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[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]

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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.

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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
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