81 FR 63764 - Qualification of Biomarker-Total Kidney Volume in Studies for Treatment of Autosomal Dominant Polycystic Kidney Disease; Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 81, Issue 180 (September 16, 2016)
Food and Drug Administration
Federal Register Volume 81, Issue 180 (September 16, 2016)
| Page Range | 63764-63766 | |
| FR Document | 2016-22347 |
[Federal Register Volume 81, Number 180 (Friday, September 16, 2016)] [Notices] [Pages 63764-63766] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2016-22347] ----------------------------------------------------------------------- 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. ----------------------------------------------------------------------- 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
![63764 Federal Register / Vol. 81, No. 180 / Friday, September 16, 2016 / Notices
ANNUAL BURDEN ESTIMATES
Total Annual Number of Annual
Average burden hours per
Instrument number of number of responses per burden
response
respondents respondents respondent hours
Entry Survey ..................................... 414,747 138,249 1 0.13333 ............................................ 18,433
Exit Survey ........................................ 331,797 110,599 1 0.25 .................................................. 27,650
Core measures ................................. 16,000 5,333 3 0.08 .................................................. 1,280
Performance Measures Data Report 279 93 2 18 for S/T; 14 for CPREP and 3,076
Form (grantees). PREIS.
Performance Measures Data Report 1,248 416 2 14 for S/T; 12 for CPREP ................ 11,472
Form (sub-awardees).
Estimated Total Annual Burden ........................ ........................ ........................ ........................................................... 61,911
Hours.
Additional Information: Copies of the guidance for industry entitled such as medical information, your or
proposed collection may be obtained by ‘‘Qualification of Biomarker—Total anyone else’s Social Security number, or
writing to the Administration for Kidney Volume in Studies for confidential business information, such
Children and Families, Office of Treatment of Autosomal Dominant as a manufacturing process. Please note
Planning, Research and Evaluation, 330 Polycystic Kidney Disease.’’ This that if you include your name, contact
C Street SW., Washington, DC 20201, guidance provides a qualified context of information, or other information that
Attn: OPRE Reports Clearance Officer. use (COU) for total kidney volume identifies you in the body of your
All requests should be identified by the (TKV), measured at baseline, to be used comments, that information will be
title of the information collection. Email as a prognostic enrichment biomarker to posted on http://www.regulations.gov.
address: OPREinfocollection@ select patients with autosomal dominant • If you want to submit a comment
acf.hhs.gov. polycystic kidney disease (ADPKD) at with confidential information that you
OMB Comment: OMB is required to high risk for a ‘‘progressive decline’’ in do not wish to be made available to the
make a decision concerning the renal function, defined as a confirmed public submit the comment as a written/
collection of information between 30 30 percent decline in the patient’s paper submission and in the manner
and 60 days after publication of this estimated glomerular filtration rate detailed (see ‘‘Written/Paper
document in the Federal Register. (eGFR), for inclusion in interventional Submissions’’ and ‘‘Instructions’’).
Therefore, a comment is best assured of clinical trials. This guidance also
having its full effect if OMB receives it describes the experimental conditions Written/Paper Submissions
within 30 days of publication. Written and constraints for which this Submit written/paper submissions as
comments and recommendations for the biomarker is qualified through the follows:
proposed information collection should Center for Drug Evaluation and Research • Mail/Hand delivery/Courier (for
be sent directly to the following: Office (CDER) Biomarker Qualification written/paper submissions): Division of
of Management and Budget, Paperwork Program. This biomarker can be used by Dockets Management (HFA–305), Food
Reduction Project. Email: OIRA_ drug developers for the qualified COU and Drug Administration, 5630 Fishers
SUBMISSION@OMB.EOP.GOV. Attn: in submissions of investigational new Lane, Rm. 1061, Rockville, MD 20852.
Desk Officer for the Administration for drug applications (INDs), new drug • For written/paper comments
Children and Families. applications (NDAs), and biologics submitted to the Division of Dockets
license applications (BLAs)without the Management, FDA will post your
Naomi Goldstein,
relevant CDER review group comment, as well as any attachments,
ACF/OPRE Certifying Officer. except for information submitted,
reconsidering and reconfirming the
[FR Doc. 2016–22316 Filed 9–15–16; 8:45 am] suitability of the biomarker. marked and identified, as confidential,
BILLING CODE 4184–37–P
DATES: Submit either electronic or if submitted as detailed in
written comments on Agency guidances ‘‘Instructions.’’
at any time. Instructions: All submissions received
DEPARTMENT OF HEALTH AND must include the Docket No. FDA–
HUMAN SERVICES ADDRESSES: You may submit comment
as follows: 2015–D–2843 for ‘‘Qualification of
Biomarker—Total Kidney Volume in
Food and Drug Administration Electronic Submissions Studies for Treatment of Autosomal
[Docket No. FDA–2015–D–2843] Submit electronic comments in the Dominant Polycystic Kidney Disease;
following way: Availability.’’ Received comments will
Qualification of Biomarker—Total • Federal eRulemaking Portal: http:// be placed in the docket and, except for
Kidney Volume in Studies for www.regulations.gov. Follow the those submitted as ‘‘Confidential
Treatment of Autosomal Dominant instructions for submitting comments. Submissions,’’ publicly viewable at
Polycystic Kidney Disease; Guidance Comments submitted electronically, http://www.regulations.gov or at the
for Industry; Availability including attachments, to http:// Division of Dockets Management
mstockstill on DSK3G9T082PROD with NOTICES
AGENCY: Food and Drug Administration, www.regulations.gov will be posted to between 9 a.m. and 4 p.m., Monday
HHS. the docket unchanged. Because your through Friday.
ACTION: Notice of availability. comment will be made public, you are • Confidential Submissions—To
solely responsible for ensuring that your submit a comment with confidential
SUMMARY: The Food and Drug comment does not include any information that you do not wish to be
Administration (FDA or Agency) is confidential information that you or a made publicly available submit your
announcing the availability of a third party may not wish to be posted, comments only as a written/paper
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![63766 Federal Register / Vol. 81, No. 180 / Friday, September 16, 2016 / Notices
Guidances/default.htm or http:// solely responsible for ensuring that your copies to the Division of Dockets
www.regulations.gov. comment does not include any Management. If you do not wish your
Dated: September 12, 2016. confidential information that you or a name and contact information to be
Leslie Kux,
third party may not wish to be posted, made publicly available, you can
such as medical information, your or provide this information on the cover
Associate Commissioner for Policy.
anyone else’s Social Security number, or sheet and not in the body of your
[FR Doc. 2016–22347 Filed 9–15–16; 8:45 am] confidential business information, such comments and you must identify this
BILLING CODE 4164–01–P as a manufacturing process. Please note information as ‘‘confidential.’’ Any
that if you include your name, contact information marked as ‘‘confidential’’
information, or other information that will not be disclosed except in
DEPARTMENT OF HEALTH AND identifies you in the body of your accordance with 21 CFR 10.20 and other
HUMAN SERVICES comments, that information will be applicable disclosure law. For more
posted on http://www.regulations.gov. information about FDA’s posting of
Food and Drug Administration
• If you want to submit a comment comments to public dockets, see 80 FR
[Docket No. FDA–2015–D–3399] with confidential information that you 56469, September 18, 2015, or access
do not wish to be made available to the the information at: http://www.fda.gov/
Recommendations for Microbial public, submit the comment as a regulatoryinformation/dockets/
Vectors Used for Gene Therapy; written/paper submission and in the default.htm.
Guidance for Industry; Availability manner detailed (see ‘‘Written/Paper Docket: For access to the docket to
AGENCY: Food and Drug Administration, Submissions’’ and ‘‘Instructions’’). read background documents or the
HHS. Written/Paper Submissions electronic and written/paper comments
ACTION: Notice of availability. Submit written/paper submissions as received, go to http://
follows: www.regulations.gov and insert the
SUMMARY: The Food and Drug • Mail/Hand delivery/Courier (for docket number, found in brackets in the
Administration (FDA or Agency) is written/paper submissions): Division of heading of this document, into the
announcing the availability of a Dockets Management (HFA–305), Food ‘‘Search’’ box and follow the prompts
document entitled ‘‘Recommendations and Drug Administration, 5630 Fishers and/or go to the Division of Dockets
for Microbial Vectors Used for Gene Lane, Rm. 1061, Rockville, MD 20852. Management, 5630 Fishers Lane, Rm.
Therapy; Guidance for Industry.’’ The • For written/paper comments 1061, Rockville, MD 20852.
guidance document provides submitted to the Division of Dockets Submit written requests for single
investigational new drug application Management, FDA will post your copies of the guidance to the Office of
(IND) sponsors, with recommendations comment, as well as any attachments, Communication, Outreach and
concerning IND submissions for except for information submitted, Development, Center for Biologics
microbial vectors used for gene therapy marked and identified, as confidential, Evaluation and Research (CBER), Food
(MVGTs) in early phase clinical trials. if submitted as detailed in and Drug Administration, 10903 New
The guidance focuses on the chemistry, ‘‘Instructions.’’ Hampshire Ave., Bldg. 71, Rm. 3128,
manufacturing, and control (CMC) Instructions: All submissions received Silver Spring, MD 20993–0002. Send
information that sponsors should must include the Docket No. FDA– one self-addressed adhesive label to
submit in an IND for MVGTs and 2015–D–3399 for ‘‘Recommendations assist the office in processing your
provides an overview of preclinical and for Microbial Vectors Used for Gene requests. The guidance may also be
clinical considerations for these Therapy; Guidance for Industry.’’ obtained by mail by calling CBER at 1–
products. The guidance announced in Received comments will be placed in 800–835–4709 or 240–402–8010. See
this notice finalizes the draft guidance the docket and, except for those the SUPPLEMENTARY INFORMATION section
of the same title dated October 2015 and submitted as ‘‘Confidential for electronic access to the guidance
supplements the guidance entitled Submissions,’’ publicly viewable at document.
‘‘Guidance for FDA Reviewers and http://www.regulations.gov or at the
Sponsors: Content and Review of FOR FURTHER INFORMATION CONTACT:
Division of Dockets Management
Chemistry, Manufacturing, and Control Tami Belouin, Center for Biologics
between 9 a.m. and 4 p.m., Monday
(CMC) Information for Human Gene Evaluation and Research, Food and
through Friday.
Therapy Investigational New Drug • Confidential Submissions—To Drug Administration, 10903 New
Applications (INDs),’’ dated April 2008. submit a comment with confidential Hampshire Ave., Bldg. 71, Rm. 7301,
DATES: Submit either electronic or information that you do not wish to be Silver Spring, MD 20993–0002, 240–
written comments on Agency guidances made publicly available, submit your 402–7911.
at any time. comments only as a written/paper SUPPLEMENTARY INFORMATION:
ADDRESSES: You may submit comments submission. You should submit two
I. Background
as follows: copies total. One copy will include the
information you claim to be confidential FDA is announcing the availability of
Electronic Submissions with a heading or cover note that states a document entitled ‘‘Recommendations
Submit electronic comments in the ‘‘THIS DOCUMENT CONTAINS for Microbial Vectors Used for Gene
following way: CONFIDENTIAL INFORMATION.’’ The Therapy; Guidance for Industry.’’ The
• Federal eRulemaking Portal: http:// Agency will review this copy, including guidance provides IND sponsors, with
mstockstill on DSK3G9T082PROD with NOTICES
www.regulations.gov. Follow the the claimed confidential information, in recommendations concerning IND
instructions for submitting comments. its consideration of comments. The submissions for microbial vectors used
Comments submitted electronically, second copy, which will have the for MVGTs in early phase clinical trials.
including attachments, to http:// claimed confidential information The guidance focuses on the CMC
www.regulations.gov will be posted to redacted/blacked out, will be available information that sponsors should
the docket unchanged. Because your for public viewing and posted on http:// submit in an IND for MVGTs and
comment will be made public, you are www.regulations.gov. Submit both provides an overview of preclinical and
VerDate Sep<11>2014 17:55 Sep 15, 2016 Jkt 238001 PO 00000 Frm 00032 Fmt 4703 Sfmt 4703 E:\FR\FM\16SEN1.SGM 16SEN1](https://thefederalregister.org/doc/81_FR_63944/page/3.svg)
Document Created: 2016-09-16 00:25:56
Document Modified: 2016-09-16 00:25:56
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of availability. | |
| Dates | Submit either electronic or written comments on Agency guidances at any time. | |
| 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. | |
| FR Citation | 81 FR 63764 |
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