Notice2023-25500
Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement
Primary source
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Published
November 20, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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.
Full Text
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<title>Federal Register, Volume 88 Issue 222 (Monday, November 20, 2023)</title>
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[Federal Register Volume 88, Number 222 (Monday, November 20, 2023)]
[Notices]
[Pages 80728-80729]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-25500]
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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.
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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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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,
<a href="/cdn-cgi/l/email-protection#7a2a1b0e131f140e3c15190f091f1e3a1c1e1b54121209541d150c"><span class="__cf_email__" data-cfemail="98c8f9ecf1fdf6ecdef7fbedebfdfcd8fefcf9b6f0f0ebb6fff7ee">[email protected]</span></a>.
[[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 <a href="https://duke.is/4/7yuu">https://duke.is/4/7yuu</a> prior to the meeting.
III. Participating in the Public Meeting
Registration: To register for the public meeting, please visit the
following website: <a href="https://duke.is/4/7yuu">https://duke.is/4/7yuu</a>. 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 <a href="/cdn-cgi/l/email-protection#afe2ceddc8c0c3c6dccad9cac1dbdcefcbdac4ca81cacbda"><span class="__cf_email__" data-cfemail="86cbe7f4e1e9eaeff5e3f0e3e8f2f5c6e2f3ede3a8e3e2f3">[email protected]</span></a> 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 <a href="https://duke.is/4/7yuu">https://duke.is/4/7yuu</a>. The transcript will also be available at <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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
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