Notice2025-20153
Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments; Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; Availability
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Published
November 18, 2025
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 for industry, FDA staff, and other stakeholders entitled "Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments." This guidance (Guidance 3) is the third in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, researchers, medical product developers, and others) can submit patient experience and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making. This guidance finalizes the draft guidance of the same title issued on June 30, 2022.
Full Text
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<title>Federal Register, Volume 90 Issue 220 (Tuesday, November 18, 2025)</title>
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[Federal Register Volume 90, Number 220 (Tuesday, November 18, 2025)]
[Notices]
[Pages 51765-51767]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2025-20153]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2022-D-1385]
Patient-Focused Drug Development: Selecting, Developing, or
Modifying Fit-for-Purpose Clinical Outcome Assessments; Guidance for
Industry, 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 for industry, FDA staff, and other
stakeholders entitled ``Patient-Focused Drug Development: Selecting,
Developing, or Modifying Fit-for-Purpose Clinical Outcome
Assessments.'' This guidance (Guidance 3) is the third in a series of
four methodological patient-focused drug development (PFDD) guidance
documents that describe how stakeholders (patients, researchers,
medical product developers, and others) can submit patient experience
and other relevant information from patients and caregivers to be used
for medical product development and regulatory decision-making. This
guidance finalizes the draft guidance of the same title issued on June
30, 2022.
DATES: The announcement of the guidance is published in the Federal
Register on November 18, 2025.
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-2022-D-1385 for ``Patient-Focused Drug Development: Selecting,
Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments;
Guidance for Industry, Food and Drug Administration Staff, and Other
Stakeholders.'' 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
[[Page 51766]]
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)).
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: Ethan Gabbour, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 6306, Silver Spring, MD 20993-0002, 301-
796-8112, <a href="/cdn-cgi/l/email-protection#a9ecddc1c8c787eec8cbcbc6dcdbe9cfcdc887c1c1da87cec6df"><span class="__cf_email__" data-cfemail="a2e7d6cac3cc8ce5c3c0c0cdd7d0e2c4c6c38ccacad18cc5cdd4">[email protected]</span></a>; or Phillip Kurs, Center for
Biologics Evaluation and Research, Food and Drug Administration, 240-
402-7911, or the Office of Strategic Partnerships and Technology
Innovation, Center for Devices and Radiological Health, <a href="/cdn-cgi/l/email-protection#86e5e2f4eeabf6f4e9c6e0e2e7a8eeeef5a8e1e9f0"><span class="__cf_email__" data-cfemail="b8dbdccad095c8cad7f8dedcd996d0d0cb96dfd7ce">[email protected]</span></a>, 800-638-2041 or 301-796-7100.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry, FDA
staff, and other stakeholders entitled ``Patient-Focused Drug
Development: Selecting, Developing, or Modifying Fit-for-Purpose
Clinical Outcome Assessments.'' This guidance (Guidance 3) is the third
in a series of four methodological PFDD guidance documents that
describe how stakeholders (patients, researchers, medical product
developers, and others) can collect and submit patient experience data
and other relevant information from patients and caregivers to be used
for medical product development and regulatory decision-making. For
purposes of this guidance a ``medical product'' refers to a drug (as
defined in section 201 of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 321)) intended for human use, a device (as defined in such
section 201) intended for human use, or a biological product (as
defined in section 351 of the Public Health Service Act (42 U.S.C.
262)).
The series of guidance documents is intended to facilitate the
advancement and use of systematic approaches to collect and use robust
and meaningful patient and caregiver input that can more consistently
inform medical product development and regulatory decision-making.
Guidance 3 discusses approaches to selecting, modifying, developing,
and evaluating clinical outcome assessments to measure outcomes of
importance to patients in clinical trials.
This guidance finalizes the draft guidance entitled ``Patient-
Focused Drug Development: Selecting, Developing, or Modifying Fit-for-
Purpose Clinical Outcome Assessments'' issued on June 30, 2022 (87 FR
39101). FDA considered comments received on the draft guidance as the
guidance was finalized. Changes from the draft to final guidance
included the following: clarification of the relationships between
relevant terminology, reformulation of the conceptual framework into a
description of the COA-based endpoint approach, addition of clarifying
language on the importance of qualitative data and patient input,
updates to the evidence-based rationale, updated references that
reflect the state of science, and moving content that was more relevant
to COA-based endpoints to PFDD Guidance 4: ``Incorporating Clinical
Outcome Assessments into Endpoints for Regulatory Decision Making.'' In
addition, editorial changes were made to improve flow and clarity.
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 ``Patient-Focused Drug Development:
Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome
Assessments.'' 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 (PRA) (44 U.S.C 3501-3521). This guidance refers to
collections of information from ``individuals under treatment or
clinical examination in connection with research,'' which are not
subject to review by the Office of Management and Budget (OMB) under 5
CFR 1320.3(h)(5). Respondents submit to FDA collections of information
to support the medical product's effectiveness and to support claims in
approved medical labeling.
The collections of information for 21 CFR part 812 for
investigational device exemptions have been approved under OMB control
number 0910-0078 and the collections of information in 21 CFR part 814
subpart H for humanitarian use devices have been approved under OMB
control number 0910-0332. The collections of information in 21 CFR part
814, subparts A through E for premarket approval applications have been
approved under OMB control number 0910-0231. The collections of
information in 21 CFR part 807, subpart E for premarket notifications
have been approved under OMB control number 0910-0120 and the
collections of information in 21 CFR 860, subpart D for De Novo
classifications have been approved under OMB control number 0910-0844.
The collections of information described in FDA's guidance entitled
``Requests for Feedback and Meetings for Medical Device Submissions:
The Q-Submission Program'' have been approved under OMB control number
0910-0756. The collections of information in 21 CFR part 822 for
postmarket surveillance have been approved under OMB control number
0910-0449.
The collections of information in 21 CFR parts 314.50, 314.26, and
601.2 are submitted to FDA to support the medical product's
effectiveness and to support claims in approved medical product
labeling. The collections of information have been approved under OMB
control numbers 0910-0001 and 0910-0338. The collections of
[[Page 51767]]
information in 21 CFR 312.23 regarding investigational new drug
applications, including clinical trial design and study protocols, have
been approved under OMB control number 0910-0014. The collections of
information in 21 CFR parts 50 and 56 regarding institutional review
boards and the protection of human subjects have been approved under
OMB control number 0910-0130. The collections of information in 21 CFR
part 11 regarding electronic records and signatures have been approved
under OMB control number 0910-0303. The collections of information
described in FDA's guidance entitled ``Formal Meetings Between the FDA
and Sponsors or Applicants of PDUFA Products'' (<a href="https://www.fda.gov/media/109951/download">https://www.fda.gov/media/109951/download</a>) have been approved under OMB control number
0910-0429.
III. Additional Information
Section 3002 of Title III, Subtitle A, of the 21st Century Cures
Act (Pub. L. 114-255) directs FDA to develop patient-focused drug
development guidance to address a number of areas, including under
section 3002(c)(2): Methodological approaches that may be used to
develop and identify what is important to patients with respect to
burden of disease, burden of treatment, and the benefits and risks in
the management of the patient's disease.
In addition, FDA committed to meet certain performance goals under
the sixth authorization of the Prescription Drug User Fee Act. These
goal commitments were developed in consultation with patient and
consumer advocates, healthcare professionals, and other public
stakeholders, as part of negotiations with regulated industry. Section
I.J.1 of the commitment letter ``Enhancing the Incorporation of the
Patient's Voice in Drug Development and Decision-Making'' (<a href="https://www.fda.gov/media/99140/download">https://www.fda.gov/media/99140/download</a>) outlines work, including the
development of a series of guidance documents and associated public
workshops to facilitate the advancement and use of systematic
approaches to collect and utilize robust and meaningful patient and
caregiver input that can more consistently inform drug development,
and, as appropriate, regulatory decision-making.
IV. Electronic Access
Persons with access to the internet may obtain the guidance at
<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/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</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>.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2025-20153 Filed 11-17-25; 8:45 am]
BILLING CODE 4164-01-P
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