Notice2023-18841
Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products; Guidance for Industry; Availability
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Published
August 31, 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 "Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products." FDA is issuing this guidance as part of its Real-World Evidence (RWE) Program for drugs 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 discusses the applicability of FDA's investigational new drug application (IND) regulations to various clinical study designs that utilize real-world data (RWD) and clarifies the Agency's expectations regarding clinical studies using RWD submitted to FDA in support of a regulatory decision regarding the effectiveness or safety of a drug that are not subject to the IND regulations. This guidance finalizes the draft guidance of the same title issued on December 9, 2021.
Full Text
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<title>Federal Register, Volume 88 Issue 168 (Thursday, August 31, 2023)</title>
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[Federal Register Volume 88, Number 168 (Thursday, August 31, 2023)]
[Notices]
[Pages 60215-60217]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-18841]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-1214]
Considerations for the Use of Real-World Data and Real-World
Evidence 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
``Considerations for the Use of Real-World Data and Real-World Evidence
to Support Regulatory Decision-Making for Drug and Biological
Products.'' FDA is issuing this guidance as part of its Real-World
Evidence (RWE) Program for drugs 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 discusses the applicability of FDA's investigational new drug
application (IND) regulations to various clinical study designs that
utilize real-world data (RWD) and clarifies the Agency's expectations
regarding clinical studies using RWD submitted to FDA in support of a
regulatory decision regarding the effectiveness or safety of a drug
that are not subject to the IND regulations. This guidance finalizes
the draft guidance of the same title issued on December 9, 2021.
DATES: The announcement of the guidance is published in the Federal
Register on August 31, 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-1214 for ``Considerations for the Use of Real-World Data and
Real-World Evidence 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
[[Page 60216]]
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, 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, <a href="/cdn-cgi/l/email-protection#95d1fcf4fbfbf0bbc5f4e7f4faf4fbd5f3f1f4bbfdfde6bbf2fae3"><span class="__cf_email__" data-cfemail="15517c747b7b703b457467747a747b557371743b7d7d663b727a63">[email protected]</span></a>; or Anne Taylor, 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, <a href="/cdn-cgi/l/email-protection#46072828236812273f2a293406202227682e2e3568212930"><span class="__cf_email__" data-cfemail="23624d4d460d77425a4f4c51634547420d4b4b500d444c55">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry
entitled ``Considerations for the Use of Real-World Data and Real-World
Evidence to Support Regulatory Decision-Making for Drug and Biological
Products.'' The guidance discusses the following major topics: (1)
applicability of 21 CFR part 312 to studies using RWD and (2)
regulatory considerations for non-interventional (observational)
studies involving the use of RWD. Regulatory considerations addressed
by the guidance include the following: (1) transparency for data
collection and analysis, (2) access to RWD, (3) study monitoring, (4)
safety reporting, (5) other sponsor responsibilities, and (6) the
analysis of RWD generated from the use of a product under an emergency
use authorization (EUA) in routine practice.
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 is 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 an 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 RWE
Program to issue draft guidances outlining considerations for the use
of RWD 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 December 9, 2021 (86 FR 70131). FDA considered comments received on
the draft guidance as the guidance was finalized. Changes from the
draft to the final guidance include transfer of relevant definitions
from the glossary to the text as well as additional language about data
generated in clinical practice for products used under an EUA
consistent with a mandate under the Food and Drug Omnibus Reform Act of
2022. Clarifying information was also added, including the use of
existing regulatory pathways for third parties to provide patient-level
data to FDA when a sponsor cannot submit such data to FDA through
traditional channels. 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 ``Considerations for the Use of Real-World
Data and Real-World Evidence 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
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 pertaining to electronic records and signatures have
been approved under OMB control number 0910-0303. The collections of
information in 21 CFR parts 50 and 56 relating to protection of human
subjects and Institutional Review Boards have been approved under OMB
control number 0910-0130. The collections of information in 21 CFR part
310 relating to postmarketing adverse drug experience reporting have
been approved under OMB control number 0910-0230. The collections of
information in 21 CFR part 600 for general records and postmarketing
adverse experience reporting pertaining to biological products have
been approved under OMB control number 0910-0308. The collections of
information in 21 CFR parts 310, 314, and 600 pertaining to adverse
event and product experience reporting 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 0910-0458.
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>.
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Dated: August 28, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-18841 Filed 8-30-23; 8:45 am]
BILLING CODE 4164-01-P
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