[Federal Register Volume 89, Number 143 (Thursday, July 25, 2024)]
[Notices]
[Pages 60430-60432]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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 This guidance finalizes the draft guidance of 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:
     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-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 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 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 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 
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/search-fda-guidance-documents, or https://www.regulations.gov.

    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