Notice2022-23383
Rare Disease Endpoint Advancement Pilot Meeting Program
Primary source
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Published
October 27, 2022
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The seventh iteration of the Prescription Drug User Fee Amendments (PDUFA VII) included as part of the FDA User Fee Reauthorization Act of 2022 highlights the goal of advancing and facilitating the development and timely approval of drugs and biological products for rare diseases, including rare diseases in children. The Food and Drug Administration (FDA or Agency) is announcing the Rare Disease Endpoint Advancement Pilot Meeting Program (RDEA Pilot Program) established under the seventh iteration of PDUFA that affords sponsors who are admitted into the RDEA Pilot Program additional engagement opportunities with the Agency to discuss efficacy endpoint development in rare disease drug and biological product development programs. Meetings under the program will be conducted by FDA's Center for Drug Evaluation and Research (CDER) or Center for Biologics Evaluation and Research (CBER) during fiscal years (FYs) 2023 to 2027. For each sponsor whose RDEA program proposal (RDEA proposal or proposal) is admitted into the program, up to four meetings that will provide an opportunity for medical product developers to discuss rare disease endpoint development will be held between the sponsor and CDER or CBER. To promote innovation and evolving science, novel endpoints developed through the RDEA Pilot Program may be presented by FDA (e.g., in a guidance or public workshop or on a public-facing website) as case studies, including novel endpoints for drugs that have not yet been approved or biological products that have not yet been licensed by FDA for a given indication.
Full Text
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<title>Federal Register, Volume 87 Issue 207 (Thursday, October 27, 2022)</title>
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[Federal Register Volume 87, Number 207 (Thursday, October 27, 2022)]
[Notices]
[Pages 65085-65088]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-23383]
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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 Meeting Program
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
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SUMMARY: The seventh iteration of the Prescription Drug User Fee
Amendments (PDUFA VII) included as part of the FDA User Fee
Reauthorization Act of 2022 highlights the goal of advancing and
facilitating the development and timely approval of drugs and
biological products for rare diseases, including rare diseases in
children. The Food and Drug Administration (FDA or Agency) is
announcing the Rare Disease Endpoint Advancement Pilot Meeting Program
(RDEA Pilot Program) established under the seventh iteration of PDUFA
that affords sponsors who are admitted into the RDEA Pilot Program
additional engagement opportunities with the Agency to discuss efficacy
endpoint development in rare disease drug and
[[Page 65086]]
biological product development programs. Meetings under the program
will be conducted by FDA's Center for Drug Evaluation and Research
(CDER) or Center for Biologics Evaluation and Research (CBER) during
fiscal years (FYs) 2023 to 2027. For each sponsor whose RDEA program
proposal (RDEA proposal or proposal) is admitted into the program, up
to four meetings that will provide an opportunity for medical product
developers to discuss rare disease endpoint development will be held
between the sponsor and CDER or CBER. To promote innovation and
evolving science, novel endpoints developed through the RDEA Pilot
Program may be presented by FDA (e.g., in a guidance or public workshop
or on a public-facing website) as case studies, including novel
endpoints for drugs that have not yet been approved or biological
products that have not yet been licensed by FDA for a given indication.
DATES: The RDEA Pilot Program will proceed from October 1, 2022,
through September 30, 2027. Sponsors may submit RDEA program proposals
beginning July 1, 2023, through June 30, 2027. Submit either electronic
or written comments about this program by December 27, 2022.
ADDRESSES: You may submit comments about the RDEA Pilot Program 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 December 27, 2022.
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-2022-N-2480 for ``Rare Disease Endpoint Advancement Pilot
Program.'' 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 20993-0002, 240-402-0420,
<a href="/cdn-cgi/l/email-protection#5c0e18191d721139392835323b2f1c3a383d7234342f723b332a"><span class="__cf_email__" data-cfemail="ce9c8a8b8fe083ababbaa7a0a9bd8ea8aaafe0a6a6bde0a9a1b8">[email protected]</span></a>, with the subject line ``RDEA Pilot Meeting
Program for CDER'' or Julienne Vaillancourt, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7252, Silver Spring, MD 20993-0002, 301-
796-1827, <a href="/cdn-cgi/l/email-protection#3f6d7b7a7e11725a5a4b5651584c7f595b5e1157574c11585049"><span class="__cf_email__" data-cfemail="affdebeaee81e2cacadbc6c1c8dcefc9cbce81c7c7dc81c8c0d9">[email protected]</span></a>, with the subject line ``RDEA Pilot
Meeting Program for CBER.'' Additional information is available on the
RDEA Pilot Program web page: <a href="https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program">https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program</a>.
SUPPLEMENTARY INFORMATION:
I. Background
In connection with the seventh iteration of PDUFA, FDA committed to
conduct a pilot program to advance rare disease drug development
programs by providing a mechanism for sponsors to collaborate with FDA
throughout the efficacy endpoint development process (see ``PDUFA
Reauthorization Performance Goals and Procedures Fiscal Years 2023
Through 2027,'' section I.K.4.a, <a href="https://www.fda.gov/media/151712/download">https://www.fda.gov/media/151712/download</a>).
FDA is announcing this pilot program to satisfy the above-mentioned
commitment. The goals of the early meeting discussions granted under
this program are to provide advice on how a proposed novel endpoint can
be used in a specific rare disease drug development program and to
promote innovation by allowing FDA to publicly present the proposed
novel endpoints (or natural history studies in which the proposed
endpoint is intended to be studied) considered through the program,
including novel endpoints for drugs or biological products that have
[[Page 65087]]
not yet been approved or licensed by FDA for a given indication. FDA
has committed to accepting a limited number of qualified proposals for
admission into the RDEA Pilot Program that increases after the first
year of PDUFA VII. For FY 2023, sponsors may submit RDEA proposals
beginning in the fourth quarter, and FDA will accept a maximum of one
proposal. For FYs 2024 through 2027, FDA will accept up to one RDEA
proposal per quarter with a maximum of three proposals per year.
Complete RDEA proposals may be submitted throughout the quarter on
a rolling basis; however, only those received by the quarterly closing
date, which will be the last day of each quarter of the fiscal year
(i.e., December 31, March 31, June 30, September 30), will be
considered for selection in the following quarter. Within 60 days after
the quarterly closing date, FDA will review the RDEA proposals, select
a proposal to proceed to disclosure discussions, and notify sponsors of
their proposal status. When FDA and the sponsor agree on the
information that FDA may share publicly, FDA will notify the sponsor of
admission into the program.
Sponsors admitted to the RDEA Pilot Program may participate in up
to four focused meetings with relevant FDA staff to discuss endpoint
development. FDA's advice provided during and between RDEA meetings
does not constitute a regulatory decision and is considered nonbinding.
Being admitted into the RDEA Pilot Program and completing four RDEA
meetings does not guarantee approval for a regulatory submission that
includes efficacy endpoints discussed during RDEA meetings. Likewise,
being denied admission into the RDEA Pilot Program does not mean that
the proposed novel endpoint is unacceptable for regulatory decision
making.
After completion of four RDEA meetings, the sponsor can request
additional input on their novel endpoint from FDA, as needed, through
other formal meeting mechanisms, such as Type B, Type C, Type C
Surrogate Endpoint, or Type D meetings. Sponsors that do not
participate in the RDEA Pilot Program will have an opportunity to
interact with the Agency through traditional channels.
The listed eligibility and selection factors outlined in this
notice reflect the current thinking at the time of publication.
Information about the process for applying to and participating in the
RDEA pilot meeting program will be communicated on the following web
page: <a href="https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program">https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program</a>.
II. General, Eligibility, and Disclosure Information for the RDEA Pilot
Program
A. General Information
The RDEA Pilot Program will be jointly administered by the
following Centers:
<bullet> CDER: CDER's Rare Diseases Team, in the Office of New
Drugs, Division of Rare Diseases and Medical Genetics, which is the
point of contact for RDEA Pilot Program communications for CDER
products.
<bullet> CBER: CBER's Rare Disease Liaison, in the Office of the
Director, Policy Staff, which is the point of contact for RDEA Pilot
Program communications for CBER products.
B. Eligibility and Selection Information
To be eligible for the RDEA Pilot Program:
<bullet> The associated development program should be active and
address a rare disease, with an active investigational new drug
application (IND) or pre-IND for the rare disease.
[cir] Sponsors that do not yet have an active development program
but have, or are initiating, a natural history study where the proposed
endpoint is intended to be studied are also eligible to apply.
[cir] FDA may also consider accepting a proposal for a development
program for a common disease that includes innovative or novel endpoint
elements, including the specific endpoint and/or the methodology being
developed if there is sufficient justification that the proposal could
be applicable to a rare disease.
<bullet> The proposed endpoint is a novel efficacy endpoint
intended to establish substantial evidence of effectiveness for a rare
disease treatment. An endpoint is considered novel if it has never been
used to support drug approval or if it has been substantially modified
from previous use to support drug approval.
Preference will be given to proposals:
<bullet> That have the potential to impact drug development more
broadly, such as one that uses a novel approach to develop an efficacy
endpoint or an endpoint that could potentially be relevant to other
diseases.
<bullet> That collectively reflect a range of different types of
endpoints.
<bullet> That have novel approaches for collecting additional
clinical data in the premarket stage to advance validation of the
endpoint for a surrogate endpoint proposal. If the sponsor is proposing
to develop a surrogate endpoint as part of a rare disease application,
participation in a prior Type C Surrogate Endpoint meeting is
encouraged.
C. Disclosure
To promote innovation in this area, novel efficacy endpoints
developed through the RDEA Pilot Program may be presented by FDA (e.g.,
in a guidance, at public workshops and conferences, or on FDA's
website) as case studies, including while the drug studied in the trial
has not yet been approved by FDA. Accordingly, before FDA grants the
initial meeting under the program, FDA and the sponsor must agree on
the information that FDA may include in these public case studies. The
specific information to be disclosed will depend on the content of each
novel efficacy endpoint and associated natural history study if
applicable. FDA intends to focus on information that is beneficial to
advancing efficacy endpoint development for drugs that treat rare
diseases and those elements relevant to understanding the novel
efficacy endpoint and its potential use in a clinical trial intended to
support regulatory approval.
Sponsors wishing to participate in the program should identify
aspects of the proposed novel endpoint and associated natural history,
if applicable, that they consider nondisclosable and provide a
rationale for withholding the information. Participation in the
program, including any agreement on information disclosure, will be
voluntary and at the discretion of the sponsor. Sponsors that do not
wish to make such disclosures may seek regulatory input through other
existing channels.
IV. Paperwork Reduction Act of 1995
While this notice contains no collection of information, it does
refer to previously approved FDA collections of information. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521) is not required
for this notice. The previously approved collections of information are
subject to review by OMB under the PRA. The collections of information
in 21 CFR part 312 for INDs and clinical trials have been approved
under OMB control number 0910-0014. The collections of information in
21 CFR part 601 for biologic new drug applications (NDAs) have been
approved under OMB control number 0910-0338. The collections of
information in 21 CFR part 314 for the submission of NDAs and for
requesting meetings with FDA about drug development programs have been
[[Page 65088]]
approved under OMB control number 0910-0001. The collections of
information relating to rare disease drug and biological product
development programs have been approved under OMB control number 0910-
0765.
Dated: October 21, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-23383 Filed 10-26-22; 8:45 am]
BILLING CODE 4164-01-P
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