Benefit-Risk Assessment for New Drug and Biological Products; Draft Guidance for Industry; Availability, 54211-54213 [2021-21194]

Download as PDF Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices 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 Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th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 See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. Sue Zuk, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 75, Rm. 6684, Silver Spring, MD 20903–0002, 240–402–9133. FOR FURTHER INFORMATION CONTACT: SUPPLEMENTARY INFORMATION: LOTTER on DSK11XQN23PROD with NOTICES1 I. Background FDA is announcing the availability of a draft guidance for industry entitled ‘‘Microbiological Quality Considerations in Non-Sterile Drug Manufacturing.’’ The guidance provides recommendations to help manufacturers assess the risk of contamination of their NSDs with objectionable microorganisms or high bioburden levels in order to establish appropriate specifications and manufacturing controls that prevent such contamination and assure the safety, quality, identity, purity, and efficacy of the NSD. The guidance also imparts specific considerations to control microbial proliferation for selected nonsterile dosage forms that may present unique manufacturing challenges and patient safety risks. This draft guidance is being issued consistent with FDA’s good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on ‘‘Microbiological Quality Considerations in Non-Sterile Drug Manufacturing.’’ 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. VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 II. 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. The collections of information in 21 CFR part 211 have been approved under OMB control number 0910–0139. In the Federal Register of July 28, 2015 (80 FR 44973), FDA published a burden analysis for preparing and maintaining CGMP records for active pharmaceutical ingredients under section 501(a)(2)(B) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 351(a)(2)(B)). III. Electronic Access Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/drugs/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ regulatory-information/search-fdaguidance-documents, or https:// www.regulations.gov. Dated: September 23, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21222 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P 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. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘BenefitRisk 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 SUMMARY: PO 00000 Frm 00063 Fmt 4703 Sfmt 4703 54211 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.’’ E:\FR\FM\30SEN1.SGM 30SEN1 LOTTER on DSK11XQN23PROD with NOTICES1 54212 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices 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-201509-18/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, VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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 benefitrisk 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 benefitrisk 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 benefitrisk 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 PO 00000 Frm 00064 Fmt 4703 Sfmt 4703 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 (DukeMargolis) 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-meetingcharacterizing-fdas-approach-benefitrisk-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 E:\FR\FM\30SEN1.SGM 30SEN1 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices development of a draft guidance on benefit-risk assessments for new drugs and biologics, to further the Agency’s implementation of structured benefitrisk assessment, including the incorporation of the patient’s voice in drug development and decision making, in the human drug review program. LOTTER on DSK11XQN23PROD with NOTICES1 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 VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/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 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2021–N–0981] Fee Rate for Using a Tropical Disease Priority Review Voucher in Fiscal Year 2022 AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA or the Agency) is announcing the fee rates for using a tropical disease priority review voucher for fiscal year (FY) 2022. The Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by the Food and Drug Administration Amendments Act of 2007 (FDAAA), authorizes FDA to determine and collect priority review user fees for certain applications for review of drug and biological products when those applications use a tropical disease priority review voucher. These vouchers are awarded to the sponsors of certain tropical disease product applications submitted after September 27, 2007, the enactment date of FDAAA, upon FDA approval of such applications. The amount of the fee submitted to FDA with applications using a tropical disease priority review voucher is determined each fiscal year based on the difference between the average cost incurred by FDA to review a human drug application designated as priority review in the previous fiscal year and the average cost incurred in the review of an application that is not subject to priority review in the previous fiscal year. This notice establishes the tropical disease priority review fee rate for FY 2022 and outlines the payment procedures for such fees. SUMMARY: PO 00000 Frm 00065 Fmt 4703 Sfmt 4703 54213 FOR FURTHER INFORMATION CONTACT: Andrew Bank, Office of Financial Management, Food and Drug Administration, 4041 Powder Mill Rd., Rm. 62019A, Beltsville, MD, 20705– 4304, 301–796–0292. SUPPLEMENTARY INFORMATION: I. Background Section 1102 of FDAAA (Pub. L. 110– 85) added section 524 to the FD&C Act (21 U.S.C. 360n). In section 524, Congress encouraged development of new drug and biological products for prevention and treatment of tropical diseases by offering additional incentives for obtaining FDA approval of such products. Under section 524, the sponsor of an eligible human drug application submitted after September 27, 2007, for a tropical disease (as defined in section 524(a)(3) of the FD&C Act) shall receive a priority review voucher upon approval of the tropical disease product application (as defined in section 524(a)(4) of the FD&C Act), assuming other criteria are met. The recipient of a tropical disease priority review voucher may either use the voucher for a future human drug application submitted to FDA under section 505(b)(1) of the FD&C Act (21 U.S.C. 355(b)(1)) or section 351(a) of the Public Health Service Act (PHS Act) (42 U.S.C. 262), or transfer (including by sale) the voucher to another party. The voucher may be transferred repeatedly until it ultimately is used for a human drug application submitted to FDA under section 505(b)(1) of the FD&C Act or section 351(a) of the PHS Act. A priority review is a review conducted with a Prescription Drug User Fee Act (PDUFA) goal date of 6 months after the receipt or filing date, depending upon the type of application. Information regarding the PDUFA goals is available at: https://www.fda.gov/media/99140/ download. The sponsor that uses a priority review voucher is entitled to a priority review but must pay FDA a priority review user fee in addition to any other fee required by PDUFA. FDA published guidance on its website about how this tropical disease priority review voucher program operates (available at: https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents/ tropical-disease-priority-reviewvouchers). This notice establishes the tropical disease priority review fee rate for FY 2022 as $1,266,651 and outlines FDA’s process for implementing the collection of the priority review user fees. This rate is effective on October 1, 2021, and will remain in effect through September 30, E:\FR\FM\30SEN1.SGM 30SEN1

Agencies

[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.

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

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, [email protected]; 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

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