[Federal Register Volume 88, Number 222 (Monday, November 20, 2023)]
[Notices]
[Pages 80728-80729]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-25500]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-N-4718]


Advancing the Development of Therapeutics Through Rare Disease 
Patient Community Engagement

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is 
announcing the following public meeting entitled ``Advancing the 
Development of Therapeutics Through Rare Disease Patient Community 
Engagement.'' Convened by the Duke-Robert J. Margolis, MD Center for 
Health Policy (Duke-Margolis) in collaboration with FDA and supported 
by a cooperative agreement between FDA and Duke-Margolis, the workshop 
will focus on how best to understand patients' experiences living with 
a rare disease and how to incorporate those experiences, as well as 
patients' priorities for treatment goals, throughout the drug 
development process.

DATES: The public meeting will be held virtually on December 14, 2023, 
from 12 p.m. to 5 p.m. Eastern Time. Either electronic or written 
comments on this public meeting must be submitted by February 12, 2024. 
See the SUPPLEMENTARY INFORMATION section for registration date and 
information.

ADDRESSES: The public meeting will be held virtually using the Zoom 
platform.
    You may submit comments as follows. Please note that late, untimely 
filed comments will not be considered. The https://www.regulations.gov 
electronic filing system will accept comments until 11:59 p.m. Eastern 
Time at the end of February 12, 2024. Comments received by mail/hand 
delivery/courier (for written/paper submissions) will be considered 
timely if they are received 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-2023-N-4718 for ``Advancing the Development of Therapeutics Through 
Rare Disease Patient Community Engagement.'' 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, 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.

FOR FURTHER INFORMATION CONTACT: Stuti Ganatra, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave, Silver Spring, MD 20993, 301-796-8112, 
[email protected].

[[Page 80729]]


SUPPLEMENTARY INFORMATION:

I. Background

    This public meeting is intended to facilitate improvements in the 
treatment of rare diseases and conditions, consistent with the 
requirements under section 3202 of the Food and Drug Omnibus Reform Act 
of 2022 (FDORA). Section 3202 of FDORA requires FDA to conduct a number 
of activities related to improving the treatment of rare diseases and 
conditions, including the convening of one or more public meetings to 
address increasing and improving engagement with rare disease patients, 
rare disease patient groups, and experts on small population studies, 
in order to improve the understanding of patient burden, treatment 
options, and the side effects of treatments (see section 3202(d)(2) of 
FDORA).

II. Topics for Discussion at the Public Meeting

    The purpose of this public meeting is to highlight and build upon 
existing actionable approaches for engaging patients, patient groups, 
and related experts when developing necessary evidence for rare disease 
drug approvals. The meeting will address approaches to increasing and 
improving engagement with rare disease patients, groups representing 
such patients, rare disease experts, and experts on small population 
studies, to improve the understanding of how to best understand 
patients' experiences living with a rare disease and how to incorporate 
those experiences and priorities throughout the drug development 
process. This includes understanding patient perspectives on the burden 
of their condition and any existing treatment options, as well as how 
their current health status and risk of disease progression may impact 
willingness to accept risks from treatment side effects.
    Meeting updates, the agenda, and background materials (if any) will 
also be made available at https://duke.is/4/7yuu prior to the meeting.

III. Participating in the Public Meeting

    Registration: To register for the public meeting, please visit the 
following website: https://duke.is/4/7yuu. Please provide complete 
contact information for each attendee, including name, title, 
affiliation, address, email, and telephone. Registration will end at 
11:59 p.m. Eastern Time on December 13, 2023.
    Registration is free and persons interested in attending this 
public meeting must register to receive a link to the meeting. 
Registrants will receive a confirmation email after they register.
    If you need special accommodations due to a disability, please 
contact [email protected] no later than November 30, 2023. Please 
note, closed captioning will be available automatically.
    Transcripts: Please be advised that as soon as a transcript of the 
public meeting is available, it will be accessible at https://duke.is/4/7yuu. The transcript will also be available at https://www.regulations.gov and may be viewed at the Dockets Management Staff 
(see ADDRESSES).

    Dated: November 14, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-25500 Filed 11-17-23; 8:45 am]
BILLING CODE 4164-01-P