Document ID: FDA-2013-D-1630-0009
Agency: fda
Document Type: Notice
Title: Guidance on Qualification of Biomarker—Galactomannan in Studies
of Treatments of Invasive Aspergillosis; Guidance for Industry; Availability
Posted Date: 2015-11-13T05:00Z

[Federal Register Volume 80, Number 219 (Friday, November 13, 2015)]
[Notices]
[Pages 70235-70236]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-28804]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-1630]

Guidance on Qualification of Biomarker--Galactomannan in Studies 
of Treatments of Invasive Aspergillosis; 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) is announcing the 
availability of a guidance for industry entitled ``Guidance on 
Qualification of Biomarker--Galactomannan in Studies of Treatments of 
Invasive Aspergillosis.'' This guidance provides a qualified context of 
use (COU) for Galactomannan detection in serum and/or bronchoalveolar 
lavage (BAL) fluid as the sole microbiological criterion to classify 
patients as having probable invasive Aspergillosis (IA) for enrollment 
in clinical trials. This guidance also describes the experimental 
conditions and constraints for which this biomarker is qualified 
through the CDER Biomarker Qualification Program. This biomarker can be 
used by drug developers for the qualified COU in submissions of 
investigational new drug applications (INDs), new drug applications 
(NDAs), and biologics license applications (BLAs) without the relevant 
CDER review group reconsidering and reconfirming the suitability of the 
biomarker.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
guidance before it begins work on the final version of the guidance, 
submit either electronic or written comments on the guidance by January 
12, 2016

ADDRESSES: You may submit comment as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to http://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 http://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): Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Division of 
Dockets Management, 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-2013-D-1630 for ``Guidance on Qualification of Biomarker--
Galactomannan in Studies of Treatments of Invasive Aspergillosis.'' 
Received comments will be placed in the docket and, except for those 
submitted as ``Confidential Submissions,'' publicly viewable at http://www.regulations.gov or at the Division of Dockets Management 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 http://www.regulations.gov. 
Submit both copies to the Division of Dockets Management. 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: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to http://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 Division of Dockets Management, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Marianne Noone, Center for Drug 
Evaluation and Research (Office of Translational Sciences, Immediate 
Office), Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
21, Rm. 4528, Silver Spring, MD 20993-0002, 301-796-2600.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Guidance on Qualification of Biomarker--Galactomannan in 
Studies of Treatments of Invasive Aspergillosis.'' In the Federal 
Register of October 27, 2014 (79 FR 63921), FDA

[[Page 70236]]

announced the availability of a draft guidance entitled ``Draft 
Guidance on Qualification of Biomarker--Galactomannan in studies of 
Treatments of Invasive Aspergillosis.'' The Agency received one comment 
during the public comment period which was supportive of the 
qualification of this biomarker. This guidance finalizes the draft 
guidance issued in October 2014.
    This guidance provides qualification recommendations for the use of 
Galactomannan detection in serum and/or BAL fluid as the sole 
microbiological criterion to classify patients with hematologic 
malignancies and recipients of allogeneic hematopoietic stem cell 
transplants and who also have radiologic evidence suggestive of 
invasive fungal infection (Ref. 1) as having probable IA for enrollment 
in clinical trials.
    Specifically, this guidance provides the COU for which this 
biomarker is qualified through the CDER Biomarker Qualification 
Program. Qualification of this biomarker for this specific COU 
represents the conclusion that analytically valid measurements of the 
biomarker can be relied on to have a specific use and interpretable 
meaning. This biomarker can be used by drug developers for the 
qualified COU in submission of INDs, NDAs, and BLAs without the 
relevant CDER review group reconsidering and reconfirming the 
suitability of the biomarker. ``Qualification'' means that the use of 
this biomarker in the specific COU is not limited to a single, specific 
drug development program. Making the qualification recommendations 
widely known and available for use by drug developers will contribute 
to drug innovation, thus supporting public health.
    Innovative and improved Drug Development Tools (DDTs) can help 
streamline the drug development process, improve the chances for 
clinical trial success, and yield more information about a treatment 
and/or disease. DDTs include, but are not limited to, biomarkers, 
clinical outcome assessments, and animal models. Refer to DDTs 
Qualification Programs at http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm for additional information.
    In the Federal Register of January 7, 2014 (79 FR 831), FDA 
announced the availability of a final guidance for industry entitled 
``Qualification Process for Drug Development Tools'' that described the 
process that would be used to qualify DDTs and to make new DDT 
qualification recommendations available on FDA's Web site at http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. The current guidance is an attachment to that final 
guidance.
    CDER has initiated this formal qualification process to work with 
developers of these biomarker DDTs to guide them as they refine and 
evaluate DDTs for use in the regulatory context. Once qualified, 
biomarker DDTs will be publicly available for use in any drug 
development program for the qualified COU. As described in the January 
2014 guidance, biomarker DDTs should be developed and reviewed using 
this process. For more information on FDA's DDTs Qualification 
Programs, refer to the following Web page: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
Agency's current thinking for the use of Galactomannan detection in 
serum and/or BAL fluid as the sole microbiological criterion to 
classify patients as having probable IA for enrollment in clinical 
trials. 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. The 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 
collections of information in 21 CFR 312.30, 21 CFR 314.50(d)(5), and 
21 CFR 314.126(b)(6) have been approved under OMB control numbers 0910-
0001 and 0910-0014.

III. Electronic Access

    Persons with access to the Internet may obtain the document at 
either http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

IV. Reference

    The following reference is on display in the Division of Dockets 
Management (see ADDRESSES) and is available for viewing by interested 
persons between 9 a.m. and 4 p.m., Monday through Friday; it is also 
available electronically at http://www.regulations.gov.

1. De Pauw, B., T. J. Walsh, J. P. Donnelly, et al., ``Revised 
Definitions of Invasive Fungal Disease from European Organization 
for Research and Treatment of Cancer/Invasive Fungal Infections 
Cooperative Group and the National Institute of Allergy and 
Infectious Diseases Mycoses Study Group (EORTC/MSG) Consensus 
Group,'' Clinical Infectious Diseases, 46:12, pp. 1813-1821, 2008.

    Dated: November 4, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-28804 Filed 11-12-15; 8:45 am]
BILLING CODE 4164-01-P