Notice2023-00840
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
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Published
January 18, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
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.
Full Text
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<title>Federal Register, Volume 88 Issue 11 (Wednesday, January 18, 2023)</title>
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[Federal Register Volume 88, Number 11 (Wednesday, January 18, 2023)]
[Notices]
[Pages 2933-2934]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-00840]
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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.
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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: <a href="https://duke.is/cy9w4">https://duke.is/cy9w4</a>.
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 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:
<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
[[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 <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-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 <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. 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: 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, <a href="/cdn-cgi/l/email-protection#db91bab6b2b7baf596acb2bfbaae9bbdbfbaf5b3b3a8f5bcb4ad"><span class="__cf_email__" data-cfemail="db91bab6b2b7baf596acb2bfbaae9bbdbfbaf5b3b3a8f5bcb4ad">[email protected]</span></a>; 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: <a href="https://duke.is/cy9w4">https://duke.is/cy9w4</a>. 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 <a href="/cdn-cgi/l/email-protection#4c212d3e2b2320253f293a2922383f0c2839272962292839"><span class="__cf_email__" data-cfemail="a3cec2d1c4cccfcad0c6d5c6cdd7d0e3c7d6c8c68dc6c7d6">[email protected]</span></a> 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
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