Document ID: FDA-2021-D-1152-0001
Agency: fda
Document Type: Notice
Title: Considerations for Rescinding Breakthrough Therapy Designation; Draft Guidance for Industry; Availability
Posted Date: 2022-06-24T04:00Z

[Federal Register Volume 87, Number 121 (Friday, June 24, 2022)]
[Notices]
[Pages 37865-37867]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-13528]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-1152]

Considerations for Rescinding Breakthrough Therapy Designation; 
Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled 
``Considerations for Rescinding Breakthrough Therapy Designation.'' 
This guidance explains how, during its evaluation of a drug development 
program, FDA may consider whether to rescind a breakthrough therapy 
designation (BTD) that has been granted. The guidance is consistent 
with, and supplements, the information on BTD contained in the guidance 
for industry entitled ``Expedited Programs for Serious Conditions--
Drugs and Biologics'' (May 2014) and in other BTD policies and 
procedures of the Center for Drug Evaluation and Research (CDER) and 
the Center for Biologics Evaluation and Research (CBER) to expedite the 
development and review of a breakthrough therapy.

DATES: Submit either electronic or written comments on the draft 
guidance by August 23, 2022 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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

[[Page 37866]]

identified, as confidential, if submitted as detailed in 
``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2021-D-1152 for ``Considerations for Rescinding Breakthrough 
Therapy Designation.'' 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 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: Dat Doan, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 51, Rm. 3334, Silver Spring, MD 20993, 240-402-8926, 
[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 ``Considerations for Rescinding Breakthrough Therapy 
Designation.'' This guidance is consistent with, and supplements, the 
information on BTD contained in the guidance for industry entitled 
``Expedited Programs for Serious Conditions--Drugs and Biologics'' (May 
2014),\1\ in CDER's Manual of Policies and Procedures 6025.6 ``Good 
Review Practice: Management of Breakthrough Therapy-Designated Drugs 
and Biologics,'' and in CBER's Standard Operating Policy and Procedure 
8212 Version 2 ``Management of Breakthrough Therapy-Designated 
Products: Sponsor Interactions and Status Assessment Including 
Rescinding.''
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    \1\ Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics.
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    Section 506(a) of the Federal Food, Drug, and Cosmetic Act (FD&C 
Act) (21 U.S.C. 356(a)) provides for the granting of BTD ``if the drug 
is intended, alone or in combination with 1 or more other drugs, to 
treat a serious or life-threatening disease or condition and 
preliminary clinical evidence indicates that the drug may demonstrate 
substantial improvement over existing therapies on 1 or more clinically 
significant endpoints, such as substantial treatment effects observed 
early in clinical development.'' The BTD program is intended to 
facilitate and expedite the development of those drugs that receive 
designation and involves a resource commitment from FDA to provide 
early and frequent advice, conduct multidisciplinary meetings involving 
senior managers, and expedite the review of resultant marketing 
applications. Thus, over the course of product development, it is 
important that available evidence relevant to an application continues 
to meet the standards for BTD.
    The information and circumstances supporting the original grant of 
BTD for a particular application may change over time. For example, if 
a different drug is approved to treat the unmet need that informed the 
rationale for granting BTD, the designated drug's preliminary clinical 
evidence may no longer meet the BTD criteria regarding substantial 
improvement over existing therapies (including the newly approved 
drug). In other cases, some drugs that appear promising in early 
development fail to meet their primary endpoints or the extent of 
benefit is more modest in later trials, and the magnitude of a 
treatment effect suggested by early development may not be replicable 
in later stages of development. Accordingly, in keeping with the 
Agency's authority to grant BTD only to drugs that meet the legal 
criteria, FDA periodically assesses whether the criteria for BTD 
continue to be met for designated products. If the designation is no 
longer supported by subsequent data, FDA may rescind the designation.
    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 
``Considerations for Rescinding Breakthrough Therapy Designation.'' 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. 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 312 have been approved under OMB control 
number 0910-0014; the collections of information in 21 CFR part 314 
have

[[Page 37867]]

been approved under OMB control number 0910-0001; the collections of 
information in 21 CFR part 601 have been approved under OMB control 
number 0910-0338; and the collections of information in section 506 of 
the FD&C Act have been approved under OMB control number 0910-0765.

III. Electronic Access

    Persons with access to the internet may obtain the draft 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.

    Dated: June 17, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-13528 Filed 6-23-22; 8:45 am]
BILLING CODE 4164-01-P