Notice2023-07243
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Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making; Draft Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; Availability
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
April 6, 2023
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Patient- Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making." This draft guidance (Guidance 4) is the fourth in a series of four methodological patient- focused drug development (PFDD) guidance documents that describe how stakeholders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.
Full Text
<html>
<head>
<title>Federal Register, Volume 88 Issue 66 (Thursday, April 6, 2023)</title>
</head>
<body><pre>
[Federal Register Volume 88, Number 66 (Thursday, April 6, 2023)]
[Notices]
[Pages 20530-20531]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-07243]
-----------------------------------------------------------------------
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Assessments Into Endpoints for Regulatory Decision-Making; Draft
Guidance for Industry, Food and Drug Administration Staff, and Other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.
-----------------------------------------------------------------------
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the availability of a draft guidance for industry entitled ``Patient-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 This draft guidance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:
<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-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 <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)).
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, <a href="/cdn-cgi/l/email-protection#4e1d262f20202120601d3e2f3c252227200e282a2f6026263d60292138"><span class="__cf_email__" data-cfemail="a8fbc0c9c6c6c7c686fbd8c9dac3c4c1c6e8ceccc986c0c0db86cfc7de">[email protected]</span></a>; 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, <a href="/cdn-cgi/l/email-protection#046760766c2974766b446260652a6c6c772a636b72"><span class="__cf_email__" data-cfemail="e88b8c9a80c5989a87a88e8c89c680809bc68f879e">[email protected]</span></a>, 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'' (<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 draft 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>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
</pre><script data-cfasync="false" src="/cdn-cgi/scripts/5c5dd728/cloudflare-static/email-decode.min.js"></script></body>
</html>Indexed from Federal Register on April 6, 2023.
This is legal information, not legal advice. Laws vary by jurisdiction and change frequently. Always verify current law with official sources and consult a licensed attorney in your jurisdiction for advice on your specific situation.