[Federal Register Volume 87, Number 125 (Thursday, June 30, 2022)]
[Notices]
[Pages 39101-39103]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-13952]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-2455]


Patient-Focused Drug Development: Selecting, Developing, or 
Modifying Fit-for-Purpose Clinical Outcome Assessments; Draft Guidance 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the availability of a draft guidance for industry entitled ``Patient-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 
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. When finalized, Guidance 3 
will represent the current thinking of the Center for Drug Evaluation 
and Research, the Center for Biologics Evaluation and Research, and the 
Center for Devices and Radiological Health on this topic.

DATES: Submit either electronic or written comments on the draft 
guidance by September 28, 2022 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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:
     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

[[Page 39102]]

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-2018-N-2455 for ``Patient-Focused Drug Development: Selecting, 
Developing, or Modifying Fit-for-Purpose Clinical Outcome 
Assessments.'' 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 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, 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. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Shannon Cole, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 6306, Silver Spring, MD 20993-0002, 301-
796-9208, [email protected]; 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; or Office of Strategic Partnerships and Technology 
Innovation, Center for Devices and Radiological Health, [email protected], 800-638-2041 or 301-796-7100.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
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 section 201), or a biological product (as defined 
in section 351 of the Public Health Service Act (42 U.S.C. 262)).
    This series of guidance documents is intended to facilitate the 
advancement and use of systematic approaches to collect and use robust 
and meaningful input that can more consistently inform medical product 
development and regulatory decision-making. Guidance 3 discusses 
approaches to selecting, modifying, developing, and validating clinical 
outcome assessments to measure outcomes of importance to patients in 
clinical trials.
    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 ``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. 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. 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 OMB under 5 CFR 1320.3(h)(5). Respondents submit 
to FDA collections of information to support the medical product's

[[Page 39103]]

effectiveness and to support claims in approved medical labeling.
    The collections of information in 21 CFR 314.50, 314.126, 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 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''` (https://www.fda.gov/media/109951/download) 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 
J.1 of the commitment letter, ``Enhancing the Incorporation of the 
Patient's Voice in Drug Development and Decision-Making'' (https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf) 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 use 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 draft guidance 
at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: June 24, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-13952 Filed 6-29-22; 8:45 am]
BILLING CODE 4164-01-P