[Federal Register Volume 85, Number 17 (Monday, January 27, 2020)]
[Notices]
[Pages 4668-4670]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01312]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-3090]


Hematologic Malignancies: Regulatory Considerations for Use of 
Minimal Residual Disease in Development of Drug and Biological Products 
for Treatment; 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 final guidance for industry entitled 
``Hematologic Malignancies: Regulatory Considerations for Use of 
Minimal Residual Disease in Development of Drug and Biological Products 
for Treatment.'' This guidance is intended to help sponsors planning to 
use minimal residual disease (MRD) as a biomarker in clinical trials 
conducted under an investigational new drug application (IND) or to 
support marketing approval of drugs and biological products for 
treating specific hematologic malignancies. An analysis of marketing 
applications showed inconsistent quality of MRD data. Based on this 
analysis and discussion at various public workshops on MRD, FDA 
identified a need to provide guidance on the use of MRD as a biomarker 
in regulatory submissions. This guidance finalizes the draft guidance 
of the same title issued on October 16, 2018.

[[Page 4669]]


DATES: The announcement of the guidance is published in the Federal 
Register on January 27, 2020.

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-2018-D-3090 for ``Hematologic Malignancies: Regulatory 
Considerations for Use of Minimal Residual Disease in Development of 
Drug 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.
     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.
    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 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: Nicole Gormley, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2310, Silver Spring, MD 20993-0002, 240-
402-0210; 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 final guidance for industry 
entitled ``Hematologic Malignancies: Regulatory Considerations for Use 
of Minimal Residual Disease in Development of Drug and Biological 
Products for Treatment.'' This guidance is intended to help sponsors 
planning to use MRD as a biomarker in clinical trials conducted under 
an IND or to support marketing approval of drugs and biological 
products for treating specific hematologic malignancies.
    This guidance finalizes the draft guidance of the same title issued 
on October 16, 2018 (83 FR 52225). FDA considered comments received on 
the draft guidance as the guidance was finalized. Changes from the 
draft to the final guidance include editorial changes and 
clarifications throughout the document and the addition of definitions 
for individual-level and trial-level associations.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). This guidance represents the 
current thinking of FDA on ``Hematologic Malignancies: Regulatory 
Considerations for Use of Minimal Residual Disease in Development of 
Drug 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

    This guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The 
collection of information in 21 CFR part 312 for submitting INDs has 
been approved under OMB control

[[Page 4670]]

number 0910-0014. The collection of information in 21 CFR part 314 for 
the submission of new drug applications has been approved under OMB 
control number 0910-0001. The submission of special protocol 
assessments has been approved under OMB control number 0910-0470. The 
submission of biologics license applications has been approved under 
OMB control number 0910-0338. The submission of investigational device 
exemptions has been approved under OMB control number 0910-0078.

III. Electronic Access

    Persons with access to the internet may obtain the 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, or 
https://www.regulations.gov.

    Dated: January 22, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01312 Filed 1-24-20; 8:45 am]
 BILLING CODE 4164-01-P


