Notice2021-21194
Benefit-Risk Assessment for New Drug and Biological Products; Draft Guidance for Industry; Availability
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Published
September 30, 2021
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Benefit- Risk Assessment for New Drug and Biological Products."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).
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<title>Federal Register, Volume 86 Issue 187 (Thursday, September 30, 2021)</title>
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[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54211-54213]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Benefit-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:
<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.''
[[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 <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: 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, <a href="/cdn-cgi/l/email-protection#185f6a79707975364c707775686b7776587e7c793670706b367f776e"><span class="__cf_email__" data-cfemail="7c3b0e1d141d1152281413110c0f13123c1a181d5214140f521b130a">[email protected]</span></a>; 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: <a href="https://healthpolicy.duke.edu/events/public-meeting-characterizing-fdas-approach-benefit-risk-assessment-throughout-medical">https://healthpolicy.duke.edu/events/public-meeting-characterizing-fdas-approach-benefit-risk-assessment-throughout-medical</a>. 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 <a href="https://www.fda.gov/media/99140/download">https://www.fda.gov/media/99140/download</a>), 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 <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>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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