[Federal Register Volume 89, Number 155 (Monday, August 12, 2024)]
[Notices]
[Pages 65631-65633]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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 https://www.fda.gov/about-fda/visitor-information.
    You may submit written 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 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:
     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-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 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: 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.

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    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: https://reaganudall.org/news-and-events/events/advancing-rare-disease-therapies-through-an-fda-rare-disease-innovation-hub. 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, 
[email protected], 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