Notice2025-10801
Rare Disease Innovation, Science, and Exploration Public Workshop Series; Request for Comments
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
June 13, 2025
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 request for comments for a future public workshop series entitled "Rare disease Innovation, Science, and Exploration (RISE) Workshop." The purpose of the public workshops will be to focus on challenges that are common to multiple diseases or a class of diseases, and for which evolving science offers innovative solutions. The workshops will primarily focus on cross-cutting or common issues and will not be focused on any specific product under review by the Agency.
Full Text
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<title>Federal Register, Volume 90 Issue 113 (Friday, June 13, 2025)</title>
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[Federal Register Volume 90, Number 113 (Friday, June 13, 2025)]
[Notices]
[Pages 25056-25058]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2025-10801]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2025-N-1559]
Rare Disease Innovation, Science, and Exploration Public Workshop
Series; Request for Comments
AGENCY: Food and Drug Administration, HHS.
[[Page 25057]]
ACTION: Notice; request for comments.
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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is
announcing the following request for comments for a future public
workshop series entitled ``Rare disease Innovation, Science, and
Exploration (RISE) Workshop.'' The purpose of the public workshops will
be to focus on challenges that are common to multiple diseases or a
class of diseases, and for which evolving science offers innovative
solutions. The workshops will primarily focus on cross-cutting or
common issues and will not be focused on any specific product under
review by the Agency.
DATES: Submit either electronic or written comments by December 31,
2025.
ADDRESSES: 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 December 31, 2025. 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-2025-N-1559 for ``Rare Disease Innovation, Science, and Exploration
Public Workshop Series; 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' 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: Philipa Friedman, Center for Biologics
Evaluation and Research, 10903 New Hampshire Ave., Silver Spring, MD
20993, 240-402-7911, and <a href="/cdn-cgi/l/email-protection#4b190f022525243d2a3f222425033e290b2d2f2a65232338652c243d"><span class="__cf_email__" data-cfemail="2270666b4c4c4d5443564b4d4c6a5740624446430c4a4a510c454d54">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
Background
The FDA is publishing this request for comments to obtain
suggestions for topics for future RISE Workshops. The FDA Rare Disease
Innovation Hub (Hub) supports the RISE Workshop series pursuant to its
commitment to further advance regulatory science of rare disease
therapies and the Agency's PDUFA VII commitments to enhance regulatory
science and expedite drug development and rare disease product review
under the Food, Drug, and Cosmetic Act.
The Hub-sponsored RISE workshop series focuses on challenges that
are common to multiple diseases or a class of diseases, and for which
evolving science offers innovative solutions. The workshops are open to
the public and designed for interaction and discourse between the
various rare disease community members and perspectives, including drug
developers, patient and disease organizations, academics, FDA
regulators and reviewers, and relevant staff from other federal
agencies. All workshops include coordination between the Center for
Biologics Evaluation and Research (CBER) and the Center for Drug
Evaluation and Research (CDER) and address the Centers' approaches to
the relevant issues. The workshops also include a discussion of the
role of patients and patient/disease organizations in the design and
implementation of innovative solutions. The workshops primarily focus
on cross-cutting or common issues and will not be focused on any
specific product under review by the Agency. Preference will be given
to submissions that are germane to multiple disease states and/or that
are submitted jointly by two or more entities.
The Agency requests that submissions include:
--a description of the proposed topic;
--suggested speakers and/or subject matter experts;
--a description of the impact of the topic on the development and
regulatory science of rare disease therapies;
--the disease state(s) affected by the challenge highlighted in the
submission;
[[Page 25058]]
--if relevant, related FDA guidances or existing programs addressing
the challenge highlighted in the submission.
Notice of this meeting series is given pursuant to 21 CFR 10.65.
Dated: June 9, 2025.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2025-10801 Filed 6-12-25; 8:45 am]
BILLING CODE 4164-01-P
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