[Federal Register Volume 88, Number 11 (Wednesday, January 18, 2023)]
[Notices]
[Pages 2933-2934]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-00840]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2022-N-3299]


Understanding the Use of Negative Controls To Assess the Validity 
of Non-Interventional Studies of Treatment Using Real-World Evidence; 
Public Workshop; 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 
the following public workshop entitled ``Understanding the Use of 
Negative Controls to Assess the Validity of Non-Interventional Studies 
of Treatment Using Real-World Evidence.'' Convened by the Duke-Margolis 
Center for Health Policy and supported by a cooperative agreement 
between FDA and Duke-Margolis, the purpose of this public workshop is 
to discuss existing negative control methodologies for studies based on 
real-world data and to outline advantages and disadvantages of the use 
of negative controls for evaluating the safety and effectiveness of 
regulated medical products. Additionally, the Center for Drug 
Evaluation and Research and the Center for Biologics Evaluation and 
Research will propose projects to develop negative control methods and 
implement new tools for use in the Sentinel System, Biologics 
Effectiveness and Safety (BEST) System, and with Federal Partners.

DATES: The public workshop will be virtually convened on March 8, 2023, 
from 10 a.m. to 3 p.m., Eastern Standard Time. Either electronic or 
written comments on this public workshop must be submitted by May 8, 
2023. See the SUPPLEMENTARY INFORMATION section for registration date 
and information.

ADDRESSES: The public workshop will be held virtually using the Zoom 
Platform. The link for the public workshop can be accessed at the 
following web page: https://duke.is/cy9w4.
    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 May 8, 2023. 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

[[Page 2934]]

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-N-3299 for ``Understanding the Use of Negative Controls to 
Assess the Validity of Non-Interventional Studies of Treatment Using 
Real-World Evidence.'' Received comments, those filed in a timely 
manner, 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. 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: Jamila Mwidau, Food and Drug 
Administration, Center for Drug Evaluation and Research, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 4481, Silver Spring, MD 20993, 301-796-
4989, [email protected]; or Stephen Ripley, Center for 
Biologics Evaluation and Research, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993, 240-
402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    In connection with the seventh iteration of the Prescription Drug 
User Fee Amendments (PDUFA VII), incorporated as part of the FDA User 
Fee Reauthorization Act of 2022, FDA has committed to enhancing and 
modernizing the FDA drug safety system, including improving the utility 
of existing tools and adopting new scientific approaches. This 
commitment includes optimizing the capabilities of the Sentinel 
Initiative to address questions of product safety and advance the 
understanding of how Real-World Evidence can be used for studying 
effectiveness.
    Under PDUFA VII, FDA agreed to conduct a public workshop by 
September 30, 2023, on the use of negative controls for assessing the 
validity of non-interventional studies of treatment. This public 
workshop, scheduled for March 8, 2023, will satisfy the PDUFA VII 
commitment. One purpose of the public workshop is to discuss current 
negative control methods in studies based on real-world data and 
discuss future implications for their use to evaluate the safety of 
regulated medical products. Another purpose of the public workshop is 
to present the proposed methods development projects that may support a 
tool for use in the Sentinel System and BEST.

II. Topics for Discussion at the Public Workshop

    Some topics FDA plans to discuss at the public workshop include but 
may not be limited to the following:
    1. What are the strengths and limitations of current negative 
control methods used in studies based on real-world data?
    2. What are known and potential benefits and challenges in using 
negative controls in evaluating regulated product safety and 
effectiveness?
    3. What additional information is needed about negative control 
methods to provide confidence regarding their use in regulatory 
decision-making?

III. Participating in the Public Workshop

    Registration: To register for the public workshop, please visit the 
following website to register: https://duke.is/cy9w4. Please provide 
complete contact information for each attendee, including name, title, 
affiliation, address, email, and telephone.
    Registration is free and open until the public workshop is 
completed. Persons interested in attending this public workshop can 
register until 2:59 p.m. Eastern Standard Time on March 8, 2023.
    If you need special accommodations due to a disability, please 
contact [email protected] no later than February 22, 2023.

    Dated: January 12, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-00840 Filed 1-17-23; 8:45 am]
BILLING CODE 4164-01-P