Document ID: FDA-2022-N-2480-0004
Agency: fda
Document Type: Notice
Title: Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development; Public Workshop;
Posted Date: 2023-04-17T04:00Z

[Federal Register Volume 88, Number 73 (Monday, April 17, 2023)]
[Notices]
[Pages 23431-23433]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-08066]

-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 https://www.regulations.gov 
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:
     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.

[[Page 23432]]

     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-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 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: Mary Jo Salerno, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993, 240-402-0420, 
[email protected].

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 https://www.fda.gov/media/151712/download. Specifically, 
section I.K.4 of the PDUFA VII Commitment Letter, ``Advancing 
Development of Drugs for Rare Diseases'' (https://www.fda.gov/media/151712/download), 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 https://duke.is/5ca3k prior to the workshop.

III. Participating in the Public Workshop

    Registration: To register for the public workshop, please visit the 
following website: https://duke.is/5ca3k. 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 [email protected] 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 https://duke.is/5ca3k. The transcript will also be available at https://www.regulations.gov 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