Notice2024-17924
Advancing Rare Disease Therapies Through a Food and Drug Administration Rare Disease Innovation Hub; Public Meeting; Request for Comments
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Published
August 12, 2024
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 Rare Disease Therapies Through an FDA Rare Disease Innovation Hub." The purpose of the public meeting is to discuss the establishment of a Rare Disease Innovation Hub, which will enhance collaboration and cooperation across the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER), as well as other centers and offices across FDA, to advance rare disease therapies. In particular, this meeting will be an opportunity for those in the rare disease community, including patients and caregiver groups, industry organizations, and scientific/academic organizations, to provide input on the priorities of the Rare Disease Innovation Hub and how the Hub can best engage with members of the rare disease community. The public meeting will be facilitated by the Reagan-Udall Foundation for the FDA.
Full Text
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<title>Federal Register, Volume 89 Issue 155 (Monday, August 12, 2024)</title>
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[Federal Register Volume 89, Number 155 (Monday, August 12, 2024)]
[Notices]
[Pages 65631-65633]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-17924]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-N-3528]
Advancing Rare Disease Therapies Through a Food and Drug
Administration Rare Disease Innovation Hub; Public Meeting; Request for
Comments
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 Rare
[[Page 65632]]
Disease Therapies Through an FDA Rare Disease Innovation Hub.'' The
purpose of the public meeting is to discuss the establishment of a Rare
Disease Innovation Hub, which will enhance collaboration and
cooperation across the Center for Biologics Evaluation and Research
(CBER) and the Center for Drug Evaluation and Research (CDER), as well
as other centers and offices across FDA, to advance rare disease
therapies. In particular, this meeting will be an opportunity for those
in the rare disease community, including patients and caregiver groups,
industry organizations, and scientific/academic organizations, to
provide input on the priorities of the Rare Disease Innovation Hub and
how the Hub can best engage with members of the rare disease community.
The public meeting will be facilitated by the Reagan-Udall Foundation
for the FDA.
DATES: The public meeting will be held on October 16, 2024, from 10
a.m. to 1 p.m. Eastern Time. Either electronic or written comments on
this public meeting must be submitted by 11:59 p.m. Eastern Time on
October 31, 2024. See the SUPPLEMENTARY INFORMATION section for
registration date and information.
ADDRESSES: The public meeting will be held at the FDA White Oak Campus,
10903 New Hampshire Ave., Bldg. 31, Rm. 1503 (Great Room), Silver
Spring, MD 20993-0002. Entrance for the public meeting participants
(non-FDA employees) is through Bldg. 1 where routine security check
procedures will be performed. For parking and security information,
please refer to <a href="https://www.fda.gov/about-fda/visitor-information">https://www.fda.gov/about-fda/visitor-information</a>.
You may submit written 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 October 31, 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-2024-N-3528 for ``Advancing Rare Disease Therapies Through an FDA
Rare Disease Innovation Hub; Public Meeting; 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 <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: Janet Goldberg, 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; or Cynthia Rothblum-Oviatt, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
22, Rm. 5390, Silver Spring, MD 20993-0002, 301-796-0957.
SUPPLEMENTARY INFORMATION:
I. Background
CBER and CDER are launching a CBER-CDER Rare Disease Innovation Hub
(the Hub) to advance rare disease therapy development through greater
communication, collaboration, and coordination across CBER and CDER, in
coordination with other centers and offices across FDA. Helmed by
senior leadership from CBER and CDER, the Hub will work to develop and
implement a rare disease comprehensive cross-center strategic agenda
that takes full advantage of our current clinical and scientific
expertise across both centers and is based on a shared vision and
comprehensive approach to (1) align review efforts; (2) identify and
enable innovative approaches in the areas of novel endpoints, biomarker
development, and innovative trial designs; and (3) streamline
communications with the rare disease community.
[[Page 65633]]
The Directors of CBER and CDER (Directors) will lead the Hub, co-
chairing the Rare Disease Innovation Hub Steering Committee, which will
include senior leaders from CBER's Office of Therapeutic Products,
CDER's Office of New Drugs, and across FDA, such as the Center for
Devices and Radiological Health, Oncology Center of Excellence, Office
of Orphan Products Development, and Office of Combination Products. The
Rare Disease Innovation Hub will leverage the activities of the CDER
Accelerating Rare disease Cures program and CBER Rare Disease Program
and enhance existing cross-center collaborations. In addition, the Hub
will be anchored by the new Director for Strategic Coalitions for the
Hub (Associate Director for Rare Disease Strategy), who will serve as a
single point of connection and engagement with stakeholders on behalf
of the Hub, including patient and caregiver groups, trade
organizations, and scientific/academic organizations, on cross-cutting
rare disease-related issues. The Directors will develop approaches to
ensure appropriate FDA staff involvement or appropriate settings for
further external engagement and will seek input from the community to
inform the priorities of the Hub.
The Reagan-Udall Foundation for the FDA will facilitate the public
meeting. The Reagan-Udall Foundation for the FDA is an independent
501(c)(3) not-for-profit organization created by Congress to advance
the mission of FDA to modernize medical, veterinary, food, food
ingredient, and cosmetic product development; accelerate innovation;
and enhance product safety. With the ultimate goal of improving public
health, the Foundation provides a unique opportunity for different
sectors (FDA, patient groups, academia, other government entities, and
industry) to work together in a transparent way to create exciting new
research and engagement projects to advance regulatory science.
II. Topics for Discussion at the Public Meeting
This public meeting is being convened for FDA to provide
information on the Rare Disease Innovation Hub, including its proposed
priorities and initiatives, and to serve as an opportunity for those in
the rare disease community, including patients and caregiver groups,
industry organizations, and scientific/academic organizations, to
provide input on the priorities of the Rare Disease Innovation Hub and
how the Hub can best engage with members of the rare disease community.
In particular, FDA is interested in receiving comments on the
following:
1. What specific rare disease-related scientific, regulatory, or
policy issues should be prioritized for consideration by the Rare
Disease Innovation Hub?
2. To the extent the issues identified in response to Question 1
are related to specific types of rare diseases or conditions, please
explain.
3. What specific types of rare disease-related activities do you
believe would benefit from enhanced collaboration, focused attention,
or increased transparency (to the extent legally permissible) under the
Rare Disease Innovation Hub? Please identify in your comments rare
disease-related activities or initiatives currently being undertaken by
CDER or CBER that you believe would benefit from being undertaken by
the Rare Disease Innovation Hub as a joint endeavor.
4. Please comment on approaches that the Rare Disease Innovation
Hub should follow for engagement with patients and caregiver groups,
industry organizations, and scientific/academic organizations
(including different approaches for different types of engagement, as
appropriate).
Comments will be accepted until 11:59 p.m. Eastern Time on October
31, 2024.
III. Participating in the Public Meeting
Registration: To register for the public meeting, please visit the
following website to register: <a href="https://reaganudall.org/news-and-events/events/advancing-rare-disease-therapies-through-an-fda-rare-disease-innovation-hub">https://reaganudall.org/news-and-events/events/advancing-rare-disease-therapies-through-an-fda-rare-disease-innovation-hub</a>. Please provide complete contact information for each
attendee, including name, title, affiliation, address, email, and
telephone number.
Registration is free and based on space availability, with priority
given to early registrants. Persons interested in attending this public
meeting must register online by October 15, 2024, at 5 p.m. Eastern
Time. Early registration is recommended because seating is limited;
therefore, FDA may limit the number of participants from each
organization.
If you need special accommodations due to a disability, please
contact Lea Ann Browning-McNee, Director of Communication and
Stakeholder Engagement, Reagan-Udall Foundation for FDA, 202-849-2075,
<a href="/cdn-cgi/l/email-protection#86caebe5e8e3e3c6f4e3e7e1e7e8f3e2e7eaeaa8e9f4e1"><span class="__cf_email__" data-cfemail="94d8f9f7faf1f1d4e6f1f5f3f5fae1f0f5f8f8bafbe6f3">[email protected]</span></a>, no later than October 9, 2024, at 5 p.m.
Eastern Time.
Requests for Oral Presentations: During online registration you may
indicate if you wish to present during a public comment session and
which topic(s) you wish to address. We will do our best to accommodate
requests to make public comments. Individuals and organizations with
common interests are urged to consolidate or coordinate their
presentations, and request time for a joint presentation, or submit
requests for designated representatives to participate in the focused
sessions. We will determine the amount of time allotted to each
presenter and the approximate time each oral presentation is to begin
and will select and notify participants by October 4, 2024. All
requests to make oral presentations must be received by 11:59 p.m. on
September 25, 2024. If selected for a presentation, you will be
contacted by Lea Ann Browning-McNee, Director of Communication and
Stakeholder Engagement, Reagan-Udall Foundation for FDA regarding
submission of a single slide in PowerPoint format. No commercial or
promotional material will be permitted to be presented or distributed
at the public meeting.
(Notice of this meeting is given pursuant to 21 CFR 10.65.)
Dated: August 7, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-17924 Filed 8-9-24; 8:45 am]
BILLING CODE 4164-01-P
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