
[Federal Register Volume 81, Number 180 (Friday, September 16, 2016)]
[Notices]
[Pages 63764-63766]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-22347]


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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; 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 guidance for industry entitled ``Qualification of 
Biomarker--Total Kidney Volume in Studies for Treatment of Autosomal 
Dominant Polycystic Kidney Disease.'' This 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 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 
(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: Submit either electronic or written comments on Agency guidances 
at any time.

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-2015-D-2843 for ``Qualification of Biomarker--Total Kidney Volume 
in Studies for Treatment of Autosomal Dominant Polycystic Kidney 
Disease; Availability.'' 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

[[Page 63765]]

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.
    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. 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: Marianne Noone, Center for Drug 
Evaluation and Research, 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 ``Qualification of Biomarker--Total Kidney Volume in Studies 
for Treatment of Autosomal Dominant Polycystic Kidney Disease.'' In the 
Federal Register of January 7, 2014 (79 FR 831), FDA announced the 
availability of a 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 at http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. The qualification recommendations in the current guidance 
were developed using the process described in that 2014 guidance, and 
the current guidance is an attachment to that 2014 guidance.
    In the Federal Register of August 17, 2015 (80 FR 49244), FDA 
announced the availability of a draft guidance entitled ``Qualification 
of Biomarker--Total Kidney Volume in Studies for Treatment of Autosomal 
Dominant Polycystic Kidney Disease.'' The Agency did not receive any 
comments on that draft guidance during the public comment period. The 
current guidance finalizes that draft guidance.
    This guidance provides 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 guidance provides the COU for which this biomarker 
is qualified through the CDER Biomarker Qualification Program. 
``Biomarker qualification'' is a conclusion that within the stated COU, 
the biomarker can be relied upon to have a specific interpretation and 
application in drug development and regulatory review. 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. After 
a biomarker is qualified for the specific COU, its qualification 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 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 under the animal rule. Refer to DDTs 
Qualification Programs at http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm for additional information.
    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.
    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 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. This guidance 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 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)).

III. Electronic Access

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

[[Page 63766]]

Guidances/default.htm or http://www.regulations.gov.

    Dated: September 12, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-22347 Filed 9-15-16; 8:45 am]
 BILLING CODE 4164-01-P


