Real-World Data: Assessing Registries To Support Regulatory Decision-Making for Drug and Biological Products; Guidance for Industry; Availability, 88631-88633 [2023-28289]
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Federal Register / Vol. 88, No. 245 / Friday, December 22, 2023 / Notices
collections of information in 21 CFR
part 812 have been approved under
OMB control number 0910–0078; the
collections of information for the De
Novo Classification Process (Evaluation
of Automatic Class III Designation) have
been approved under OMB control
number 0910–0844; and the collections
of information in the guidance
document entitled ‘‘Requests for
Feedback and Meetings for Medical
Device Submissions: The Q-Submission
Program’’ have been approved under
OMB control number 0910–0756. The
collections of information in 21 CFR
part 314 (Applications for FDA
Approval to Market a New Drug) and 21
CFR part 601 (General Licensing
Provisions: Biologics License
Application, Changes to an Approved
Application, Labeling, Revocation and
Suspension) have been approved under
OMB control numbers 0910–0001 and
0910–0338, respectively. The
collections of information in 21 CFR
parts 50 and 56 (Protection of Human
Subjects: Informed Consent;
Institutional Review Boards) have been
approved under OMB control number
0910–0130.
III. Electronic Access
Persons with access to the internet
may obtain the guidance at https://
www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances,https://
www.fda.gov/medical-devices/deviceadvice-comprehensive-regulatoryassistance/guidance-documentsmedical-devices-and-radiation-emittingproducts, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
Dated: December 18, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023–28262 Filed 12–21–23; 8:45 am]
ddrumheller on DSK120RN23PROD with NOTICES1
BILLING CODE 4164–01–P
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DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2021–D–1146]
Real-World Data: Assessing Registries
To Support Regulatory DecisionMaking for Drug and Biological
Products; Guidance for Industry;
Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘RealWorld 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 RealWorld 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:
SUMMARY:
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.
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• 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–
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 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
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88632
Federal Register / Vol. 88, No. 245 / Friday, December 22, 2023 / Notices
ddrumheller on DSK120RN23PROD with NOTICES1
the information at: https://
www.govinfo.gov/content/pkg/FR-201509-18/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.
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 ‘‘RealWorld 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:
• 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;
• Considerations when linking a
registry to another data source for
VerDate Sep<11>2014
18:10 Dec 21, 2023
Jkt 262001
supplemental information, such as data
from medical claims, electronic health
records, digital health technologies, or
other registries; and
• 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 decisionmaking 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.
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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 https://
www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, https://
www.fda.gov/regulatory-information/
E:\FR\FM\22DEN1.SGM
22DEN1
Federal Register / Vol. 88, No. 245 / Friday, December 22, 2023 / Notices
search-fda-guidance-documents, or
https://www.regulations.gov.
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2021–D–0548]
Data Standards for Drug and Biological
Product Submissions Containing RealWorld Data; Guidance for Industry;
Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘Data
Standards for Drug and Biological
Product Submissions Containing RealWorld Data.’’ This guidance provides
recommendations to sponsors to help
support compliance with the Federal
Food, Drug, and Cosmetic Act (FD&C
Act) when submitting study data
derived from real-world data (RWD)
sources in applicable regulatory
submissions using standards specified
in the Data Standards Catalog. FDA is
publishing this guidance as part of a
series of guidance documents under its
program to evaluate the use of realworld evidence (RWE) in regulatory
decision making. This guidance
finalizes the draft guidance of the same
title issued on October 22, 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:
SUMMARY:
ddrumheller on DSK120RN23PROD with NOTICES1
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
VerDate Sep<11>2014
18:10 Dec 21, 2023
Jkt 262001
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–
2021–D–0548 for ‘‘Data Standards for
Drug and Biological Product
Submissions Containing Real-World
Data.’’ Received comments 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
PO 00000
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88633
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-201509-18/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.
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. 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. 3326,
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 ‘‘Data
Standards for Drug and Biological
Product Submissions Containing RealWorld Data.’’ Section 3022 of the 21st
Century Cures Act (Cures Act) amended
the FD&C Act to add section 505F,
Utilizing Real World Evidence (21
U.S.C. 355g). In addition, under the
Prescription Drug User Fee
Amendments of 2017 (PDUFA VI), FDA
committed to publishing draft guidance
on how RWE can contribute to the
assessment of safety and effectiveness in
E:\FR\FM\22DEN1.SGM
22DEN1
]]>Agencies
[Federal Register Volume 88, Number 245 (Friday, December 22, 2023)]
[Notices]
[Pages 88631-88633]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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:
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-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 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
[[Page 88632]]
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.
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:
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;
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
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
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances,
https://www.fda.gov/regulatory-information/
[[Page 88633]]
search-fda-guidance-documents, or https://www.regulations.gov.
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
