[Federal Register Volume 87, Number 39 (Monday, February 28, 2022)]
[Notices]
[Pages 11077-11079]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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draft guidance of the same title issued on October 1, 2019.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:
     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.''

[[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 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 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, [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.

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'' (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 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: 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