Notice2021-26640
Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products; Draft Guidance for Industry; Availability
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Published
December 9, 2021
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological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 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. FDA created a framework 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 to support or satisfy postapproval study requirements. 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 (e.g., as part of a new drug application or a biologics license application) that are not subject to the IND regulations.
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<title>Federal Register, Volume 86 Issue 234 (Thursday, December 9, 2021)</title>
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[Federal Register Volume 86, Number 234 (Thursday, December 9, 2021)]
[Notices]
[Pages 70131-70132]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2021-26640]
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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Products; Draft Guidance for Industry; Availability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 draft 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 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. FDA created a framework
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 to support or satisfy postapproval study requirements. 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
(e.g., as part of a new drug application or a biologics license
application) that are not subject to the IND regulations.
DATES: Submit either electronic or written comments on the draft
guidance by March 9, 2022 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final
version of the guidance.
ADDRESSES: You may submit comments on any guidance 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 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 the draft 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, 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
[[Page 70132]]
INFORMATION section for electronic access to the draft 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, <a href="/cdn-cgi/l/email-protection#4e0a272f20202b601e2f3c2f212f200e282a2f6026263d60292138"><span class="__cf_email__" data-cfemail="53173a323d3d367d033221323c323d133537327d3b3b207d343c25">[email protected]</span></a>; or Stephen Ripley, 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#f7a48392879f9299d9a59e879b928eb7919396d99f9f84d9909881"><span class="__cf_email__" data-cfemail="aaf9decfdac2cfc484f8c3dac6cfd3eacccecb84c2c2d984cdc5dc">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft 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 draft guidance discusses the following major topics:
(1) Applicability of part 312 (21 CFR part 312) to studies using RWD
and (2) regulatory considerations for non-interventional
(observational) studies using RWD. Topics covered under regulatory
considerations include the following: (1) Transparency for data
collection and analysis, (2) data access, (3) study monitoring, (4)
safety reporting, and (5) sponsor responsibilities.
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). This section requires the establishment of a program to evaluate
the potential use of RWE to help support the approval of a new
indication for a drug approved under section 505(c) of the FD&C Act (21
U.S.C. 355(c)) and to help support or satisfy postapproval study
requirements. This section also requires that FDA utilize the program
to inform guidance for industry on the circumstances under which
sponsors of drugs may rely on RWE and the appropriate standards and
methodologies for collection and analysis of RWE submitted to evaluate
the potential use of RWE for those purposes. Further, under the
Prescription Drug User Fee Amendments of 2017 (PDUFA VI), FDA is
committed to publishing draft guidance on how RWE can contribute to the
assessment of safety and effectiveness in regulatory submissions. FDA
is issuing the draft guidance entitled ``Considerations for the Use of
Real-World Data and Real-World Evidence to Support Regulatory Decision-
Making for Drug and Biological Products'' as part of a series of
guidance documents to satisfy the Cures Act mandate and the PDUFA VI
commitment.
This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent 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. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. The previously approved collections of
information are subject to review by OMB under the PRA. 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 part 54 have been approved under
OMB control number 0910-0396. The collections of information in 21 CFR
part 310 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 0910-0645. 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 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. The collections of information in FDA's
guidance for industry entitled ``Oversight of Clinical Investigations:
A Risk-Based Approach to Monitoring'' have been approved under OMB
control number 0910-0733. The collections of information in FDA's
guidance for industry entitled ``Formal Meetings with Sponsors and
Applicants for PDUFA Products'' have been approved under OMB control
number 0910-0429.
III. Electronic Access
Persons with access to the internet may obtain the draft 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: December 2, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021-26640 Filed 12-8-21; 8:45 am]
BILLING CODE 4164-01-P
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