Notice2024-16338
Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products; Guidance for Industry; Availability
Primary source
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Published
July 25, 2024
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 Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products." 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 about the use of RWE in regulatory decision making. This guidance is intended to provide sponsors and other interested parties with considerations when proposing to use electronic health records (EHRs) or medical claims data in clinical studies to support a regulatory decision for effectiveness or safety. This guidance finalizes the draft guidance of the same title issued on September 30, 2021.
Full Text
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<title>Federal Register, Volume 89 Issue 143 (Thursday, July 25, 2024)</title>
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[Federal Register Volume 89, Number 143 (Thursday, July 25, 2024)]
[Notices]
[Pages 60430-60432]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-16338]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2020-D-2307]
Real-World Data: Assessing Electronic Health Records and Medical
Claims Data To Support Regulatory Decision-Making for Drug and
Biological Products; Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a final guidance for industry entitled ``Real-World
Data: Assessing Electronic Health Records and Medical Claims Data To
Support Regulatory Decision-Making for Drug and Biological Products.''
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 about the use of
RWE in regulatory decision making. This guidance is intended to provide
sponsors and other interested parties with considerations when
proposing to use electronic health records (EHRs) or medical claims
data in clinical studies to support a regulatory decision for
effectiveness or safety. This guidance finalizes the draft guidance of
the same title issued on September 30, 2021.
DATES: The announcement of the guidance is published in the Federal
Register on July 25, 2024.
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-2020-D-2307 for ``Real-World Data:
[[Page 60431]]
Assessing Electronic Health Records and Medical Claims Data 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 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 to 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 Electronic Health Records and
Medical Claims Data To Support Regulatory Decision-Making for Drug and
Biological Products.'' This guidance discusses the following topics
related to the potential use of EHRs and medical claims in clinical
studies to support regulatory decisions: selection of data sources that
appropriately address the study question and sufficiently capture study
populations, exposure, outcomes of interest, and key covariates;
development and validation of definitions for study design elements
(e.g., exposure, outcomes, covariates); and data traceability and
quality during data accrual, data curation, and incorporation into the
final study-specific dataset.
Section 3022 of the 21st Century Cures Act (Cures Act) of 2016
amended the FD&C Act to add section 505F, Utilizing Real World Evidence
(21 U.S.C. 355g), which requires FDA to issue guidance about the use of
RWE in regulatory decision making. In addition, under the Prescription
Drug User Fee Amendments of 2017 (PDUFA VI), FDA committed 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 RWE 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 program to issue
draft guidances outlining considerations for the use of real-world data
and RWE in regulatory decision making to, among other things, help
satisfy the Cures Act mandate and the PDUFA VI commitment.
This guidance finalizes the draft guidance of the same title issued
on September 30, 2021 (86 FR 54219). FDA considered comments received
on the draft guidance as the guidance was finalized. Changes from the
draft to the final guidance include: (1) clarifying that the selection
of study variables for validation and the extent of effort required for
validation depends on the necessary level of certainty and the
implication of potential misclassification on study inference; (2)
noting that choice of a reference standard for validation may vary by
the study design and question, variable of interest, and the necessary
level of certainty; (3) recommending the use of quantitative
approaches, such as quantitative bias analyses, either a priori for
feasibility assessment, or to facilitate interpretation of study
results, or for both purposes, to demonstrate whether and how
misclassification, if present, might impact study findings; and (4)
removing defined terms that are generally understood and transferring
other relevant definitions from a 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 Electronic
Health Records and Medical Claims Data 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 (PRA) (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 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; and the collections
[[Page 60432]]
of information in 21 CFR part 601 have been approved under OMB control
number 0910-0338.
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>,
<a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Dated: July 22, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-16338 Filed 7-24-24; 8:45 am]
BILLING CODE 4164-01-P
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</html>Indexed from Federal Register on July 25, 2024.
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