[Federal Register Volume 84, Number 190 (Tuesday, October 1, 2019)]
[Notices]
[Pages 52114-52115]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-21226]


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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Important to Patients; Draft Guidance for Industry, Food and Drug 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the availability of a draft guidance for industry, FDA staff, and other 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.

DATES: Submit either electronic or written comments on the draft 
guidance by December 30, 2019 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-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.
     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

[[Page 52115]]

the information at: https://www.gpo.gov/fdsys/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.
    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 draft guidance document.

FOR FURTHER INFORMATION CONTACT: Meghana Chalasani, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993-0002, 240-
402-6525, Fax: 301-847-8443, Meghana.Chalasani@fda.hhs.gov; 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 draft 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. 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 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: 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. 
This guidance is not subject to Executive Order 12866.

II. 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'' (available at 
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 use robust and meaningful patient and 
caregiver input that can more consistently inform drug development, 
and, as appropriate, regulatory decision-making.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or https://www.regulations.gov.

    Dated: September 25, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-21226 Filed 9-30-19; 8:45 am]
 BILLING CODE 4164-01-P


