[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54211-54213]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-21194]


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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; 
Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Benefit-
Risk Assessment for New Drug and Biological Products.'' FDA has 
developed this guidance document in accordance with commitments 
associated with the Prescription Drug User Fee Act of 2017 (PDUFA VI) 
under the FDA Reauthorization Act of 2017 and requirements under the 
21st Century Cures Act (Cures Act). The intent of this guidance is to 
provide drug sponsors and other stakeholders with a clearer 
understanding of how considerations about a drug's benefits, risks, and 
risk management options factor into certain FDA pre- and postmarket 
regulatory decisions about new drug applications (NDAs) submitted under 
the Federal Food, Drug, and Cosmetic Act (FD&C Act) and biologics 
license applications (BLAs).

DATES: Submit either electronic or written comments on the draft 
guidance by November 29, 2021 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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 information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''

[[Page 54212]]

    Instructions: All submissions received must include the Docket No. 
FDA-2020-D-2316 for ``Benefit-Risk Assessment for New Drug and 
Biological Products; Draft Guidance for Industry.'' 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, 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: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993-0002, 301-
796-5003, Graham.Thompson@fda.hhs.gov; 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.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Benefit-Risk Assessment for New Drug and Biological 
Products.'' This guidance articulates important considerations that 
factor into the Center for Drug Evaluation and Research and the Center 
for Biologics Evaluation and Research's benefit-risk assessments for 
drug products, including how patient experience data may be used to 
inform benefit-risk assessment. It discusses how sponsors can inform 
FDA's benefit-risk assessment through the design and conduct of the 
development program, as well as how they may present benefit and risk 
information in the marketing application. It also discusses 
opportunities for interaction between FDA and sponsors to discuss 
benefit-risk considerations in connection with the development of a BLA 
or NDA. The guidance concludes with additional considerations on 
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.
    In May 2019, FDA participated in a public meeting conducted by Duke 
University's Robert J. Margolis, MD, Center for Health Policy (Duke-
Margolis) on ``Characterizing FDA's Approach to Benefit-Risk Assessment 
Throughout the Medical Product Life Cycle'' (84 FR 17176, April 24, 
2019). Materials from this meeting are available here: https://healthpolicy.duke.edu/events/public-meeting-characterizing-fdas-approach-benefit-risk-assessment-throughout-medical. This meeting was 
intended to gather industry, patient, researcher, and other stakeholder 
input on applying FDA's benefit-risk framework throughout the human 
drug lifecycle and best approaches to communicating FDA's benefit-risk 
assessment. This meeting was intended to meet an FDA commitment 
included in the sixth authorization of the PDUFA VI. Input from this 
meeting supported development of this draft guidance for industry.
    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 ``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. Additional Information

    Section 3002 of Title III, Subtitle A of the Cures Act (Pub. L. 
114-255), directs FDA to develop patient-focused drug development 
guidance to address a number of areas, including under section 
3002(c)(8) of the Cures Act how the Secretary, if appropriate, 
anticipates using relevant patient experience data and related 
information, including with respect to the structured risk-benefit 
assessment framework described in section 505(d) of the FD&C Act (21 
U.S.C. 355(d)), to inform regulatory decision making.
    In addition, FDA committed to meet certain performance goals under 
the sixth authorization of PDUFA. These goal commitments were developed 
in consultation with patient and consumer advocates, healthcare 
professionals, and other public stakeholders, as part of negotiations 
with regulated industry. Section I.J.2. of the commitment letter, 
``Enhancing Benefit-Risk Assessment in Regulatory Decision-Making'' 
(available at https://www.fda.gov/media/99140/download), outlines work, 
including the

[[Page 54213]]

development of a draft guidance on benefit-risk assessments for new 
drugs and biologics, to further the Agency's implementation of 
structured benefit-risk assessment, including the incorporation of the 
patient's voice in drug development and decision making, in the human 
drug review program.

III. 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 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-
0771.

IV. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either 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, or https://www.regulations.gov.

    Dated: September 24, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21194 Filed 9-29-21; 8:45 am]
BILLING CODE 4164-01-P


