Notice2024-05969
Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products; Draft Guidance for Industry; Availability
Primary source
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Published
March 21, 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products." FDA is issuing this draft guidance as part of a series of guidance documents under its Real-World Evidence (RWE) Program and to satisfy, in part, a mandate under the Federal Food, Drug, and Cosmetic Act (FD&C Act) to issue guidance about the use of RWE in regulatory decision-making. The draft guidance provides recommendations to sponsors who are considering submitting a non- interventional study, also referred to as an observational study, to FDA to contribute to a demonstration of substantial evidence of effectiveness and/or evidence of safety of a drug. This draft guidance was developed in response to stakeholders' growing interest in the potential use of non-interventional studies to contribute to a demonstration of the effectiveness or safety of a drug.
Full Text
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<title>Federal Register, Volume 89 Issue 56 (Thursday, March 21, 2024)</title>
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[Federal Register Volume 89, Number 56 (Thursday, March 21, 2024)]
[Notices]
[Pages 20207-20209]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-05969]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-D-5470]
Real-World Evidence: Considerations Regarding Non-Interventional
Studies for Drug and Biological Products; Draft Guidance for Industry;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the availability of a draft guidance for industry entitled ``Real-World
Evidence: Considerations Regarding Non-Interventional Studies for Drug
and Biological Products.'' FDA is issuing this draft guidance as part
of a series of guidance documents under its Real-World Evidence (RWE)
Program and to satisfy, in part, a mandate under the Federal Food,
Drug, and Cosmetic Act (FD&C Act) to issue guidance about the use of
RWE in regulatory decision-making. The draft guidance provides
recommendations to sponsors who are considering submitting a non-
interventional study, also referred to as an observational study, to
FDA to contribute to a demonstration of substantial evidence of
effectiveness and/or evidence of safety of a drug. This draft guidance
was developed in response to stakeholders' growing interest in the
potential use of non-interventional studies to contribute to a
demonstration of the effectiveness or safety of a drug.
DATES: Submit either electronic or written comments on the draft
guidance by June 18, 2024 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
[[Page 20208]]
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-2023-D-5470 for ``Real-World Evidence: Considerations Regarding
Non-Interventional Studies 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 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 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#2a4e434b44444f045a4b584b454b446a4c4e4b04424259044d455c"><span class="__cf_email__" data-cfemail="7a1e131b14141f540a1b081b151b143a1c1e1b54121209541d150c">[email protected]</span></a>; 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 draft guidance for industry
entitled ``Real-World Evidence: Considerations Regarding Non-
Interventional Studies for Drug and Biological Products.'' This draft
guidance discusses attributes regarding the design and analysis of a
non-interventional study that sponsors should consider when proposing
such a study to contribute to a demonstration of substantial evidence
of effectiveness and/or evidence of safety of a product. Examples of
non-interventional study designs for evaluating the effectiveness and/
or safety of a drug include, but are not limited to, cohort studies,
case-control studies, and self-controlled studies.
Identifying and addressing the presence of confounding and other
forms of bias is critical when planning and conducting non-
interventional studies. Before choosing a non-interventional study
design for a study intended to support regulatory decisions regarding
the safety and effectiveness of a drug, sponsors should consider how
likely it is that such a study design and its conduct will be able to
distinguish a true treatment effect from other influences. The draft
guidance assists sponsors in identifying and addressing commonly
encountered challenges when considering the use of a non-interventional
study to contribute to a demonstration of substantial evidence of
effectiveness and/or evidence of safety of a drug, including topics
sponsors should consider before developing a prespecified protocol and
statistical analysis plan. This draft guidance discusses the following
major topics: (1) summary of the proposed approach, (2) study design,
(3) data sources, and (4) analytical approach. The topics in this draft
guidance should be considered in conjunction with the recommendations
in other published guidances under the RWE Program.
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 ``Real-World
Evidence: Considerations Regarding Non-Interventional Studies 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 312 (investigational new drug applications) have been
approved under OMB control number 0910-0014; the collections of
information in 21 CFR part 314 (new drug marketing applications) have
been approved under OMB control number 0910-0001; and the collections
of information in 21 CFR part 601 (biologic license applications) have
been approved under OMB control number 0910-0338. The collections of
information in 21 CFR part 11 (electronic records and signatures) have
been approved under OMB control number 0910-0303. The collections of
information in 21 CFR parts 50 and 56 (protection of human subjects and
institutional review boards) have been approved under OMB control
number 0910-0130.
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>, https://www.fda.gov/
[[Page 20209]]
vaccines-blood-biologics/guidance-compliance-regulatory-information-
biologics/biologics-guidances, <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: March 15, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-05969 Filed 3-20-24; 8:45 am]
BILLING CODE 4164-01-P
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