
[Federal Register Volume 80, Number 158 (Monday, August 17, 2015)]
[Notices]
[Pages 49244-49246]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-20228]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-2843]


Qualification of Biomarker--Total Kidney Volume in Studies for 
Treatment of Autosomal Dominant Polycystic Kidney Disease; Draft 
Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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[[Page 49245]]

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled 
``Qualification of Biomarker--Total Kidney Volume in Studies for 
Treatment of Autosomal Dominant Polycystic Kidney Disease.'' This draft 
guidance provides a qualified context of use (COU) for total kidney 
volume (TKV), measured at baseline, to be used as a prognostic 
enrichment biomarker to select patients with autosomal dominant 
polycystic kidney disease (ADPKD) at high risk for a ``progressive 
decline'' in renal function, defined as a confirmed 30 percent decline 
in the patient's estimated glomerular filtration rate (eGFR), for 
inclusion in interventional clinical trials. This draft guidance also 
describes the experimental conditions and constraints for which this 
biomarker is qualified through the Center for Drug Evaluation and 
Research (CDER) Biomarker Qualification Program. This biomarker can be 
used by drug developers for the qualified COU in submissions of 
investigational new drug applications, new drug applications, and 
biologics license applications without the relevant CDER review group 
reconsidering and reconfirming the suitability of the biomarker.
    In the Federal Register of January 7, 2014, 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 Drug Development Tools (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qualification recommendations in this draft guidance were developed 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draft guidance before it begins work on the final version of the 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 October 16, 2015.

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, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, 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 ``Qualification of Biomarker--Total Kidney Volume in Studies 
for Treatment of Autosomal Dominant Polycystic Kidney Disease.'' This 
draft guidance provides qualification recommendations for the use of 
TKV, measured at baseline, as a prognostic enrichment biomarker to 
select patients with ADPKD at high risk for a ``progressive decline'' 
in renal function, defined as a confirmed 30 percent decline in the 
patient's eGFR, for inclusion in interventional clinical trials. This 
biomarker may be used in combination with the patient's age and 
baseline eGFR as an enrichment factor in these interventional clinical 
trials. Specifically, this draft 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 investigational new drug applications, 
new drug applications, and biologics license applications 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.
    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 draft 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 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 the use of 
TKV, measured at baseline, as a prognostic enrichment biomarker to 
select patients with ADPKD at high risk for a progressive decline in 
renal function, defined as a confirmed 30 percent decline in eGFR, for 
inclusion in interventional clinical trials. This biomarker may be used 
in combination with patient age and baseline eGFR, as an enrichment 
factor in these interventional clinical trials. It does not establish 
any rights for any person and 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 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 this 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)).

[[Page 49246]]

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.

    Dated: August 12, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-20228 Filed 8-14-15; 8:45 am]
 BILLING CODE 4164-01-P


