[Federal Register Volume 87, Number 200 (Tuesday, October 18, 2022)]
[Notices]
[Pages 63077-63079]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-22618]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-1298]


Acute Myeloid Leukemia: Developing Drugs and Biological Products 
for Treatment; Guidance for Industry; Availability

AGENCY: Food and Drug Administration, Department of Health and Human 
Services (HHS).

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a final guidance for industry entitled ``Acute 
Myeloid Leukemia: Developing Drugs

[[Page 63078]]

and Biological Products for Treatment.'' This guidance is intended to 
assist sponsors in the clinical development of drugs and biological 
products for the treatment of acute myeloid leukemia (AML). This 
guidance addresses FDA's current thinking regarding the overall 
development program and clinical trial designs for the development of 
drugs and biological products to support an indication of treatment of 
AML, including indications limited to an individual phase of treatment 
(for example, maintenance, transplantation preparative regimen, etc.). 
The guidance addresses the topics of general drug development, efficacy 
endpoints, and exploratory and confirmatory trial considerations for 
AML drug development. In addition, the guidance addresses 
investigational new drug applications, new drug applications, and 
biologics licensing applications for AML drugs. This guidance finalizes 
the draft guidance of the same title ``Acute Myeloid Leukemia: 
Developing Drugs and Biological Products for Treatment'' issued August 
2020.

DATES: The announcement of the guidance is published in the Federal 
Register on October 18, 2022.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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.''
    Instructions: All submissions received must include the Docket No. 
FDA-2020-D-1298 for ``Acute Myeloid Leukemia: Developing Drugs and 
Biological Products for Treatment.'' 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 this 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 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 guidance document.

FOR FURTHER INFORMATION CONTACT: Donna Przepiorka, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2116, Silver Spring, MD 20993-0002, 301-
796-5358; 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 guidance for industry 
entitled ``Acute Myeloid Leukemia: Developing Drugs and Biological 
Products for Treatment.'' This guidance is intended to assist sponsors 
in the clinical development of drugs and biological products for the 
treatment of AML. This guidance includes FDA's current thinking 
regarding the overall development program and clinical trial designs to 
support an indication of treatment of AML, including indications 
limited to an individual phase of treatment.
    New classes of drugs are being developed as alternatives to the 
standard cytotoxic drugs for the treatment of AML. The following 
factors contribute substantially to the complexity of clinical 
development programs for such new drugs: the expansion of treatment 
intent, broadening of the intended population, and development of a 
wide range of new drug classes as alternatives to cytotoxic

[[Page 63079]]

drugs. This guidance includes FDA's thinking regarding general drug 
development considerations, efficacy endpoints, exploratory and 
confirmatory trial considerations, and regulatory submissions for AML 
drugs to facilitate the development of new drugs for the treatment of 
AML.
    This guidance finalizes the draft guidance entitled ``Acute Myeloid 
Leukemia: Developing Drugs and Biological Products for Treatment'' 
issued August 13, 2020 (85 FR 49383). FDA considered comments received 
on the draft guidance as the guidance was finalized. Changes from the 
draft to the final guidance include editorial changes, clarifications 
of the time frame for marrow sampling and peripheral blood tests to 
establish complete remission, the inclusion of marker-negative patients 
in studies of targeted therapies, and recommended operating 
characteristics for safety-stopping rules.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Acute Myeloid Leukemia: Developing Drugs 
and Biological Products for Treatment.'' 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 312 have been approved under OMB control 
number 0910-0014; 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 601 have been approved under 0910-0338; 
and the collections of information in 21 CFR 201.56 and 201.57 have 
been approved under OMB control number 0910-0572.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
https://www.fda.gov/regulatory-information/search-fda-guidance-documents, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or 
https://www.regulations.gov.

    Dated: October 11, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-22618 Filed 10-17-22; 8:45 am]
BILLING CODE 4164-01-P


