Notice2022-22618
Acute Myeloid Leukemia: Developing Drugs and Biological Products for Treatment; Guidance for Industry; Availability
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
October 18, 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 final 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 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 This guidance finalizes the draft guidance of the same title "Acute Myeloid Leukemia: Developing Drugs and Biological Products for Treatment" issued August 2020.
Full Text
<html>
<head>
<title>Federal Register, Volume 87 Issue 200 (Tuesday, October 18, 2022)</title>
</head>
<body><pre>
[Federal Register Volume 87, Number 200 (Tuesday, October 18, 2022)]
[Notices]
[Pages 63077-63079]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-22618]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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:
<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 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 <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 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
<a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</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>, or
<a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
</pre></body>
</html>Indexed from Federal Register on October 18, 2022.
This is legal information, not legal advice. Laws vary by jurisdiction and change frequently. Always verify current law with official sources and consult a licensed attorney in your jurisdiction for advice on your specific situation.