Notice2022-04152
Patient-Focused Drug Development: Methods To Identify What Is Important to Patients; Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; Availability
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Published
February 28, 2022
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: Methods To Identify What Is Important to Patients." This guidance (Guidance 2) is the second in a series of four methodological guidance documents that FDA committed to develop to describe how to collect and submit 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 This guidance finalizes the draft guidance of the same title issued on October 1, 2019.
Full Text
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<title>Federal Register, Volume 87 Issue 39 (Monday, February 28, 2022)</title>
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[Federal Register Volume 87, Number 39 (Monday, February 28, 2022)]
[Notices]
[Pages 11077-11079]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-04152]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2019-D-4247]
Patient-Focused Drug Development: Methods To Identify What Is
Important to Patients; 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: Methods To
Identify What Is Important to Patients.'' This guidance (Guidance 2) is
the second in a series of four methodological guidance documents that
FDA committed to develop to describe how to collect and submit
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draft guidance of the same title issued on October 1, 2019.
DATES: The announcement of the guidance is published in the Federal
Register on February 28, 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: <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.''
[[Page 11078]]
Instructions: All submissions received must include the Docket No.
FDA-2019-D-4247 for ``Patient-Focused Drug Development: Methods To
Identify What Is Important to Patients.'' 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 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, 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 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, <a href="/cdn-cgi/l/email-protection#03506b626d6d6c6d2d406c6f66436567622d6b6b702d646c75"><span class="__cf_email__" data-cfemail="90c3f8f1fefefffebed3fffcf5d0f6f4f1bef8f8e3bef7ffe6">[email protected]</span></a>, 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.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry, FDA
staff, and other stakeholders entitled ``Patient-Focused Drug
Development: Methods To Identify What Is Important to Patients.'' This
guidance (Guidance 2) is the second in a series of four methodological
patient-focused drug development guidance documents that FDA committed
to develop to describe how stakeholders (patients, researchers, medical
product developers, and others) can collect and submit 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 patient and caregiver input
that can more consistently inform medical product development and
regulatory decision making. The purpose of Guidance 2 is to present a
range of methods and established best research practices to 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 particular, the methods and best practices
presented can help elicit relevant information from patients and other
stakeholders, such as how their disease affects their daily lives; what
they find most troublesome; and the challenges, problems, and burdens
of the treatment for the disease.
This guidance finalizes the draft guidance entitled ``Patient-
Focused Drug Development: Methods To Identify What Is Important to
Patients'' issued on October 1, 2019 (84 FR 52114). FDA considered
comments received on the draft guidance as the guidance was finalized.
Changes from the draft to the final guidance include incorporating the
definitions of relevant terms within the body of the document instead
of as part of a glossary. In addition, editorial changes and
methodological clarifications were made to improve 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: Methods
To Identify What Is Important to Patients.'' 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). Therefore, clearance by the OMB under
the Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. This guidance also refers to previously
approved FDA collections of information. The previously approved
collections of information are subject to review by OMB under the PRA.
The information collections described in this guidance are submitted to
FDA to support the medical product's effectiveness and to support
claims in approved medical product labeling (see 21 CFR 314.50,
314.126, and 601.2). The information collections have been approved
under OMB control numbers 0910-0001 and 0910-0338. The collections of
information in 21 CFR parts 50 and 56 have been approved under OMB
control number 0910-0130.
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
[[Page 11079]]
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'' (<a href="https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf">https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf</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 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 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/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>,
<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: February 22, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-04152 Filed 2-25-22; 8:45 am]
BILLING CODE 4164-01-P
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