Public Workshop on Optimizing the Use of Real-World Evidence in Regulatory Decision-Making for Drugs and Biological Products-Looking Forward; Request for Comments, 89646-89647 [2024-26297]
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<(1) and Sec. 811 [42 U.S.C. 2992].
Mary C. Jones,
ACF/OPRE Certifying Officer.
[FR Doc. 2024–26230 Filed 11–12–24; 8:45 am]
BILLING CODE 4184–34–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2024–N–5057]
Public Workshop on Optimizing the
Use of Real-World Evidence in
Regulatory Decision-Making for Drugs
and Biological Products—Looking
Forward; Request for Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public workshop;
request for comments.
ACTION:
The Food and Drug
Administration (FDA or Agency) is
announcing a public workshop titled
‘‘Optimizing the Use of Real-World
Evidence in Regulatory DecisionMaking for Drugs and Biological
Products—Looking Forward.’’ The
purpose of the public workshop is to
provide interested parties with an
update on FDA’s current activities
related to real-world evidence (RWE)
and to share accomplishments, ongoing
challenges, and future opportunities.
The public workshop will discuss
potential next steps to promote the
continued evolution and consistent
application of real-world data (RWD) in
drug development. This public
workshop will be convened and
supported by a cooperative agreement
between FDA and the Duke University,
Duke-Margolis Institute for Health
Policy.
DATES: The public workshop will be
held on December 12, 2024, from 12:30
p.m. to 5 p.m., Eastern Time. Either
electronic or written comments on this
public workshop must be submitted by
January 13, 2025. See the
SUPPLEMENTARY INFORMATION section for
registration date and information.
ADDRESSES: The public workshop will
be held in-person at the Bethesda North
Marriott & Conference Center, 5701
Marinelli Rd., North Bethesda, MD
20852 and virtually using the Zoom
platform.
You may submit comments as
follows. Please note that late, untimely
filed comments will not be considered.
lotter on DSK11XQN23PROD with NOTICES1
SUMMARY:
VerDate Sep<11>2014
17:40 Nov 12, 2024
Jkt 265001
The https://www.regulations.gov
electronic filing system will accept
comments until 11:59 p.m. Eastern Time
at the end of January 13, 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:
• 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.
• 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–
2024–N–5057 for ‘‘Optimizing the Use
of Real-World Evidence in Regulatory
Decision-Making for Drugs and
Biological Products—Looking Forward.’’
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
PO 00000
Frm 00078
Fmt 4703
Sfmt 4703
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-201509-18/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:
Dianne Paraoan, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD
20993, 301–796–3161,
Dianne.Paraoan@fda.hhs.gov or CDERRWE@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
The volume and complexity of RWD
available to support drug development
have increased substantially over the
past several decades. This increase,
combined with enhanced computing
power and emerging technologies, is
transforming how drugs are developed.
E:\FR\FM\13NON1.SGM
13NON1
Federal Register / Vol. 89, No. 219 / Wednesday, November 13, 2024 / Notices
lotter on DSK11XQN23PROD with NOTICES1
FDA published a ‘‘Framework for FDA’s
Real-World Evidence Program’’
(Framework) (https://www.fda.gov/
media/120060/download?attachment)
for drugs and biological products in
2018. This Framework describes a
multifaceted FDA program to evaluate
the potential use of RWE in regulatory
decision-making to help support the
approval of a new indication for a drug
already approved under section 505(c)
of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355(c)) or to help support
or satisfy drug post-approval study
requirements. As described below, the
growing reliance on RWD to assess
pharmaceutical use, safety and
effectiveness, and use of new
technologies in these analyses will
influence the generation of RWE to
support regulatory decision-making.
FDA’s RWE Program for drugs and
biological products involves multiple
components: guidance development to
assist developers interested in using
RWD to develop RWE and to support
Agency decisions; internal processes
that involve senior leadership in the
evaluation of RWE and promote shared
learning and consistency in applying
the Framework; demonstration projects
with a focus on evaluating/improving
data quality and use of RWD, advancing
study design, and developing rigorous
evaluation tools; and external
engagement, including listening
sessions, presentations, publications,
and international collaborations. In
addition, the seventh iteration of the
Prescription Drug User Fees Act
included new RWE-related provisions,
including the Advancing RWE Program
that enables early discussions with
sponsors regarding RWE-based study
proposals and greater transparency
around the submission of RWE to the
Center for Drug Evaluation and Research
(CDER) and the Center for Biologics
Evaluation and Research. In related
work, FDA has published guidance
documents and/or discussion papers on
digital health technologies and the use
of artificial intelligence (AI).
These activities lay a foundation for
expansion of the use of RWD in
evidence generation, but continued
growth of these efforts requires
increased coordination, knowledge
management, internal support
(including training), external visibility,
and external engagement.
II. Topics for Discussion at the Public
Workshop
FDA supports the continued
evolution of using RWD to generate
RWE that can support pre- and postmarket regulatory decision-making at
FDA for drugs and biological products.
VerDate Sep<11>2014
17:40 Nov 12, 2024
Jkt 265001
Topics for this public workshop will
focus on FDA’s current activities around
RWE, ongoing accomplishments and
challenges, and future opportunities.
Advancing existing priorities and
activities of the RWE Program remains
a focus area, including promoting
consistency in review processes of
submissions that contain RWE, and
leveraging the full potential of RWD as
well as emerging technologies to inform
the effectiveness and safety of drugs and
biological products.
To help facilitate the future direction
of the RWE Program, FDA seeks input
on the following questions.
1. Regulators, sponsors, and other
interested parties are gaining experience
with RWE in regulatory submissions.
What are critical issues that need to be
addressed to further advance the use of
RWE in regulatory decision-making for
drugs and biological products?
2. To advance our understanding of
RWE, FDA has funded various
demonstration (research) projects on
topics such as RWD sources, study
designs, and specific ‘‘tools.’’ What
research priorities, including emerging
technologies and AI, should CDER
consider supporting?
3. FDA has published RWD/RWE
guidance documents focused on data
considerations, study design, and
regulatory considerations. What
additional topics could be prioritized
for consideration?
4. FDA has utilized various
mechanisms (e.g., public meetings,
webinars, ‘‘listening sessions’’) to
engage interested parties; the Agency
has also facilitated discussions with
international regulators. What are
optimal communication and
engagement strategies to interact with
the external community regarding RWE?
III. Participating in the Public
Workshop
Registration: To register for this
hybrid public workshop, please visit the
following website: https://duke.is/6/
y7t5. Please register for either in-person
or virtual attendance and provide
complete contact information for each
attendee, including name, title,
affiliation, address, email, and
telephone number.
Registration is free and based on
space availability, with priority given to
early registrants. Persons interested in
attending this public workshop inperson must register by December 6,
2024, 11:59 p.m. Eastern Time. Early
registration is recommended because
seating is limited; therefore, FDA may
limit the number of participants from
each organization. Registrants will
PO 00000
Frm 00079
Fmt 4703
Sfmt 4703
89647
receive confirmation when they have
been accepted.
If you need special accommodations
due to a disability, please contact Luke
Durocher at luke.durocher@duke.edu no
later than December 6, 2024.
Streaming of the Public Workshop:
This public workshop will also be
available via Zoom webinar to virtual
attendees who register at https://
duke.is/6/y7t5. For more information
about Zoom, please visit https://
support.zoom.us/hc/en-us/articles/
206175806-Frequently-asked-questions.
Notice of this workshop is given
pursuant to 21 CFR 10.65.
Dated: November 6, 2024.
Kimberlee Trzeciak,
Deputy Commissioner for Policy, Legislation,
and International Affairs.
[FR Doc. 2024–26297 Filed 11–12–24; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2024–P–2514]
Determination That NUPLAZID
(Pimavanserin Tartrate) Tablet,
Equivalent 17 Milligram Base, Was Not
Withdrawn From Sale for Reasons of
Safety or Effectiveness
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA or Agency) has
determined that NUPLAZID
(pimavanserin tartrate) tablet,
equivalent (EQ) 17 milligram (mg) base,
was not withdrawn from sale for reasons
of safety or effectiveness. This
determination will allow FDA to
approve abbreviated new drug
applications (ANDAs) for NUPLAZID
(pimavanserin tartrate) tablet, EQ 17 mg
base, if all other legal and regulatory
requirements are met.
FOR FURTHER INFORMATION CONTACT:
Stacy Kane, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 51, Rm. 6236, Silver Spring,
MD 20993–0002, 301–796–8363,
Stacy.Kane@fda.hhs.gov.
SUPPLEMENTARY INFORMATION: Section
505(j) of the Federal Food, Drug, and
Cosmetic Act (FD&C Act) (21 U.S.C.
355(j)) allows the submission of an
ANDA to market a generic version of a
previously approved drug product. To
obtain approval, the ANDA applicant
must show, among other things, that the
SUMMARY:
E:\FR\FM\13NON1.SGM
13NON1
]]>Agencies
[Federal Register Volume 89, Number 219 (Wednesday, November 13, 2024)]
[Notices]
[Pages 89646-89647]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-26297]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-N-5057]
Public Workshop on Optimizing the Use of Real-World Evidence in
Regulatory Decision-Making for Drugs and Biological Products--Looking
Forward; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
a public workshop titled ``Optimizing the Use of Real-World Evidence in
Regulatory Decision-Making for Drugs and Biological Products--Looking
Forward.'' The purpose of the public workshop is to provide interested
parties with an update on FDA's current activities related to real-
world evidence (RWE) and to share accomplishments, ongoing challenges,
and future opportunities. The public workshop will discuss potential
next steps to promote the continued evolution and consistent
application of real-world data (RWD) in drug development. This public
workshop will be convened and supported by a cooperative agreement
between FDA and the Duke University, Duke-Margolis Institute for Health
Policy.
DATES: The public workshop will be held on December 12, 2024, from
12:30 p.m. to 5 p.m., Eastern Time. Either electronic or written
comments on this public workshop must be submitted by January 13, 2025.
See the SUPPLEMENTARY INFORMATION section for registration date and
information.
ADDRESSES: The public workshop will be held in-person at the Bethesda
North Marriott & Conference Center, 5701 Marinelli Rd., North Bethesda,
MD 20852 and virtually using the Zoom platform.
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 at the end of January 13, 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:
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.
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-2024-N-5057 for ``Optimizing the Use of Real-World Evidence in
Regulatory Decision-Making for Drugs and Biological Products--Looking
Forward.'' 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: Dianne Paraoan, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993, 301-796-3161,
[email protected] or [email protected].
SUPPLEMENTARY INFORMATION:
I. Background
The volume and complexity of RWD available to support drug
development have increased substantially over the past several decades.
This increase, combined with enhanced computing power and emerging
technologies, is transforming how drugs are developed.
[[Page 89647]]
FDA published a ``Framework for FDA's Real-World Evidence Program''
(Framework) (https://www.fda.gov/media/120060/download?attachment) for
drugs and biological products in 2018. This Framework describes a
multifaceted FDA program to evaluate the potential use of RWE in
regulatory decision-making to help support the approval of a new
indication for a drug already approved under section 505(c) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(c)) or to help
support or satisfy drug post-approval study requirements. As described
below, the growing reliance on RWD to assess pharmaceutical use, safety
and effectiveness, and use of new technologies in these analyses will
influence the generation of RWE to support regulatory decision-making.
FDA's RWE Program for drugs and biological products involves
multiple components: guidance development to assist developers
interested in using RWD to develop RWE and to support Agency decisions;
internal processes that involve senior leadership in the evaluation of
RWE and promote shared learning and consistency in applying the
Framework; demonstration projects with a focus on evaluating/improving
data quality and use of RWD, advancing study design, and developing
rigorous evaluation tools; and external engagement, including listening
sessions, presentations, publications, and international
collaborations. In addition, the seventh iteration of the Prescription
Drug User Fees Act included new RWE-related provisions, including the
Advancing RWE Program that enables early discussions with sponsors
regarding RWE-based study proposals and greater transparency around the
submission of RWE to the Center for Drug Evaluation and Research (CDER)
and the Center for Biologics Evaluation and Research. In related work,
FDA has published guidance documents and/or discussion papers on
digital health technologies and the use of artificial intelligence
(AI).
These activities lay a foundation for expansion of the use of RWD
in evidence generation, but continued growth of these efforts requires
increased coordination, knowledge management, internal support
(including training), external visibility, and external engagement.
II. Topics for Discussion at the Public Workshop
FDA supports the continued evolution of using RWD to generate RWE
that can support pre- and post-market regulatory decision-making at FDA
for drugs and biological products. Topics for this public workshop will
focus on FDA's current activities around RWE, ongoing accomplishments
and challenges, and future opportunities.
Advancing existing priorities and activities of the RWE Program
remains a focus area, including promoting consistency in review
processes of submissions that contain RWE, and leveraging the full
potential of RWD as well as emerging technologies to inform the
effectiveness and safety of drugs and biological products.
To help facilitate the future direction of the RWE Program, FDA
seeks input on the following questions.
1. Regulators, sponsors, and other interested parties are gaining
experience with RWE in regulatory submissions. What are critical issues
that need to be addressed to further advance the use of RWE in
regulatory decision-making for drugs and biological products?
2. To advance our understanding of RWE, FDA has funded various
demonstration (research) projects on topics such as RWD sources, study
designs, and specific ``tools.'' What research priorities, including
emerging technologies and AI, should CDER consider supporting?
3. FDA has published RWD/RWE guidance documents focused on data
considerations, study design, and regulatory considerations. What
additional topics could be prioritized for consideration?
4. FDA has utilized various mechanisms (e.g., public meetings,
webinars, ``listening sessions'') to engage interested parties; the
Agency has also facilitated discussions with international regulators.
What are optimal communication and engagement strategies to interact
with the external community regarding RWE?
III. Participating in the Public Workshop
Registration: To register for this hybrid public workshop, please
visit the following website: https://duke.is/6/y7t5. Please register
for either in-person or virtual attendance and provide complete contact
information for each attendee, including name, title, affiliation,
address, email, and telephone number.
Registration is free and based on space availability, with priority
given to early registrants. Persons interested in attending this public
workshop in-person must register by December 6, 2024, 11:59 p.m.
Eastern Time. Early registration is recommended because seating is
limited; therefore, FDA may limit the number of participants from each
organization. Registrants will receive confirmation when they have been
accepted.
If you need special accommodations due to a disability, please
contact Luke Durocher at [email protected] no later than December
6, 2024.
Streaming of the Public Workshop: This public workshop will also be
available via Zoom webinar to virtual attendees who register at https://duke.is/6/y7t5. For more information about Zoom, please visit https://support.zoom.us/hc/en-us/articles/206175806-Frequently-asked-questions.
Notice of this workshop is given pursuant to 21 CFR 10.65.
Dated: November 6, 2024.
Kimberlee Trzeciak,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2024-26297 Filed 11-12-24; 8:45 am]
BILLING CODE 4164-01-P
