Support for Clinical Trials Advancing Rare Disease Therapeutics Pilot Program; Program Announcement, 67763-67765 [2023-21235]
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Federal Register / Vol. 88, No. 189 / Monday, October 2, 2023 / Notices
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LIHEAP Detailed Model Plan—FY24 ..............................................................
LIHEAP Detailed Model Plan—FY25 ..............................................................
LIHEAP Detailed Model Plan FY26 .................................................................
Estimated Total Burden Hours: ................................................................
Average Annual Burden Hours: ...............................................................
210
206
206
........................
........................
1
1
1
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.5
1
.5
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[FR Doc. 2023–21663 Filed 9–29–23; 8:45 am]
BILLING CODE 4184–80–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2023–N–3976]
Support for Clinical Trials Advancing
Rare Disease Therapeutics Pilot
Program; Program Announcement
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration’s (FDA or Agency)
Center for Biologics Evaluation and
Research’s (CBER) Office of Therapeutic
Products (OTP) and Center for Drug
Evaluation and Research’s (CDER)
Office of New Drugs are announcing the
opportunity for a limited number of
SUMMARY:
lotter on DSK11XQN23PROD with NOTICES1
Annual Burden Estimates
VerDate Sep<11>2014
18:41 Sep 29, 2023
Jkt 262001
development programs to participate in
the Support for clinical Trials
Advancing Rare disease Therapeutics
(START) Pilot Program, with the goal of
further accelerating the pace of
development of certain CBER- and
CDER-regulated products (novel drug
and biological products) that are
intended to treat a rare disease. Because
each Center has identified specific
needs concerning regulated products for
rare diseases, the eligibility criteria for
the pilot differ between CBER and
CDER. This pilot would augment the
currently available formal meetings
between FDA and sponsors by
addressing issues related to the
development of individual products
through more rapid, ad-hoc
communication mechanisms. Sponsors,
if selected for the pilot, would receive
more frequent advice related to such
specific issues through additional
interactions to facilitate novel drug and
biological product program
development and generate high quality
and reliable data intended to support a
Biologics License Application (BLA) or
New Drug Application (NDA). This
notice outlines the eligibility criteria,
what to submit in a request to
participate in the pilot, selection
criteria, process, and FDA-Sponsor
interactions expected to occur for
programs participating in the pilot.
DATES: From January 2, 2024, to March
1, 2024, FDA will accept requests to
participate in the START Pilot Program
and select no more than three
participants from each Center (CBER
and CDER). See the ‘‘Participation’’
section for eligibility criteria,
instructions on how to submit a request
to participate, and information
regarding the selection process.
FOR FURTHER INFORMATION CONTACT:
Andrew Harvan, Center for Biologics
Evaluation and Research, Food and
PO 00000
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Fmt 4703
Sfmt 4703
Annual
burden hours
for each form
105
206
103
414
138
Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7268,
Silver Spring, MD 20993–0002, 240–
402–7911; or Quyen Tran, Center for
Drugs Evaluation and Research, Food
and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6301,
Silver Spring, MD 20993–0002, 301–
796–2771.
For general questions about the
START Pilot Program for CBER:
Industry.biologics@fda.hhs.gov. For
general questions about the START Pilot
Program for CDER:
CDER.STARTProgram@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
The purpose of the START Pilot
Program is to further accelerate the pace
of development of novel drug and
biological products that are intended to
address an unmet medical need as a
treatment for a rare disease. The pilot is
designed to be milestone-driven (i.e., to
facilitate the progression of a
development program to pivotal clinical
study stage or the pre-BLA or pre-NDA
meeting stage) where product
development programs selected would
benefit from enhanced communications
with FDA. Participation in the pilot will
be considered concluded when the
development program has reached a
significant regulatory milestone such as
initiation of the pivotal clinical study
stage or the pre-BLA or pre-NDA
meeting stage as agreed upon with the
sponsor. Pilot participants will be
selected based on demonstrated
development program readiness. The
START Pilot Program is intended to
provide a mechanism for addressing
clinical development issues that
otherwise would delay or prevent a
promising novel drug or biological
product from progressing to the pivotal
E:\FR\FM\02OCN1.SGM
02OCN1
67764
Federal Register / Vol. 88, No. 189 / Monday, October 2, 2023 / Notices
clinical trial stage or pre-BLA/pre-NDA
meeting stage.
The pilot would augment the
currently available formal meetings
between FDA and sponsors (see FDA’s
draft guidance for industry entitled
‘‘Formal Meetings Between the FDA and
Sponsors or Applicants of PDUFA
Products’’ ((September 2023) (Ref. 1)))
through more rapid, ad-hoc
communications with FDA by
addressing issues with specific
programmatic needs for individual
products. For example, these issues can
be related to clinical study design,
choice of control group, fine-tuning the
choice of patient population, selecting
appropriate endpoints for efficacy trials
to support marketing approval, selecting
statistical methodology, leveraging
nonclinical information, or product
characterization. For eligible
development programs sponsors and
FDA could benefit from such additional
communication beyond the currently
available formal meeting mechanisms to
address specific programmatic needs
that require in-depth discussions. The
increased communication between FDA
review staff and sponsors is intended to
facilitate program development for
specific products and to help generate
high quality and reliable data intended
to support a BLA or NDA.
II. Participation
From January 2, 2024, to March 1,
2024, FDA will accept requests to
participate in the START Pilot Program
and will initially select up to three
participants in each Center. Taking into
consideration lessons and sponsors’
experiences from the initial iteration of
this program, a second iteration of the
pilot may be conducted to include more
participants in the future. At a later
date, FDA may also publish another
notice in the Federal Register to
announce a second iteration of the
program.
Sponsors who are interested in
participating in the START Pilot
Program should submit a request to
participate as an amendment to their
Investigational New Drug (IND)
application.
lotter on DSK11XQN23PROD with NOTICES1
A. Eligibility Criteria
To be considered for the START Pilot
Program, participants must meet the
following eligibility criteria:
1. Joint CBER and CDER Eligibility
Criteria
• IND has been submitted in or
converted to Electronic Common
Technical Document (eCTD) format,
unless the IND is of a type granted a
waiver from eCTD format (see FDA’s
VerDate Sep<11>2014
18:41 Sep 29, 2023
Jkt 262001
guidance for industry entitled
‘‘Providing Regulatory Submissions in
Electronic Format—Certain Human
Pharmaceutical Product Applications
and Related Submissions using the
eCTD Specifications’’ ((February 2020)
(Ref. 2))) and remains in active status.
• Sponsor has demonstrated
substantial effort to ensure that that
Chemistry, Manufacturing, and Controls
(CMC) development aligns with clinical
development, for example, through
documented control of manufacturing
and testing procedures to ensure clinical
and CMC development timeline are in
alignment.
Given the specific identified needs for
the products regulated by each Center
for rare diseases, the following
eligibility criteria differ between CBER
and CDER:
2. CBER-Specific Eligibility Criteria
• Existing OTP-regulated IND for a
cellular or gene therapy under which
the product is being developed toward
a marketing application.
• Such product is intended to address
an unmet medical need as a treatment
for a rare disease 1 or serious condition,
which is likely to lead to significant
disability or death within the first
decade of life.
3. CDER-Specific Eligibility Criteria
• Such product is intended to treat
rare neurodegenerative conditions
(including those of rare genetic
metabolic etiology).
B. What To Submit in a Request To
Participate in the START Pilot
To participate in the START Pilot
Program, sponsors should submit a
written request as an amendment to the
IND. The cover letter should (1) state
‘‘Request to participate in the START
Pilot Program’’, (2) note whether there is
a breakthrough therapy (BT) designation
for the product and for CBER-regulated
products only—whether there is a BT
designation and/or regenerative
medicine advanced therapy (RMAT)
designation, and (3) provide a point of
contact.
The request should include the initial
specific development issue(s) for a given
product for enhanced communication
and a proposed communication plan
between the sponsor and review staff. In
1 A rare disease or condition ‘‘means any disease
or condition which affects less than 200,000
persons in the United States . . .’’ (Section
526(a)(2) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360bb(a)(2))). The START Pilot
Program in CBER is not intended to encompass all
rare diseases, but only a subset of rare diseases that
are likely to lead to significant disability or death
within the first decade of life.
PO 00000
Frm 00038
Fmt 4703
Sfmt 4703
addition, the following information
should be provided:
1. Program development plan.
The plan should describe the current
state of program development, including
any ongoing activities not already
detailed in the IND.
• CMC development plan and current
status.
• Nonclinical development plan and
current status.
• Clinical development plan and
current status.
2. Any specific issue(s) (grouped by
review disciplines) for which the
prospective applicants are seeking
enhanced communications with FDA
review staff to facilitate program
development, including, for example, to
ensure a mutual understanding of
information needed to facilitate
initiating the pivotal clinical study or to
the pre-BLA/pre-NDA meeting stage.
3. The planned timeline for initiation
of the clinical study(ies) intended to
provide the primary evidence of
effectiveness to support a marketing
application or for a pre-BLA/pre-NDA
meeting request.
4. The proposed communication plan
for interactions between FDA review
staff and the sponsor, including the
proposed timing (i.e., month and year)
for the initial teleconference and format
(e.g., email or teleconference) of the
subsequent communications on a
scheduled and/or as needed basis.
C. Selection Criteria and Process
FDA intends to select participant
CBER and CDER INDs based on the
criteria outlined below. FDA will make
its determination of participants
following the close of the application
period. FDA intends to issue a letter to
notify each sponsor of FDA’s decision
on sponsor requests to participate
within 90 days of the application
deadline.
For the initial selection of up to three
INDs from each Center for the START
Pilot Program from eligible applicants,
FDA intends to consider factors such as:
(1) potential clinical benefits of the
product, (2) whether resolution of the
specific issues noted by the sponsor in
their request to participate in the pilot
could be facilitated through enhanced
communication to improve efficiency of
program development, (3) whether there
is an BT or RMAT designation for the
product, (4) whether CMC development
timeline aligns with clinical
development plans, and (5) while INDs
for combination products (21 CFR
3.2(e)(1)) may be eligible, products that
require significant cross-Center
interactions (e.g., complex combination
products) may be less likely to be
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Federal Register / Vol. 88, No. 189 / Monday, October 2, 2023 / Notices
https://www.fda.gov/media/135373/
download.
selected for the pilot. Overall, pilot
participants will be selected based on
application readiness (e.g., sponsors
who demonstrate having the ability to
move the program forward towards a
marketing application).
Dated: September 25, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–21235 Filed 9–29–23; 8:45 am]
D. FDA-Sponsor Interactions During the
START Pilot Program
If selected for the START Pilot
Program, sponsors will receive
enhanced communications with FDA
review staff. These enhanced
communications may vary between
CBER and CDER but will include at a
minimum an initial meeting to review
features of the pilot, discuss a pathway
intended to support a marketing
application, and to discuss specific
issues for which a sponsor requests
enhanced communication with FDA.
Additional communications will
include ongoing interactions via email
or teleconference that take place on a
scheduled and/or as needed basis as
agreed upon by the sponsor and FDA.
This notice refers to previously
approved FDA collections of
information. These collections of
information are subject to review by the
Office of Management and Budget
(OMB) under the Paperwork Reduction
Act of 1995 (44 U.S.C. 3501–3521). The
collections of information in 21 CFR
part 312 have been approved under
OMB control number 0910–0014 and
the collections of information in 21 CFR
part 601 have been approved under
OMB control number 0910–0338.
lotter on DSK11XQN23PROD with NOTICES1
IV. References
The following references are on
display at the Dockets Management Staff
(HFA–305), Food and Drug
Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852, 240–402–
7500, and are available for viewing by
interested persons between 9 a.m. and 4
p.m., Monday through Friday; they are
also available electronically at https://
www.regulations.gov. FDA has verified
the website addresses, as of the date this
document publishes in the Federal
Register, but websites are subject to
change over time.
1. FDA Draft Guidance for Industry
‘‘Formal Meetings Between the FDA and
Sponsors or Applicants of PDUFA
Products’’ (September 2023): https://
www.fda.gov/media/172311/download.
2. FDA Guidance for Industry
‘‘Providing Regulatory Submissions in
Electronic Format—Certain Human
Pharmaceutical Product Applications
and Related Submissions using the
eCTD Specifications’’ (February 2020):
18:41 Sep 29, 2023
Jkt 262001
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2022–D–0219]
Human Prescription Drug and
Biological Products—Labeling for
Dosing Based on Weight or Body
Surface Area for Ready-to-Use
Containers—‘‘Dose Banding’’;
Guidance for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘Human
Prescription Drug and Biological
Products—Labeling for Dosing Based on
Weight or Body Surface Area for Readyto-Use Containers—‘Dose Banding.’ ’’
The guidance is intended to assist
applicants in incorporating dose
banding information, based on dosing
information of a previously approved
drug product that is based on weight or
body surface area (BSA), into the
proposed labeling of injectable drug
products that are the subject of certain
marketing applications submitted to
FDA. This guidance finalizes the draft
guidance of the same title issued on July
21, 2022.
DATES: The announcement of the
guidance is published in the Federal
Register on October 2, 2023.
ADDRESSES: You may submit either
electronic or written comments on
Agency guidances at any time as
follows:
SUMMARY:
III. Paperwork Reduction Act of 1995
VerDate Sep<11>2014
BILLING CODE 4164–01–P
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
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67765
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–
2022–D–0219 for ‘‘Human Prescription
Drug and Biological Products—Labeling
for Dosing Based on Weight or Body
Surface Area for Ready-to-Use
Containers—‘Dose Banding.’ ’’ Received
comments 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
E:\FR\FM\02OCN1.SGM
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]]>Agencies
[Federal Register Volume 88, Number 189 (Monday, October 2, 2023)]
[Notices]
[Pages 67763-67765]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-21235]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-N-3976]
Support for Clinical Trials Advancing Rare Disease Therapeutics
Pilot Program; Program Announcement
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration's (FDA or Agency) Center for
Biologics Evaluation and Research's (CBER) Office of Therapeutic
Products (OTP) and Center for Drug Evaluation and Research's (CDER)
Office of New Drugs are announcing the opportunity for a limited number
of development programs to participate in the Support for clinical
Trials Advancing Rare disease Therapeutics (START) Pilot Program, with
the goal of further accelerating the pace of development of certain
CBER- and CDER-regulated products (novel drug and biological products)
that are intended to treat a rare disease. Because each Center has
identified specific needs concerning regulated products for rare
diseases, the eligibility criteria for the pilot differ between CBER
and CDER. This pilot would augment the currently available formal
meetings between FDA and sponsors by addressing issues related to the
development of individual products through more rapid, ad-hoc
communication mechanisms. Sponsors, if selected for the pilot, would
receive more frequent advice related to such specific issues through
additional interactions to facilitate novel drug and biological product
program development and generate high quality and reliable data
intended to support a Biologics License Application (BLA) or New Drug
Application (NDA). This notice outlines the eligibility criteria, what
to submit in a request to participate in the pilot, selection criteria,
process, and FDA-Sponsor interactions expected to occur for programs
participating in the pilot.
DATES: From January 2, 2024, to March 1, 2024, FDA will accept requests
to participate in the START Pilot Program and select no more than three
participants from each Center (CBER and CDER). See the
``Participation'' section for eligibility criteria, instructions on how
to submit a request to participate, and information regarding the
selection process.
FOR FURTHER INFORMATION CONTACT: Andrew Harvan, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7268, Silver Spring, MD 20993-0002, 240-
402-7911; or Quyen Tran, Center for Drugs Evaluation and Research, Food
and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6301,
Silver Spring, MD 20993-0002, 301-796-2771.
For general questions about the START Pilot Program for CBER:
[email protected]. For general questions about the START
Pilot Program for CDER: [email protected].
SUPPLEMENTARY INFORMATION:
I. Background
The purpose of the START Pilot Program is to further accelerate the
pace of development of novel drug and biological products that are
intended to address an unmet medical need as a treatment for a rare
disease. The pilot is designed to be milestone-driven (i.e., to
facilitate the progression of a development program to pivotal clinical
study stage or the pre-BLA or pre-NDA meeting stage) where product
development programs selected would benefit from enhanced
communications with FDA. Participation in the pilot will be considered
concluded when the development program has reached a significant
regulatory milestone such as initiation of the pivotal clinical study
stage or the pre-BLA or pre-NDA meeting stage as agreed upon with the
sponsor. Pilot participants will be selected based on demonstrated
development program readiness. The START Pilot Program is intended to
provide a mechanism for addressing clinical development issues that
otherwise would delay or prevent a promising novel drug or biological
product from progressing to the pivotal
[[Page 67764]]
clinical trial stage or pre-BLA/pre-NDA meeting stage.
The pilot would augment the currently available formal meetings
between FDA and sponsors (see FDA's draft guidance for industry
entitled ``Formal Meetings Between the FDA and Sponsors or Applicants
of PDUFA Products'' ((September 2023) (Ref. 1))) through more rapid,
ad-hoc communications with FDA by addressing issues with specific
programmatic needs for individual products. For example, these issues
can be related to clinical study design, choice of control group, fine-
tuning the choice of patient population, selecting appropriate
endpoints for efficacy trials to support marketing approval, selecting
statistical methodology, leveraging nonclinical information, or product
characterization. For eligible development programs sponsors and FDA
could benefit from such additional communication beyond the currently
available formal meeting mechanisms to address specific programmatic
needs that require in-depth discussions. The increased communication
between FDA review staff and sponsors is intended to facilitate program
development for specific products and to help generate high quality and
reliable data intended to support a BLA or NDA.
II. Participation
From January 2, 2024, to March 1, 2024, FDA will accept requests to
participate in the START Pilot Program and will initially select up to
three participants in each Center. Taking into consideration lessons
and sponsors' experiences from the initial iteration of this program, a
second iteration of the pilot may be conducted to include more
participants in the future. At a later date, FDA may also publish
another notice in the Federal Register to announce a second iteration
of the program.
Sponsors who are interested in participating in the START Pilot
Program should submit a request to participate as an amendment to their
Investigational New Drug (IND) application.
A. Eligibility Criteria
To be considered for the START Pilot Program, participants must
meet the following eligibility criteria:
1. Joint CBER and CDER Eligibility Criteria
IND has been submitted in or converted to Electronic
Common Technical Document (eCTD) format, unless the IND is of a type
granted a waiver from eCTD format (see FDA's guidance for industry
entitled ``Providing Regulatory Submissions in Electronic Format--
Certain Human Pharmaceutical Product Applications and Related
Submissions using the eCTD Specifications'' ((February 2020) (Ref. 2)))
and remains in active status.
Sponsor has demonstrated substantial effort to ensure that
that Chemistry, Manufacturing, and Controls (CMC) development aligns
with clinical development, for example, through documented control of
manufacturing and testing procedures to ensure clinical and CMC
development timeline are in alignment.
Given the specific identified needs for the products regulated by
each Center for rare diseases, the following eligibility criteria
differ between CBER and CDER:
2. CBER-Specific Eligibility Criteria
Existing OTP-regulated IND for a cellular or gene therapy
under which the product is being developed toward a marketing
application.
Such product is intended to address an unmet medical need
as a treatment for a rare disease \1\ or serious condition, which is
likely to lead to significant disability or death within the first
decade of life.
---------------------------------------------------------------------------
\1\ A rare disease or condition ``means any disease or condition
which affects less than 200,000 persons in the United States . . .''
(Section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 360bb(a)(2))). The START Pilot Program in CBER is not
intended to encompass all rare diseases, but only a subset of rare
diseases that are likely to lead to significant disability or death
within the first decade of life.
---------------------------------------------------------------------------
3. CDER-Specific Eligibility Criteria
Such product is intended to treat rare neurodegenerative
conditions (including those of rare genetic metabolic etiology).
B. What To Submit in a Request To Participate in the START Pilot
To participate in the START Pilot Program, sponsors should submit a
written request as an amendment to the IND. The cover letter should (1)
state ``Request to participate in the START Pilot Program'', (2) note
whether there is a breakthrough therapy (BT) designation for the
product and for CBER-regulated products only--whether there is a BT
designation and/or regenerative medicine advanced therapy (RMAT)
designation, and (3) provide a point of contact.
The request should include the initial specific development
issue(s) for a given product for enhanced communication and a proposed
communication plan between the sponsor and review staff. In addition,
the following information should be provided:
1. Program development plan.
The plan should describe the current state of program development,
including any ongoing activities not already detailed in the IND.
CMC development plan and current status.
Nonclinical development plan and current status.
Clinical development plan and current status.
2. Any specific issue(s) (grouped by review disciplines) for which
the prospective applicants are seeking enhanced communications with FDA
review staff to facilitate program development, including, for example,
to ensure a mutual understanding of information needed to facilitate
initiating the pivotal clinical study or to the pre-BLA/pre-NDA meeting
stage.
3. The planned timeline for initiation of the clinical study(ies)
intended to provide the primary evidence of effectiveness to support a
marketing application or for a pre-BLA/pre-NDA meeting request.
4. The proposed communication plan for interactions between FDA
review staff and the sponsor, including the proposed timing (i.e.,
month and year) for the initial teleconference and format (e.g., email
or teleconference) of the subsequent communications on a scheduled and/
or as needed basis.
C. Selection Criteria and Process
FDA intends to select participant CBER and CDER INDs based on the
criteria outlined below. FDA will make its determination of
participants following the close of the application period. FDA intends
to issue a letter to notify each sponsor of FDA's decision on sponsor
requests to participate within 90 days of the application deadline.
For the initial selection of up to three INDs from each Center for
the START Pilot Program from eligible applicants, FDA intends to
consider factors such as: (1) potential clinical benefits of the
product, (2) whether resolution of the specific issues noted by the
sponsor in their request to participate in the pilot could be
facilitated through enhanced communication to improve efficiency of
program development, (3) whether there is an BT or RMAT designation for
the product, (4) whether CMC development timeline aligns with clinical
development plans, and (5) while INDs for combination products (21 CFR
3.2(e)(1)) may be eligible, products that require significant cross-
Center interactions (e.g., complex combination products) may be less
likely to be
[[Page 67765]]
selected for the pilot. Overall, pilot participants will be selected
based on application readiness (e.g., sponsors who demonstrate having
the ability to move the program forward towards a marketing
application).
D. FDA-Sponsor Interactions During the START Pilot Program
If selected for the START Pilot Program, sponsors will receive
enhanced communications with FDA review staff. These enhanced
communications may vary between CBER and CDER but will include at a
minimum an initial meeting to review features of the pilot, discuss a
pathway intended to support a marketing application, and to discuss
specific issues for which a sponsor requests enhanced communication
with FDA. Additional communications will include ongoing interactions
via email or teleconference that take place on a scheduled and/or as
needed basis as agreed upon by the sponsor and FDA.
III. Paperwork Reduction Act of 1995
This notice refers to previously approved FDA collections of
information. These collections of information are subject to review by
the Office of Management and Budget (OMB) under the Paperwork Reduction
Act of 1995 (44 U.S.C. 3501-3521). The collections of information in 21
CFR part 312 have been approved under OMB control number 0910-0014 and
the collections of information in 21 CFR part 601 have been approved
under OMB control number 0910-0338.
IV. References
The following references are on display at the Dockets Management
Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm.
1061, Rockville, MD 20852, 240-402-7500, and are available for viewing
by interested persons between 9 a.m. and 4 p.m., Monday through Friday;
they are also available electronically at https://www.regulations.gov.
FDA has verified the website addresses, as of the date this document
publishes in the Federal Register, but websites are subject to change
over time.
1. FDA Draft Guidance for Industry ``Formal Meetings Between the
FDA and Sponsors or Applicants of PDUFA Products'' (September 2023):
https://www.fda.gov/media/172311/download.
2. FDA Guidance for Industry ``Providing Regulatory Submissions in
Electronic Format--Certain Human Pharmaceutical Product Applications
and Related Submissions using the eCTD Specifications'' (February
2020): https://www.fda.gov/media/135373/download.
Dated: September 25, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-21235 Filed 9-29-23; 8:45 am]
BILLING CODE 4164-01-P
