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[Federal Register Volume 89, Number 235 (Friday, December 6, 2024)]
[Notices]
[Pages 97011-97013]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-28392]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-D-2033]

Expedited Program for Serious Conditions--Accelerated Approval of 
Drugs and Biologics; Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Expedited 
Program for Serious Conditions--Accelerated Approval of Drugs and 
Biologics.'' Accelerated approval is one of FDA's expedited programs 
intended to facilitate and expedite development and review of certain 
drugs and biological products for serious or life-threatening 
conditions. This guidance provides information on FDA's policies and 
procedures for the accelerated approval program, including discussions 
of which products may be candidates for accelerated approval, the 
standards for granting accelerated approval, and the procedures for 
withdrawing accelerated approval. When finalized, this draft guidance 
will replace the accelerated approval-related content in the final 
guidance for industry entitled ``Expedited Programs for Serious 
Conditions--Drugs and Biologics'' issued on May 30, 2014 (the 2014 
final guidance). Additional programs to expedite product development 
are covered in the 2014 final guidance as well as other guidances.

DATES: Submit either electronic or written comments on the draft 
guidance by February 4, 2025. 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comment on this draft guidance before it begins work on the final 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

[[Page 97012]]

well as any attachments, except for information submitted, marked and 
identified, as confidential, if submitted as detailed in 
``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2024-D-2033 for ``Expedited Program for Serious Conditions--
Accelerated Approval of Drugs and Biologics.'' 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 20993-0002, 240-402-8926 or 301-
796-2500, [email protected]; James Myers, 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; or Paul Kluetz, Oncology Center of Excellence, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 2223, Silver 
Spring, MD 20993, 301-796-9567, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Expedited Program for Serious Conditions--Accelerated 
Approval of Drugs and Biologics.'' Accelerated approval allows drugs 
for serious conditions that fill an unmet medical need to be approved 
based on a surrogate endpoint or an intermediate clinical endpoint that 
is reasonably likely to predict an effect on irreversible morbidity or 
mortality or other clinical benefit. Accelerated approval is one of 
FDA's expedited programs intended to facilitate and expedite 
development and review of new drugs to address an unmet medical need in 
the treatment of a serious or life-threatening condition.
    In 1992, FDA issued accelerated approval regulations. In 1997, 
Congress codified the accelerated approval program in the Food and Drug 
Administration Modernization Act of 1997 (Pub. L. 105-115), adding 
section 506 to the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 
U.S.C. 356). In 2012, Congress amended section 506 of the FD&C Act via 
the Food and Drug Administration Safety and Innovation Act (Pub. L. 
112-144) to provide that FDA should consider the ``severity, rarity, or 
prevalence of the condition and the availability or lack of alternative 
treatments.'' In 2014, FDA issued a final guidance for industry 
entitled ``Expedited Programs for Serious Conditions--Drugs and 
Biologics,'' which provides information on FDA's policies and 
procedures for four expedited programs (fast track designation, 
breakthrough therapy designation, accelerated approval, and priority 
review designation), as well as threshold criteria applicable to 
concluding that a drug is a candidate for these expedited programs.
    Section 506(c) of the FD&C Act was most recently amended by the 
Consolidated Appropriations Act, 2023 (Pub. L. 117-328), which granted 
FDA additional authorities and imposed on FDA additional obligations 
regarding the accelerated approval pathway. Among other revisions, 
section 3210 of the Consolidated Appropriations Act, 2023 requires FDA 
to set forth conditions, not later than the date of accelerated 
approval, for confirmatory trials, which ``may include enrollment 
targets, the study protocol, and milestones, including the target date 
of study completion.'' Congress also revised the provisions in section 
506(c) of the FD&C Act related to the expedited withdrawal of approval 
of a product approved under accelerated approval, including by adding 
new procedures for expedited withdrawal. Section V of this draft 
guidance describes these recently added procedures for the expedited 
withdrawal of approval of a product approved under accelerated 
approval.
    This draft guidance addresses the accelerated approval process, 
including granting accelerated approval (e.g., discussion of endpoints, 
evidentiary criteria, confirmatory trials and other conditions of 
accelerated approval), and withdrawal of accelerated approval. Changes 
from the 2014 final guidance include early consultation on novel 
endpoints, timely conduct of confirmatory trials, other aspects of 
confirmatory trials, and the expedited withdrawal of accelerated 
approval. Other changes include a revised title and editorial changes 
for clarity, as well as updated references and contact information for 
FDA. When finalized, this guidance will replace the accelerated 
approval-related content contained in the 2014 final guidance issued on 
May 30, 2014 (79 FR 31117).
    Additionally, via the Consolidated Appropriations Act, 2023, 
Congress gave FDA the authority to require, as appropriate, that a 
confirmatory trial be underway prior to accelerated approval or within 
a specified time period after the date of accelerated approval. The

[[Page 97013]]

Agency intends to address this authority in a separate guidance.
    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 ``Expedited 
Program for Serious Conditions--Accelerated Approval of Drugs and 
Biologics.'' 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

    Under the Paperwork Reduction Act of 1995 (the PRA) (44 U.S.C. 
3501-3521), Federal Agencies must obtain approval from the Office of 
Management and Budget (OMB) for each collection of information they 
conduct or sponsor. This draft guidance contains proposed collections 
of information including information submitted to FDA in support of 
maintaining an accelerated approval. ``Collection of information'' is 
defined in 44 U.S.C. 3502(3) and 5 CFR 1320.3(c) and includes Agency 
requests or requirements that members of the public submit reports, 
keep records, or provide information to a third party. Section 
3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A)) requires Federal 
Agencies to publish a 60-day notice in the Federal Register soliciting 
public comment on each proposed collection of information before 
submitting the collection to OMB for approval. To comply with this 
requirement, FDA will publish a 60-day notice on the proposed 
collections of information in this draft guidance in a separate issue 
of the Federal Register. This notice will encompass the collection of 
information discussed in the guidance relating to expedited procedures 
for the withdrawal of accelerated approval of drugs and biologics. 
Those procedures include the sponsor's submission of their objections 
to withdrawal as well as their supporting data and evidence. This 
collection of information is not included in any currently approved 
information collection.
    This draft guidance also refers to previously approved FDA 
collections of information. The collections of information in 21 CFR 
parts 10, 12-16, and 19 pertaining to administrative practice and 
procedures have been approved under OMB control number 0910-0191. The 
collections of information in 21 CFR part 312 relating to clinical 
trials associated with accelerated approval pathways have been approved 
under OMB control number 0910-0014. The collections of information in 
21 CFR part 314 relating to the submission of new drug applications, 
including accelerated approval of new drugs for serious or life-
threatening conditions, have been approved under OMB control number 
0910-0001. The collections of information in 21 CFR part 601 relating 
to the submission of biologics license applications have been approved 
under OMB control number 0910-0338. The collections of information 
pertaining to expedited programs for serious conditions for drugs and 
biologics and breakthrough therapy-designation for drugs and biologics 
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: November 27, 2024.
P. Ritu Nalubola,
Associate Commissioner for Policy.
[FR Doc. 2024-28392 Filed 12-5-24; 8:45 am]
BILLING CODE 4164-01-P
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