[Federal Register Volume 84, Number 220 (Thursday, November 14, 2019)]
[Notices]
[Pages 61912-61913]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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as input on examples that could be illustrated in the forthcoming draft 
guidance, where approaches proposed in the discussion document have 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

[[Page 61913]]

Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6304, 
Silver Spring, MD 20993-0002, 240-402-6525, Fax: 301-847-8443, 
[email protected].

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 301-796-0621 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