
[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]


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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 
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 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finalizes the draft guidance of the same title issued on November 30, 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


