Notice2023-23161
Benefit-Risk Assessment for New Drug and Biological Products; Guidance for Industry; Availability
Primary source
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Published
October 20, 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 "Benefit- Risk Assessment for Human Drug and Biological Products." FDA has developed this guidance document in accordance with goals associated with the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI) under Title I of the FDA Reauthorization Act of 2017 and requirements under the 21st Century Cures Act. The intent of this guidance is to provide drug sponsors and other stakeholders with better clarity on how considerations about a drug's benefits, risks, and risk management options factor into FDA's pre- and postmarket regulatory decisions about new drug applications (NDAs) or biologics license applications (BLAs). This guidance finalizes the draft guidance of the same title issued in September 2021.
Full Text
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<title>Federal Register, Volume 88 Issue 202 (Friday, October 20, 2023)</title>
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[Federal Register Volume 88, Number 202 (Friday, October 20, 2023)]
[Notices]
[Pages 72491-72493]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-23161]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2020-D-2316]
Benefit-Risk Assessment for New 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 ``Benefit-
Risk Assessment for Human Drug and Biological Products.'' FDA has
developed this guidance document in accordance with goals associated
with the sixth authorization of the Prescription Drug User Fee Act
(PDUFA VI) under Title I of the FDA Reauthorization Act of 2017 and
requirements under the 21st Century Cures Act. The intent of this
guidance is to provide drug sponsors and other stakeholders with better
clarity on how considerations about a drug's benefits, risks, and risk
management options factor into FDA's pre- and postmarket regulatory
decisions about new drug applications (NDAs) or biologics license
applications (BLAs). This guidance finalizes the draft guidance of the
same title issued in September 2021.
DATES: The announcement of the guidance is published in the Federal
Register on October 20, 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-2020-D-2316 for ``Benefit-Risk Assessment for New 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
[[Page 72492]]
<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 this guidance to the
Division of Drug Information, Center for Drug Evaluation and Research
(CDER), 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 (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: Graham Thompson, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-5003,
<a href="/cdn-cgi/l/email-protection#7e390c1f161f13502a1611130e0d11103e181a1f5016160d50191108"><span class="__cf_email__" data-cfemail="2067524148414d0e74484f4d50534f4e604644410e4848530e474f56">[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.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a final guidance for industry
entitled ``Benefit-Risk Assessment for Human Drug and Biological
Products.'' This guidance articulates the key considerations that
factor into CDER's and CBER's benefit-risk assessments, including how
patient experience data may be used to inform benefit-risk assessment.
It discusses how sponsors, through their decisions and activities
throughout the drug lifecycle, can inform FDA's benefit-risk
assessment, as well as opportunities for interaction between FDA and
sponsors to discuss benefit-risk considerations. It also discusses
unique considerations for benefit-risk assessments that inform
regulatory decision-making that occurs in the postmarket setting.
Industry stakeholders have indicated having a clearer understanding
of FDA's decision-making context and benefit-risk considerations can
help inform sponsors' decisions about their drug development programs
and the evidence they generate in support of their NDA or BLA. Patients
and other stakeholders may gain further insight into the key issues
that inform FDA's assessment of benefit and risk, and a clearer
understanding of how these issues fit into the regulatory framework of
drug development and evaluation.
This guidance finalizes the draft guidance entitled ``Benefit-Risk
Assessment for New Drug and Biological Products'' issued on September
30, 2021 (86 FR 54211). FDA considered comments received on the draft
guidance in developing the final guidance. Changes from the draft to
the final guidance include clarifying language on FDA's approach to
benefit-risk assessment of new drugs and biologics, clarifying language
on important considerations for FDA's premarket benefit-risk assessment
of drugs and biologics, clarifying language on activities that occur in
premarket development that inform benefit-risk assessment, and other
editorial changes.
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 ``Benefit-Risk Assessment for New 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 314 have been approved under OMB control number 0910-
0001 as follows: (1) the content and format of investigational new
drugs applications, (2) expanded access uses and treatment of patients
with immediately life-threatening conditions or serious diseases or
conditions, (3) regulatory requirements pertaining to postmarketing
study commitments, and (4) risk evaluation and mitigation strategies
pertaining to benefit-risk assessments. The collections of information
in 21 CFR part 312 have been approved under OMB control number 0910-
0014 as follows: (1) the content and format of NDAs, (2) the submission
of the patient population, (3) the submission of clinical trial data,
and (4) benefit-risk planning, including early consultations with FDA
meetings in end-of-phase 2 and pre-NDA meetings. The collections of
information for good laboratory practices for nonclinical laboratory
studies have been approved under OMB control number 0910-0119. The
collections of information for the submission of postmarketing adverse
drug experience reporting have been approved under OMB control number
0910-0230. The collections of information in 21 CFR 201.56 and 201.57
for the content and format requirements for labeling of drugs and
biologics have been approved under OMB control number 0910-0572. The
collections of information in the guidance for industry entitled
``Expedited Programs for Serious Conditions--Drugs and Biologics'' have
been approved under OMB control number 0910-0765. The collections of
information in the guidance for industry entitled ``Providing
Postmarket Periodic Safety Reports in the International Conference on
Harmonisation E2C(R2) Format (Periodic Benefit-Risk Evaluation
Report)'' have been approved under OMB control number 0910-0230.
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>.
[[Page 72493]]
Dated: October 17, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-23161 Filed 10-19-23; 8:45 am]
BILLING CODE 4164-01-P
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