[Federal Register Volume 79, Number 207 (Monday, October 27, 2014)]
[Notices]
[Pages 63921-63922]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-25532]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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


Draft Guidance for Industry on Qualification for the Use of 
Galactomannan in Serum and Bronchoalveolar Lavage Fluid; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Draft Guidance 
on Qualification of Biomarker--Galactomannan in Studies of Treatments 
of Invasive Aspergillosis.'' This draft guidance provides 
recommendations on the use of 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 This draft 
guidance provides the context of use for which this biomarker drug 
development tool (DDT) is qualified through the Center for Drug 
Evaluation and Research (CDER) DDT Qualification Program. In the 
Federal Register of January 7, 2014, FDA announced the availability of 
a guidance for industry entitled ``Qualification Process for Drug 
Development Tools,'' which described the process that would be used to 
qualify DDTs and to make new DDT qualification recommendations 
available on FDA's Web site. The qualification recommendations in this 
draft guidance were developed using the process described in that 
guidance.

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 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by December 26, 2014.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 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, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 2201, 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.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 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 draft guidance for industry 
entitled ``Draft Guidance on Qualification of Biomarker--Galactomannan 
in studies of treatments of invasive Aspergillosis.'' This draft 
guidance provides recommendations on 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. The draft guidance provides the context of use for which this 
biomarker DDT is qualified through the CDER DDT Qualification Program. 
Qualification of this biomarker for this specific context of use 
represents the conclusion that analytically valid measurements of the 
biomarker can be relied on to have a specific use and interpretable 
meaning. Further, the biomarker can be used by drug developers for the 
qualified context in submission of investigational new drug 
applications, new drug applications, and biologics licensing 
applications without the relevant CDER review group reconsidering and 
reconfirming the suitability of the DDT. Qualification means that the 
use of this biomarker in the specific context of use 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. The 
draft guidance is an attachment to the guidance for industry entitled 
``Qualification Process for Drug Development Tools.''
    In March 2006, FDA issued the ``Critical Path Opportunities Report 
and List,'' in which FDA described six key areas along the critical 
path to improved therapies and listed specific opportunities for 
advancement within these topic areas. The report noted that a new 
product development toolkit containing new scientific and technical 
methods was needed to improve the efficiency of drug development.
    In 2008, the Mycoses Study Group proposed using Galactomannan in 
serum and BAL fluid as an indicator of IA in lieu of culture in 
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). A 
qualification review team of experts evaluated the data supporting the 
proposed context of use and rendered a qualification recommendation. 
The qualification recommendation in the draft guidance includes the 
following information:
    <bullet> A use statement;
    <bullet> conditions for qualified use of the assay;
    <bullet> patient populations;

[[Page 63922]]

    <bullet> limitations for use of the Galactomannan assay;
    <bullet> considerations for sample acquisition and documentation;
    <bullet> analysis of study results; and
    <bullet> performance characteristics of the assay.
    Innovative and improved 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.
    As stated previously in the Federal Register of January 7, 2014 (79 
FR 831), FDA announced the availability of the guidance for industry 
entitled ``Qualification Process for Drug Development Tools,'' which 
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. CDER has developed this formal process to work 
with developers of these biomarker DDTs to guide the developers as they 
refine the tools and rigorously evaluate them for use in the regulatory 
context. Once qualified, DDTs will be publicly available for use in any 
drug development program for the qualified context of use. As described 
in the January 2014 guidance, biomarker DDTs should be developed and 
reviewed using this process.
    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 Agency's current thinking on 
Galactomannan in serum and BAL fluid. It does not create or confer any 
rights for or on any person and does not operate to bind FDA or the 
public. An alternative approach may be used if such approach satisfies 
the requirements of the applicable statutes and regulations.

II. The Paperwork Reduction Act of 1995

    This guidance contains an information collection that is 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 information collection 
has been approved under the OMB control numbers 0910-0001 and 0910-
0014. The information requested in the guidance is currently submitted 
to FDA to support medical product effectiveness (see 21 CFR 312.30, 21 
CFR 314.50(d)(5), and 21 CFR 314.126(b)(6)).

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. 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.

V. Reference

    The following reference has been placed on display in the Division 
of Dockets Management (see ADDRESSES) and may be seen by interested 
persons between 9 a.m. and 4 p.m., Monday through Friday, and is 
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 (2008) 46: 1813-1821.

    Dated: October 21, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-25532 Filed 10-24-14; 8:45 am]
BILLING CODE 4164-01-P