Notice2022-13952
Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments; Draft Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; Availability
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Published
June 30, 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Patient- Focused Drug Development: Selecting, Developing, or Modifying Fit-for- Purpose Clinical Outcome Assessments."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.
Full Text
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<title>Federal Register, Volume 87 Issue 125 (Thursday, June 30, 2022)</title>
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[Federal Register Volume 87, Number 125 (Thursday, June 30, 2022)]
[Notices]
[Pages 39101-39103]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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Modifying Fit-for-Purpose Clinical Outcome Assessments; Draft Guidance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the availability of a draft guidance for industry entitled ``Patient-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:
<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
[[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 <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-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 <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, 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, <a href="/cdn-cgi/l/email-protection#5a09323b34343534741935363f1a3c3e3b74323229743d352c"><span class="__cf_email__" data-cfemail="beedd6dfd0d0d1d090fdd1d2dbfed8dadf90d6d6cd90d9d1c8">[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; or Office of Strategic Partnerships and Technology
Innovation, Center for Devices and Radiological Health, <a href="/cdn-cgi/l/email-protection#6b080f1903461b19042b0d0f0a45030318450c041d"><span class="__cf_email__" data-cfemail="61020513094c11130e210705004f0909124f060e17">[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: 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''` (<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
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 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/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: 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
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