66, Rm. 5608, Silver Spring, MD 20993-0002, 301-
796-6884; 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, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
A PRO instrument can be used in a clinical investigation to measure
the effects of a medical intervention or changes in the health status
of a patient. PRO instruments allow for collection of certain data as
valid scientific evidence of safety and effectiveness that is
complementary to other clinical outcomes and/or biomarkers. Information
from well-defined and reliable PRO instruments can provide valuable
evidence for benefit-risk assessments and can be used in medical device
labeling to communicate the effect of a treatment on patient symptoms,
functioning, or quality of life when the labeling is consistent with
the PRO instrument's documented measurement capability. PRO instruments
may be used to inform a patient's eligibility for inclusion within a
study, to capture safety or effectiveness outcomes, and may be aligned
as primary or secondary endpoints or used as a stand-alone outcome
assessment or component of a composite endpoint. When data from a PRO
instrument is used in the evaluation of a medical device, FDA
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determines the validity evidence needed to support the PRO instrument's
specified use for a regulatory purpose. FDA uses the term ``fit-for-
purpose'' to describe this flexible approach. As part of providing
valid scientific evidence to assess the safety and effectiveness of
medical devices, PRO instruments can measure the impact of medical
devices on patient well-being and other concepts that may influence
payers, healthcare providers, and patients when making decisions about
potential treatments or management options.
A notice of availability of the draft guidance appeared in the
Federal Register of August 31, 2020 (85 FR 53820). FDA considered
comments received and revised the guidance as appropriate in response
to the comments, including clarifying and expanding examples, making
clear the language relating to PRO instrument scores, as well as
clarifying the applicability of PRO instruments throughout the total
product life cycle and within clinical studies. Additional language on
recommendations to document modifications, ensure content is relevant,
and consider patient burden was also included. This guidance is not
meant to replace the Patient-Focused Drug Development (PFDD) guidance
series. Comments received in the docket related to the PFDD guidance
series have been shared with the Center for Drug Evaluation and
Research (CDER) to be considered as part of the development of PFDD
Guidance #3 entitled ``Selecting, Developing or Modifying Fit-for-
Purpose Clinical Outcome Assessments'' and PFDD Guidance #4 entitled
``Incorporating Clinical Outcome Assessments into Endpoints for
Regulatory Decision Making.'' \1\
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\1\ For more information, please see the FDA PFDD Guidance
Series website: <a href="https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical">https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical</a>.
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This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Principles for Selecting, Developing,
Modifying, and Adapting Patient-Reported Outcome Instruments for Use in
Medical Device Evaluation.'' 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. Electronic Access
Persons interested in obtaining a copy of the guidance may do so by
downloading an electronic copy from the internet. A search capability
for all Center for Devices and Radiological Health guidance documents
is available at <a href="https://www.fda.gov/medical-devices/device-advice-comprehensive-regulatory-assistance/guidance-documents-medical-devices-and-radiation-emitting-products">https://www.fda.gov/medical-devices/device-advice-comprehensive-regulatory-assistance/guidance-documents-medical-devices-and-radiation-emitting-products</a>. This guidance is also available at
<a href="https://www.regulations.gov">https://www.regulations.gov</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a> or <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>. Persons unable to download an electronic
copy of ``Principles for Selecting, Developing, Modifying, and Adapting
Patient-Reported Outcome Instruments for Use in Medical Device
Evaluation'' may send an email request to <a href="/cdn-cgi/l/email-protection#440700160c6903312d20252a2721042220256a2c2c376a232b32"><span class="__cf_email__" data-cfemail="9edddaccd6b3d9ebf7fafff0fdfbdef8faffb0f6f6edb0f9f1e8">[email protected]</span></a> to
receive an electronic copy of the document. Please use the document
number 18042 and complete title to identify the guidance you are
requesting.
III. Paperwork Reduction Act of 1995
While this guidance contains no new 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 the following FDA regulations and guidance have been
approved by OMB as listed in the following table:
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OMB control
21 CFR part or guidance Topic No.
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807, subpart E................. Premarket notification. 0910-0120
814, subparts A through E...... Premarket approval..... 0910-0231
814, subpart H................. Humanitarian Device 0910-0332
Exemption.
812............................ Investigational Device 0910-0078
Exemption.
860, subpart D................. De Novo classification 0910-0844
process.
``FDA and Industry Procedures 513(g) Request for 0910-0705
for Section 513(g) Requests Information.
for Information under the
Federal Food, Drug, and
Cosmetic Act''.
``Requests for Feedback on Q-submissions.......... 0910-0756
Medical Device Submissions:
The Pre-Submission Program and
Meetings with Food and Drug
Administration Staff''.
800, 801, and 809.............. Medical Device Labeling 0910-0485
Regulations.
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Dated: January 19, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-01377 Filed 1-25-22; 8:45 am]
BILLING CODE 4164-01-P
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