Document ID: FDA-2016-D-3456-0001
Agency: fda
Document Type: Notice
Title: Bacillus Calmette-Guerin--Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biologics for Treatment; Draft Guidance 
for Industry; Availability
Posted Date: 2016-11-18T05:00Z

[Federal Register Volume 81, Number 223 (Friday, November 18, 2016)]
[Notices]
[Pages 81778-81779]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-27762]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2016-D-3456]

Bacillus Calmette-Guerin--Unresponsive Nonmuscle Invasive Bladder 
Cancer: Developing Drugs and Biologics for Treatment; Draft Guidance 
for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: Summary: The Food and Drug Administration (FDA or Agency) is 
announcing the availability of a draft guidance for industry entitled 
``Bacillus Calmette-Guerin (BCG)--Unresponsive Nonmuscle Invasive 
Bladder Cancer: Developing Drugs and Biologics for Treatment.'' The 
purpose of this guidance is to assist sponsors in the development of 
drugs and biologics to treat patients with a high-risk form of bladder 
cancer. The alternative is radical cystectomy, a surgical procedure 
with significant morbidity and mortality. This guidance will help 
overcome some of the obstacles in conducting the studies needed to 
establish efficacy of drugs and biologics for these patients with an 
unmet medical need.

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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by February 16, 2017.

ADDRESSES: You may submit comments 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-2016-D-3456] for ``BCG-Unresponsive Nonmuscle Invasive Bladder 
Cancer: Developing Drugs and Biologics for Treatment; Draft Guidance 
for Industry; 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 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 the draft guidance to 
the

[[Page 81779]]

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, or the Office of 
Communication, Outreach, and Development, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, 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.

FOR FURTHER INFORMATION CONTACT: V. Ellen Maher, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2352, Silver Spring, MD 20993-0002, 301-
796-5017; or Stephen Ripley, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: 
Developing Drugs and Biologics for Treatment.'' This draft guidance is 
intended to provide a framework for industry to facilitate the 
development of drugs and biologics to treat patients with nonmuscle 
invasive bladder cancer (NMIBC). The focus is on the subset of patients 
with BCG-unresponsive disease. In addition, the pathological diagnosis 
and staging, risk stratification, and trial design, including 
assessment of appropriate clinical endpoints, are discussed.
    The preferred trial design for demonstrating efficacy of drugs 
developed to treat NMIBC is a randomized, controlled trial with a time-
to-event endpoint of recurrence-free survival. Single-arm trials are 
appropriate in clinical settings for which a randomized, controlled 
trial is either unethical or not feasible. Therefore, single-arm trials 
of patients with BCG-unresponsive carcinoma in situ with or without 
papillary disease using an endpoint of complete response rate (and 
duration) may be appropriate.
    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 current thinking of FDA on developing 
drugs and biologics for the treatment of BCG-unresponsive NMIBC. It 
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 draft guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are 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 
collections of information in 21 CFR parts 312 and 314 have been 
approved under OMB control numbers 0910-0014 and 0910-0001, 
respectively.

III. Electronic Access

    Persons with access to the Internet may obtain the draft guidance 
at http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or http://www.regulations.gov.

    Dated: November 14, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-27762 Filed 11-17-16; 8:45 am]
 BILLING CODE 4164-01-P