Notice2023-08066
Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development; Public Workshop; Request for Comments
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Published
April 17, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA, the Agency, or we) is announcing a public workshop entitled "Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development." Convened by the Duke-Robert J. Margolis, MD Center for Health Policy (Duke-Margolis) and supported by a cooperative agreement between FDA and Duke-Margolis, the workshop will include discussions of the Rare Disease Endpoint Advancement (RDEA) Pilot Program and novel endpoint development for rare disease drug development.
Full Text
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<title>Federal Register, Volume 88 Issue 73 (Monday, April 17, 2023)</title>
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[Federal Register Volume 88, Number 73 (Monday, April 17, 2023)]
[Notices]
[Pages 23431-23433]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-08066]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2022-N-2480]
Rare Disease Endpoint Advancement Pilot Program Workshop: Novel
Endpoints for Rare Disease Drug Development; Public Workshop; Request
for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is
announcing a public workshop entitled ``Rare Disease Endpoint
Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease
Drug Development.'' Convened by the Duke-Robert J. Margolis, MD Center
for Health Policy (Duke-Margolis) and supported by a cooperative
agreement between FDA and Duke-Margolis, the workshop will include
discussions of the Rare Disease Endpoint Advancement (RDEA) Pilot
Program and novel endpoint development for rare disease drug
development.
DATES: The public workshop will be held virtually on June 7, 2023, and
June 8, 2023, from 1 p.m. to 5 p.m., Eastern Time. Either electronic or
written comments on this public workshop must be submitted by July 23,
2023. See the SUPPLEMENTARY INFORMATION section for registration date
and information.
ADDRESSES: The public workshop will be held virtually using the Zoom
platform. The link for the public workshop will be sent to registrants
upon registration.
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 on July 23, 2023. 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>.
[[Page 23432]]
<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<ls-thn-eq> 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-2022-N-2480 for ``Rare Disease Endpoint Advancement Pilot Program
Workshop: Novel Endpoints for Rare Disease Drug Development.'' 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: Mary Jo Salerno, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993, 240-402-0420,
<a href="/cdn-cgi/l/email-protection#6c3e28292d422109091805020b1f2c0a080d4204041f420b031a"><span class="__cf_email__" data-cfemail="f7a5b3b2b6d9ba9292839e999084b7919396d99f9f84d9909881">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
I. Background
This public workshop is intended to support the RDEA Pilot Program
consistent with the requirements under section 3208 of the Food and
Drug Omnibus Reform Act of 2022 (FDORA). Section 3208 of FDORA requires
FDA to establish a pilot program to provide increased interaction with
sponsors of rare disease drug development programs for purposes of
advancing the development of efficacy endpoints, including surrogate
and intermediate endpoints, for drugs intended to treat rare diseases.
Section 3208 of FDORA also requires FDA to conduct up to three public
workshops to discuss various topics relevant to the development of
endpoints for rare diseases on or before September 30, 2026. This is
the first of up to three public workshops to satisfy the FDORA
requirement.
The public workshop is also intended to meet a performance goal
under the FDA User Fee Reauthorization Act of 2022, in accordance with
the PDUFA Reauthorization Performance Goals and Procedures Fiscal Years
2023 Through 2027 letter (PDUFA VII Commitment Letter), which is
available at <a href="https://www.fda.gov/media/151712/download">https://www.fda.gov/media/151712/download</a>. Specifically,
section I.K.4 of the PDUFA VII Commitment Letter, ``Advancing
Development of Drugs for Rare Diseases'' (<a href="https://www.fda.gov/media/151712/download">https://www.fda.gov/media/151712/download</a>), outlines commitments, including up to three public
workshops to discuss various topics relevant to endpoint development
for rare diseases.
II. Topics for Discussion at the Public Workshop
The purpose of this public workshop is to: (1) provide an overview
of the RDEA Pilot Program (a joint program of the Center for Drug
Evaluation and Research and the Center for Biologics Evaluation and
Research), (2) discuss the scientific and technical issues associated
with developing study endpoints for rare diseases and highlight
resources to assist with addressing these issues, (3) discuss lessons
learned from previous PDUFA meeting programs that can be applied to the
RDEA Pilot Program, and (4) provide opportunity for public comment on
the RDEA Pilot Program and issues associated with rare disease endpoint
development.
Meeting sessions will focus on: (1) the RDEA Pilot Program and
process, (2) elements of RDEA proposal and meeting packages, (3)
addressing issues in developing rare disease endpoints, and (4)
experience with other FDA pilot programs. At the end of the public
workshop, there will be an opportunity for public comment.
Meeting updates, the agenda, and background materials (if any) will
be made available at <a href="https://duke.is/5ca3k">https://duke.is/5ca3k</a> prior to the workshop.
III. Participating in the Public Workshop
Registration: To register for the public workshop, please visit the
following website: <a href="https://duke.is/5ca3k">https://duke.is/5ca3k</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 June 6, 2023.
Registration is free, and persons interested in attending this
public workshop 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#2865495a4f4744415b4d5e4d465c5b684c5d434d064d4c5d"><span class="__cf_email__" data-cfemail="064b677461696a6f756370636872754662736d6328636273">[email protected]</span></a> no later than May 5, 2023. Please note,
closed captioning will be available automatically.
Requests for Oral Comments: During online registration you may
indicate if you wish to speak during a public comment session. 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 comment and request time for joint
commentary. All requests to make oral comments must be received by
11:59 p.m. Eastern Time on May 26, 2023. FDA will determine the amount
of time
[[Page 23433]]
allotted to each commenter and the approximate time each comment is to
begin and will select and notify participants by June 2, 2023.
Transcript: Please be advised that as soon as a transcript of the
public workshop is available, it will be accessible at <a href="https://duke.is/5ca3k">https://duke.is/5ca3k</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: April 12, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-08066 Filed 4-14-23; 8:45 am]
BILLING CODE 4164-01-P
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