[Federal Register Volume 88, Number 202 (Friday, October 20, 2023)]
[Notices]
[Pages 72491-72493]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-23161]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 This guidance finalizes the draft guidance of the 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: 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.
    <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 https://www.regulations.gov 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]]

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 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 
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, 
https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.


[[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


</pre><script data-cfasync="false" src="/cdn-cgi/scripts/5c5dd728/cloudflare-static/email-decode.min.js"></script></body>
</html>