Notice2024-19712
Identifying Priority Focus Areas for Future Guidance Development and Engagement With Interested Parties in Model-Informed Drug Development; Request for Information
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Published
September 3, 2024
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) within the Food and Drug Administration (FDA or Agency) are announcing a request for information (RFI) for advancing model-informed drug development (MIDD). The purpose of this request is to obtain feedback on how to increase application of established MIDD approaches in regulatory decision making, to identify how emerging MIDD approaches are being incorporated within drug product development, and to identify opportunities to enhance interactions with FDA when discussing MIDD approaches. We intend to use the information submitted in response to this request to identify and prioritize potential focus areas for future policy or guidance development and enhance engagement with interested parties, including interactions as part of the MIDD Paired Meeting Program and other formal meetings with drug developers.
Full Text
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<title>Federal Register, Volume 89 Issue 170 (Tuesday, September 3, 2024)</title>
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[Federal Register Volume 89, Number 170 (Tuesday, September 3, 2024)]
[Notices]
[Pages 71374-71376]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-19712]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-N-3904]
Identifying Priority Focus Areas for Future Guidance Development
and Engagement With Interested Parties in Model-Informed Drug
Development; Request for Information
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice; request for Information.
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SUMMARY: The Center for Drug Evaluation and Research (CDER) and Center
for Biologics Evaluation and Research (CBER) within the Food and Drug
Administration (FDA or Agency) are announcing a request for information
(RFI) for advancing model-informed drug development (MIDD). The purpose
of this request is to obtain feedback on how to increase application of
established MIDD approaches in regulatory decision making, to identify
how emerging MIDD approaches are being incorporated within drug product
development, and to identify opportunities to enhance interactions with
FDA when discussing MIDD approaches. We intend to use the information
submitted in response to this request to identify and prioritize
potential focus areas for future policy or guidance development and
enhance engagement with interested parties, including interactions as
part of the
[[Page 71375]]
MIDD Paired Meeting Program and other formal meetings with drug
developers.
DATES: Either electronic or written comments on the notice must be
submitted by November 4, 2024.
ADDRESSES: 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 at the end of November 4, 2024. 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-2024-N-3904 for ``Identifying Priority Focus Areas for Future
Guidance Development and Engagement with Interested Parties in Model-
Informed Drug Development; Request for Information.'' 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: Yvonne Knight, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 2142, Silver Spring, MD 20993, 301-796-
2133, <a href="/cdn-cgi/l/email-protection#623b140d0c0c074c290c0b050a16220406034c0a0a114c050d14"><span class="__cf_email__" data-cfemail="1b426d7475757e355075727c736f5b7d7f7a35737368357c746d">[email protected]</span></a>, with the subject line ``MIDD Meetings
Program for CDER''; or Christopher Egelebo, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 5340, Silver Spring, MD 20993, 240-402-
8625, <a href="/cdn-cgi/l/email-protection#286b405a415b5c4758404d5a066d4f4d444d4a47684e4c490640405b064f475e"><span class="__cf_email__" data-cfemail="7e3d160c170d0a110e161b0c503b191b121b1c113e181a1f5016160d50191108">[email protected]</span></a>, with the subject line ``MIDD
Meetings Program for CBER.''
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing a request for information entitled ``Identifying
Priority Focus Areas for Future Guidance Development and Engagement
with Interested Parties in Model-Informed Drug Development.''
Information submitted in response to this notice will be used by CDER,
including by its Quantitative Medicine Center of Excellence, and CBER
to assist in identifying and prioritizing potential focus areas for
future policy or guidance development and engagement with interested
parties.
MIDD approaches integrate exposure-based biological and statistical
models derived from nonclinical and clinical data sources in drug
development and decision making. MIDD applications span the life cycle
of new drug product development. MIDD approaches use a variety of
quantitative methods (e.g., population pharmacokinetic (popPK)
modeling, exposure-response (E-R) modeling, physiologically based
pharmacokinetic (PBPK) modeling, systems pharmacology/mechanistic
modeling, disease progression modeling, drug-trial-disease modeling and
simulation, artificial intelligence/machine learning (AI/ML)
approaches) to help assess the risks and benefits of drug products,
contribute to the evidentiary framework for efficacy and/or safety, and
optimize dosing regimens for patients, among other applications. When
successfully applied, MIDD approaches might reduce animal testing,
improve clinical trial design and efficiency, inform identification of
dosing regimens with improved benefit-risk profiles, increase the
probability of regulatory success through synergetic engagement with
interested parties, and optimize drug dosing/therapeutic
individualization in the absence of dedicated trials.
Beginning with Prescription Drug User Fee Act (PDUFA) VI, FDA began
granting focused meetings as part of the MIDD Paired Meeting Pilot to:
(1) provide a forum for regulatory interaction between drug developers
and FDA on the application of MIDD approaches in specific drug
development programs; and (2) provide
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an opportunity for FDA to provide advice regarding how particular MIDD
approaches can be used in a specific drug development program. Other
deliverables as part of PDUFA VI included increasing regulatory science
and review capacity in MIDD approaches and convening multiple workshops
to identify best practices for MIDD (topics including E-R, PBPK,
disease progression modeling, and immunogenicity assessments). In
addition, FDA published or revised multiple guidances on MIDD. As part
of PDUFA VII, the MIDD Paired Meeting Program has been continued and
this RFI is to elicit public input on future focus areas for advancing
MIDD. More information on the MIDD Paired Meeting program can be found
at <a href="https://www.fda.gov/drugs/development-resources/model-informed-drug-development-paired-meeting-program">https://www.fda.gov/drugs/development-resources/model-informed-drug-development-paired-meeting-program</a>.
II. Request for Information
FDA is interested in detailed comments on the topics listed in this
section below to identify and inform future priorities for MIDD-related
policy, including guidance development and engagement with interested
parties. The topics identified in this section are not meant to be
exhaustive. FDA is also interested in any other pertinent information
that interested parties would like to share related to guidance and
enhancing MIDD-related interactions with FDA. FDA encourages interested
parties to provide the specific rationale and basis for their comments,
including any available supporting data and information.
A. Methods and Best Practices
Several quantitative approaches, such as popPK, E-R, and PBPK, are
routinely employed in drug development and regulatory assessment. The
Agency aims to identify areas within these approaches that would
benefit from the development of additional policies or guidance on
methodology and best practices. In addition, with this RFI, the Agency
is seeking input to explore potential guidance needs and appropriately
identify and prioritize potential topics for guidance development in
all emerging MIDD approaches for drug and biological products,
including but not limited to, AI/ML used in both drug design and
evaluation and digital-twin technology.
B. Context-Specific Considerations
MIDD approaches that leverage comprehensive information--including
disease and patient population characteristics (e.g., intrinsic and
extrinsic factors), drug properties, placebo effects, nonclinical and
clinical E-R relationships--are potent tools and can be utilized across
all stages of the drug development life cycle to support decision
making. This is particularly important for rare diseases and emerging
therapeutic and prophylactic/preventative modalities where there may be
practical and ethical challenges in conducting traditional drug
development programs or where there is limited drug development
experience. We seek input on the need to develop guidances that discuss
considerations to facilitate MIDD methods development, application,
uptake, and acceptance in specific therapeutic areas. Related topics
include identification of opportunities for incorporation of real-world
data, specific therapeutic modality considerations, and preclinical to
clinical translations and to appropriately identify and prioritize
potential topics in this area.
C. Regulatory Engagement
Building on the success of the MIDD Paired Meeting Program, FDA is
interested in better understanding ways to facilitate discussion around
MIDD approaches outside the MIDD Paired Meeting Program as part of
regulatory meetings and regulatory submissions. This includes
identifying what is currently working well and what are the barriers
(e.g., technical, regulatory) encountered while trying to interact with
FDA on MIDD-related activities.
D. Communication of Policies and Interested Parties' Engagement
FDA continues to engage on MIDD approaches as part of external
workshops with interested parties, including workshops described and
completed under PDUFA VI. FDA seeks to identify and prioritize
potential topics and better ways for communication and engagement with
interested parties.
Dated: August 28, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-19712 Filed 8-30-24; 8:45 am]
BILLING CODE 4164-01-P
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