Notice2023-28289
Real-World Data: Assessing Registries To Support Regulatory Decision-Making for Drug and Biological Products; Guidance for Industry; Availability
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
December 22, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products." This guidance provides considerations for sponsors proposing to design a registry or to use an existing registry to support regulatory decision-making about a drug's effectiveness or safety. FDA is issuing this guidance as part of its Real-World Evidence (RWE) Program and to satisfy, in part, the mandate under the Federal Food, Drug, and Cosmetic Act (FD&C Act) to issue guidance on the use of RWE in regulatory decision-making. This guidance finalizes the draft guidance of the same title issued on November 30, 2021.
Full Text
<html>
<head>
<title>Federal Register, Volume 88 Issue 245 (Friday, December 22, 2023)</title>
</head>
<body><pre>
[Federal Register Volume 88, Number 245 (Friday, December 22, 2023)]
[Notices]
[Pages 88631-88633]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-28289]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-1146]
Real-World Data: Assessing Registries To Support Regulatory
Decision-Making for Drug and Biological Products; Guidance for
Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a final guidance for industry entitled ``Real-World
Data: Assessing Registries to Support Regulatory Decision-Making for
Drug and Biological Products.'' This guidance provides considerations
for sponsors proposing to design a registry or to use an existing
registry to support regulatory decision-making about a drug's
effectiveness or safety. FDA is issuing this guidance as part of its
Real-World Evidence (RWE) Program and to satisfy, in part, the mandate
under the Federal Food, Drug, and Cosmetic Act (FD&C Act) to issue
guidance on the use of RWE in regulatory decision-making. This guidance
finalizes the draft guidance of the same title issued on November 30,
2021.
DATES: The announcement of the guidance is published in the Federal
Register on December 22, 2023.
ADDRESSES: You may submit either electronic or written comments on
Agency guidances at any time as follows:
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-2021-D-1146 for ``Real-World Data: Assessing Registries to Support
Regulatory Decision-Making for Drug and Biological Products.'' Received
comments 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
[[Page 88632]]
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.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single copies of this guidance to the
Division of Drug Information, Center for Drug Evaluation and Research,
Food and Drug Administration, 10001 New Hampshire Ave., Hillandale
Building, 4th Floor, Silver Spring, MD 20993-0002, or the Office of
Communication, Outreach and Development, Center for Biologics
Evaluation and Research (CBER), Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send
one self-addressed adhesive label to assist that office in processing
your requests. See the SUPPLEMENTARY INFORMATION section for electronic
access to the guidance document.
FOR FURTHER INFORMATION CONTACT: Dianne Paraoan, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 3226, Silver Spring, MD 20993-0002, 301-
796-3161, or James Myers, Center for Biologics Evaluation and Research,
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm.
7301, Silver Spring, MD 20993-0002, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry
entitled ``Real-World Data: Assessing Registries to Support Regulatory
Decision-Making for Drug and Biological Products.'' FDA is issuing this
guidance as part of its RWE Program and to satisfy, in part, the
mandate under section 505F of the FD&C Act (21 U.S.C. 355g) to issue
final guidance about the use of RWE in regulatory decision-making.
Topics covered in this guidance include:
<bullet> Considerations regarding a registry's fitness-for-use in
regulatory decision-making, focusing on attributes of a registry that
support the collection of relevant and reliable data;
<bullet> Considerations when linking a registry to another data
source for supplemental information, such as data from medical claims,
electronic health records, digital health technologies, or other
registries; and
<bullet> Considerations for supporting FDA review of submissions
that include registry data.
Section 3022 of the 21st Century Cures Act (Cures Act) (Pub. L.
114-255) amended the FD&C Act to add section 505F, Utilizing Real World
Evidence. In addition, the Prescription Drug User Fee Amendments of
2017 (PDUFA VI) committed FDA to publish draft guidance on how RWE can
contribute to the assessment of safety and effectiveness in regulatory
submissions. In 2018, FDA created an RWE Framework and Program to
evaluate the potential use of RWE to help support the approval of a new
indication for a drug already approved under the FD&C Act or to help
support or satisfy postapproval study requirements. In late 2021, FDA
utilized the RWE Program to issue draft guidances outlining
considerations for the use of real-world data and RWE in regulatory
decision-making to satisfy the Cures Act mandate and the PDUFA VI
commitment.
This guidance finalizes the draft guidance of the same title issued
on November 30, 2021 (86 FR 67956). FDA considered comments received on
the draft guidance as the guidance was finalized. Changes from the
draft to the final guidance include noting that sponsors proposing to
use registry data to support regulatory decision-making by FDA are
responsible for ensuring that attributes of the registry support the
collection of relevant and reliable data, including in situations where
the data are from a registry not managed or designed by the sponsor,
and sponsors should have access to the metadata associated with the
registry data. In addition, statements were added to note that registry
data are sometimes used to evaluate a drug received during routine
medical practice, such as to evaluate clinical outcomes in populations
underrepresented in clinical trials, to note that registries should
have a plan to reduce missing assessments and minimize loss to followup
of participants, and to provide additional considerations related to
linkage to other data sources. Examples of pregnancy-related
information that may be collected by a registry were removed because
this information is addressed in a separate guidance. Terms that may be
defined differently by different stakeholders were removed from the
guidance if they were not necessary to understand the content of the
guidance. Other relevant definitions were transferred from the glossary
to the text. In addition, editorial changes were made to improve
clarity.
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Real-World Data: Assessing Registries to
Support Regulatory Decision-Making for Drug and Biological Products.''
It does not establish any rights for any person and is not binding on
FDA or the public. You can use an alternative approach if it satisfies
the requirements of the applicable statutes and regulations.
II. Paperwork Reduction Act of 1995
While this guidance contains no collection of information, it does
refer to previously approved FDA collections of information. The
previously approved collections of information are subject to review by
the Office of Management and Budget (OMB) under the Paperwork Reduction
Act of 1995 (44 U.S.C. 3501-3521). The collections of information in 21
CFR part 11 have been approved under OMB control number 0910-0303. The
collections of information in 21 CFR parts 50 and 56 have been approved
under OMB control number 0910-0130. The collections of information in
21 CFR 201.56 and 201.57 have been approved under OMB control number
0910-0572. The collections of information in 21 CFR parts 310 and 314
have been approved under OMB control number 0910-0230. The collections
of information in 21 CFR parts 310, 314, and 600 have been approved
under OMB control number 0910-0291. The collections of information in
21 CFR part 312 have been approved under OMB control number 0910-0014.
The collections of information in 21 CFR part 314 have been approved
under OMB control number 0910-0001. The collections of information in
21 CFR part 601 have been approved under OMB control number 0910-0338.
The collections of information in 21 CFR part 600 have been approved
under OMB control number 0910-0308. The collections of information
resulting from formal meetings between sponsors or applicants and FDA
have been approved under OMB control number 0910-0001.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
<a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>,
https://www.fda.gov/regulatory-information/
[[Page 88633]]
search-fda-guidance-documents, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Dated: December 19, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-28289 Filed 12-21-23; 8:45 am]
BILLING CODE 4164-01-P
</pre></body>
</html>Indexed from Federal Register on December 22, 2023.
This is legal information, not legal advice. Laws vary by jurisdiction and change frequently. Always verify current law with official sources and consult a licensed attorney in your jurisdiction for advice on your specific situation.