[Federal Register Volume 91, Number 7 (Monday, January 12, 2026)]
[Notices]
[Pages 1190-1192]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2026-00325]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2025-D-3217]
Use of Bayesian Methodology in Clinical Trials of 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Biological Products; Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Use of
Bayesian Methodology in Clinical Trials of Drug and Biological
Products.'' This document provides guidance on the appropriate use of
Bayesian methods in clinical trials. The primary focus is on the use of
Bayesian methods to support primary inference in pivotal clinical
trials designed to support the effectiveness and safety of drug and
biological products.
DATES: Submit either electronic or written comments on the draft
guidance by March 13, 2026 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final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:
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
[[Page 1191]]
information submitted, marked and identified, as confidential, if
submitted as detailed in ``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-2025-D-3217 for ``Use of Bayesian Methodology in Clinical Trials of
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 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. 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: Scott N. Goldie, Center for Drug
Evaluation and Research, 10903 New Hampshire Ave., Bldg. 21, Rm. 3557,
Silver Spring, MD 20993-0002, 301-796-2055, [email protected],
or Phillip Kurs, Center for Biologics Evaluation and Research, Food and
Drug Administration, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Use of Bayesian Methodology in Clinical Trials of Drug and
Biological Products.'' This document provides guidance to sponsors and
applicants submitting investigational new drug applications (INDs), new
drug applications (NDAs), biologics licensing applications (BLAs) or
supplemental applications on the appropriate use of Bayesian methods in
clinical trials. The primary focus of this guidance is on the use of
Bayesian methods to support primary inference in pivotal clinical
trials designed to support the effectiveness and safety of drug and
biological products. This draft guidance is being issued to satisfy a
commitment under the sixth reauthorization of the Prescription Drug
User Fee Act to publish draft guidance on the use of Bayesian
methodology in clinical trials of drugs and biological products.
Bayesian statistics is an approach to estimation and inference
based on the use of Bayes' theorem. In a Bayesian analysis, data
collected in a study are combined with a prior distribution that
expresses a pre-study position about a parameter of interest to form a
posterior distribution that expresses the updated, post-study position
about the parameter of interest. The prior distribution often
represents a summary of data or information available before the study
begins. The posterior distribution is used for inference and to draw
conclusions about the study results.
The draft guidance addresses relevant considerations on how to
appropriately design clinical trials using Bayesian approaches. The
document describes settings where Bayesian methods have been used and
provides recommendations related to the choice of success criteria,
evaluation of trial operating characteristics, and determination of the
prior distribution. It includes detailed considerations for use of an
informative prior to borrow external information in the primary
analysis of a prospective trial. The document also discusses estimands
and missing data, software and computation, and documentation and
reporting considerations for trials that use Bayesian methods.
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 ``Use of
Bayesian Methodology in Clinical Trials of 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.
As we develop final guidance on this topic, FDA will consider
comments on costs or cost savings the guidance may generate, relevant
for Executive Order 14192.
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 312 pertaining to the submissions of investigational new drug
applications have been approved under OMB control number 0910-0014. The
collections of information in 21 CFR 314 and 21 CFR 601 pertaining to
the submission of new drug applications and biologic licensing
applications have been approved under OMB control numbers 0910-0001 and
0910-0338, respectively.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or https://www.regulations.gov.
[[Page 1192]]
Lowell M. Zeta,
Acting Deputy Commissioner for Policy, Legislation, and International
Affairs.
[FR Doc. 2026-00325 Filed 1-9-26; 8:45 am]
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