[Federal Register Volume 88, Number 66 (Thursday, April 6, 2023)]
[Notices]
[Pages 20530-20531]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-07243]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-D-0026]


Patient-Focused Drug Development: Incorporating Clinical Outcome 
Assessments Into Endpoints for Regulatory Decision-Making; Draft 
Guidance for Industry, Food and Drug Administration Staff, and Other 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the availability of a draft guidance for industry entitled ``Patient-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: Incorporating Clinical Outcome Assessments 
Into Endpoints for Regulatory Decision-Making.'' This draft guidance 
(Guidance 4) is the fourth 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.

DATES: Submit either electronic or written comments on the draft 
guidance by July 5, 2023 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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 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-2023-D-0026 for ``Patient-Focused Drug Development: Incorporating 
Clinical Outcome Assessments Into Endpoints for Regulatory Decision-
Making.'' 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. 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 Sparklin, 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 Diane Maloney, 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

[[Page 20531]]

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: Incorporating Clinical 
Outcome Assessments Into Endpoints for Regulatory Decision-Making.'' 
This draft guidance (Guidance 4) is the fourth of a series of four 
methodological patient-focused drug development 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. 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.
    The purpose of Guidance 4 is to: (1) address methods to better 
incorporate clinical outcome assessment into endpoints that are 
considered significantly robust for regulatory decision-making; (2) 
address methodologies, standards, and technologies that may be used for 
the collection, capture, storage, and analysis of patient perspective 
data; and (3) identify resources that offer considerations regarding 
submissions of patient experience data.
    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 
``Incorporating Clinical Outcome Assessments Into Endpoints for 
Regulatory Decision-Making.'' 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

    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). This guidance 
also refers to previously approved FDA collections of information. 
These collections of information are subject to review by OMB under the 
Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521). The collections 
of information in 21 CFR parts 312 and 812 for investigational new drug 
applications and investigational device exemptions have been approved 
under OMB control numbers 0910-0014 and 0910-0078, respectively. The 
collections of information in 21 CFR parts 314 and 601 for new drug 
applications and biologic license applications have been approved under 
OMB control numbers 0910-0001 and 0910-0338, respectively, and the 
collections of information in 21 CFR part 814, subparts A through E, 21 
CFR part 860, subpart D, and 21 CFR part 807, subpart E, for premarket 
approval applications, De Novo classification requests, and premarket 
notifications have been approved under OMB control numbers 0910-0231, 
0910-0844, and 0910-0120, respectively.

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)(4): methodologies, standards, and technologies to 
collect and analyze clinical outcome assessments for purposes of 
regulatory decision-making.
    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'' (https://www.fda.gov/media/99140/download) 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 draft guidance 
at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: April 3, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-07243 Filed 4-5-23; 8:45 am]
BILLING CODE 4164-01-P