[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Treatment of Autosomal Dominant Polycystic Kidney Disease; Draft
Guidance for Industry; Availability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the availability of a draft guidance for industry entitled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 This draft
guidance provides a qualified context of use (COU) for total kidney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 This draft guidance also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qualification recommendations in this draft guidance were developed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draft guidance before it begins work on the final version of the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]
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