Notice2022-13528
Considerations for Rescinding Breakthrough Therapy Designation; Draft Guidance for Industry; Availability
Primary source
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Published
June 24, 2022
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Considerations for Rescinding Breakthrough Therapy Designation."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.
Full Text
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<title>Federal Register, Volume 87 Issue 121 (Friday, June 24, 2022)</title>
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[Federal Register Volume 87, Number 121 (Friday, June 24, 2022)]
[Notices]
[Pages 37865-37867]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled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:
<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
[[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 <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 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,
<a href="/cdn-cgi/l/email-protection#bbffdacf95ffd4dad5fbdddfda95d3d3c895dcd4cd"><span class="__cf_email__" data-cfemail="4f0b2e3b610b202e210f292b2e6127273c61282039">[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 ``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: <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics">https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics</a>.
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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 <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>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
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