[Federal Register Volume 87, Number 17 (Wednesday, January 26, 2022)]
[Notices]
[Pages 4033-4035]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-01377]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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: 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-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 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)).
    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

[[Page 4035]]

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\
---------------------------------------------------------------------------

    \1\ For more information, please see the FDA PFDD Guidance 
Series website: https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical.
---------------------------------------------------------------------------

    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 https://www.fda.gov/medical-devices/device-advice-comprehensive-regulatory-assistance/guidance-documents-medical-devices-and-radiation-emitting-products. This guidance is also available at 
https://www.regulations.gov, https://www.fda.gov/regulatory-information/search-fda-guidance-documents or https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances. 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:

------------------------------------------------------------------------
                                                            OMB control
    21 CFR part or guidance               Topic                 No.
------------------------------------------------------------------------
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.
------------------------------------------------------------------------


    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


</pre><script data-cfasync="false" src="/cdn-cgi/scripts/5c5dd728/cloudflare-static/email-decode.min.js"></script></body>
</html>