[Federal Register Volume 84, Number 220 (Thursday, November 14, 2019)]
[Notices]
[Pages 61912-61913]
[FR Doc No: 2019-24726]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-N-4900]

Patient-Focused Drug Development Guidance: Incorporating Clinical
Outcome Assessments Into Endpoints for Regulatory Decision Making;
Public Workshop; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop; request for comments.

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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is
announcing a public workshop to convene a discussion on incorporating
clinical outcome assessments (COAs) into endpoints for regulatory
decision making. This workshop will inform development of patient-
focused drug development guidance as required by the 21st Century Cures
Act (Cures Act) and as part of commitments made by FDA under the sixth
authorization of the Prescription Drug User Fee Amendments (PDUFA VI).
The Agency will publish a discussion document approximately 1 month
before the workshop date. FDA is interested in seeking information and
comments on the approaches proposed in the discussion document, as well
as input on examples that could be illustrated in the forthcoming draft
guidance, where approaches proposed in the discussion document have
been successfully applied.

DATES: The public workshop will be held on December 6, 2019, from 9
a.m. to 5 p.m. Submit either electronic or written comments on this
public workshop by February 4, 2020. See the SUPPLEMENTARY INFORMATION
section for registration date and information.

ADDRESSES: The public workshop will be held at FDA's White Oak Campus,
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room
(Rm. 1503), Silver Spring, MD 20993. Entrance for the public workshop
participants (non-FDA employees) is through Building 1 where routine
security check procedures will be performed. For parking and security
information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    You may submit comments as follows. Please note that late, untimely
filed comments will not be considered. Electronic comments must be
submitted on or before February 4, 2020. The https://www.regulations.gov electronic filing system will accept comments until
11:59 p.m. Eastern Time at the end of February 4, 2020. Comments
received by mail/hand delivery/courier (for written/paper submissions)
will be considered timely if they are postmarked or the delivery
service acceptance receipt is on or before that date.

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-N-4900 for ``Patient-Focused Drug Development Guidance:
Incorporating Clinical Outcome Assessments into Endpoints for
Regulatory Decision Making; Public Workshop; Request for Comments.''
Received comments, those filed in a timely manner (see ADDRESSES), 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 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.

FOR FURTHER INFORMATION CONTACT: Meghana Chalasani, Center for Drug
Evaluation and Research, Food and

Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6304,
Silver Spring, MD 20993-0002,<PHONE_NUMBER>, Fax:<PHONE_NUMBER>,
<EMAIL_ADDRESS>
SUPPLEMENTARY INFORMATION:

I. Background

    This public workshop is intended to support FDA implementation of
requirements for guidance development under section 3002 of the Cures
Act (Pub. L. 114-255) and to meet a performance goal included in PDUFA
VI. Section 3002 of Title III, Subtitle A of the Cures Act directs FDA
to develop patient-focused drug development guidance to address a
number of areas, including methodologies, standards, and technologies
to collect and analyze COA data for purposes of regulatory decision-
making.
    In addition, FDA committed to meet certain performance goals under
PDUFA VI. This reauthorization, part of the FDA Reauthorization Act of
2017 (Pub. L. 115-52), signed by President Trump on August 18, 2017,
includes a number of performance goals and procedures that are
documented in the PDUFA VI Commitment Letter, which is available at
https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf.
    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 utilize
robust and meaningful patient and caregiver input that can more
consistently inform drug development, and, as appropriate, regulatory
decision making.
    Prior to the issuance of each guidance, as part of the development,
FDA will conduct a public workshop to gather input from the wider
community of patients, patient advocates, academic researchers, expert
practitioners, drug developers, and other stakeholders.

II. Topics for Discussion at the Public Workshop

    During the public workshop, speakers and participants will address
a range of issues and considerations related to incorporating COAs into
endpoints for regulatory decision-making. The range of issues and
considerations includes: (1) Endpoint development; (2) estimands and
analysis models; (3) addressing heterogeneity in disease symptoms and
functional status between patients and within the same patient over
time; and (4) data collection, storage, transmission, and analysis.

III. Participating in the Public Workshop

    Registration: Interested parties are encouraged to register early.
To register electronically, please visit https://patientfocuseddrugdevelopment.eventbrite.com. Registration for in-
person attendance will close on December 3, 2019. Registration for the
webcast will remain open until the day of the workshop. Persons without
access to the internet can call<PHONE_NUMBER> to register. If you are
unable to attend the workshop in person, you can register to view a
live webcast of the workshop. You will be asked to indicate in your
registration if you plan to attend in person or via the webcast.
Seating will be limited, so early registration is recommended.
    Registration is free. FDA may limit the number of participants from
each organization based on space limitations. Registrants will receive
confirmation once they have been accepted. Onsite registration on the
day of the workshop will be based on space availability.
    If you need special accommodations due to a disability, please
contact Meghana Chalasani (see FOR FURTHER INFORMATION CONTACT) at
least 7 days before the workshop.
    Requests for Oral Presentations: There will be time allotted during
the workshop for open public comment. Sign-up for this session will be
on a first-come, first-served basis on the day of the workshop.
Individuals and organizations with common interests are urged to
consolidate or coordinate, and request time for a joint presentation.
No commercial or promotional material will be permitted to be presented
or distributed at the public workshop.
    Streaming Webcast of the Public Workshop: This public workshop will
also be webcast at https://collaboration.fda.gov/pfddg123119/. If you
have never attended a Connect Pro event before, test your connection at
https://collaboration.fda.gov/common/help/en/support/meeting_test.htm.
To get a quick overview of the Connect Pro program, visit https://www.adobe.com/go/connectpro_overview. FDA has verified the website
addresses in this document, as of the date this document publishes in
the Federal Register, but websites are subject to change over time.
    Transcripts: Please be advised that as soon as a transcript of the
public workshop is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Dockets Management Staff
(see ADDRESSES). A link to the transcript will also be available on the
internet at https://www.fda.gov/drugs/news-events-human-drugs/patient-focused-drug-development-guidance-collection-and-analysis-clinical-outcome-assessment-data.

    Dated: November 8, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-24726 Filed 11-13-19; 8:45 am]
 BILLING CODE 4164-01-P