Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings; Public Workshop, 6582-6583 [2018-02990]
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Federal Register / Vol. 83, No. 31 / Wednesday, February 14, 2018 / Notices
for determining the amount of extension
an applicant may receive.
A regulatory review period consists of
two periods of time: A testing phase and
an approval phase. For human drug
products, the testing phase begins when
the exemption to permit the clinical
investigations of the drug becomes
effective and runs until the approval
phase begins. The approval phase starts
with the initial submission of an
application to market the human drug
product and continues until FDA grants
permission to market the drug product.
Although only a portion of a regulatory
review period may count toward the
actual amount of extension that the
Director of USPTO may award (for
example, half the testing phase must be
subtracted as well as any time that may
have occurred before the patent was
issued), FDA’s determination of the
length of a regulatory review period for
a human drug product will include all
of the testing phase and approval phase
as specified in 35 U.S.C. 156(g)(1)(B).
FDA has approved for marketing the
human drug product IBRANCE
(palbociclib). IBRANCE is indicated for
the treatment of hormone receptorpositive, human epidermal growth
factor receptor 2-negative advanced or
metastatic breast cancer in combination
with:
• An aromatase inhibitor as initial
endocrine based therapy in
postmenopausal women; or
• fulvestrant in women with disease
progression following endocrine
therapy.
Subsequent to this approval, the
USPTO received patent term restoration
applications for IBRANCE (U.S. Patent
Nos. 6,936,612 and 7,208,489) from
Warner-Lambert Company, LLC, and the
USPTO requested FDA’s assistance in
determining the patents’ eligibility for
patent term restoration. In a letter dated
July 12, 2016, FDA advised the USPTO
that this human drug product had
undergone a regulatory review period
and that the approval of IBRANCE
represented the first permitted
commercial marketing or use of the
product. Thereafter, the USPTO
requested that FDA determine the
product’s regulatory review period.
daltland on DSKBBV9HB2PROD with NOTICES
II. Determination of Regulatory Review
Period
FDA has determined that the
applicable regulatory review period for
IBRANCE is 3,954 days. Of this time,
3,779 days occurred during the testing
phase of the regulatory review period,
while 175 days occurred during the
approval phase. These periods of time
were derived from the following dates:
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22:07 Feb 13, 2018
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1. The date an exemption under
section 505(i) of the Federal Food, Drug,
and Cosmetic Act (FD&C Act) (21 U.S.C.
355(i)) became effective: April 9, 2004.
FDA has verified the Warner-Lambert
Company, LLC, claim that April 9, 2004,
is the date the investigational new drug
application (IND) became effective.
2. The date the application was
initially submitted with respect to the
human drug product under section
505(b) of the FD&C Act: August 13,
2014. FDA has verified the applicant’s
claim that the new drug application
(NDA) for IBRANCE (NDA 207103) was
initially submitted on August 13, 2014.
3. The date the application was
approved: February 3, 2015. FDA has
verified the applicant’s claim that NDA
207103 was approved on February 3,
2015.
This determination of the regulatory
review period establishes the maximum
potential length of a patent extension.
However, the USPTO applies several
statutory limitations in its calculations
of the actual period for patent extension.
In its applications for patent extension,
this applicant seeks 1,810 days or 1,509
days of patent term extension.
III. Petitions
Anyone with knowledge that any of
the dates as published are incorrect may
submit either electronic or written
comments and, under 21 CFR 60.24, ask
for a redetermination (see DATES).
Furthermore, as specified in § 60.30 (21
CFR 60.30), any interested person may
petition FDA for a determination
regarding whether the applicant for
extension acted with due diligence
during the regulatory review period. To
meet its burden, the petition must
comply with all the requirements of
§ 60.30, including but not limited to:
Must be timely (see DATES), must be
filed in accordance with § 10.20, must
contain sufficient facts to merit an FDA
investigation, and must certify that a
true and complete copy of the petition
has been served upon the patent
applicant. (See H. Rept. 857, part 1, 98th
Cong., 2d sess., pp. 41–42, 1984.)
Petitions should be in the format
specified in 21 CFR 10.30.
Submit petitions electronically to
https://www.regulations.gov at Docket
No. FDA–2013–S–0610. Submit written
petitions (two copies are required) to the
Dockets Management Staff (HFA–305),
Food and Drug Administration, 5630
Fishers Lane, Rm. 1061, Rockville, MD
20852.
PO 00000
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Dated: February 8, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–03029 Filed 2–13–18; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–N–0001]
Utilizing Innovative Statistical Methods
and Trial Designs in Rare Disease
Settings; Public Workshop
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of public workshop.
The Food and Drug
Administration (FDA) is announcing the
following 1-day public workshop
entitled ‘‘Utilizing Innovative Statistical
Methods and Trial Designs in Rare
Disease Settings.’’ This workshop is
convened by the Duke-Robert J.
Margolis, MD, Center for Health Policy
at Duke University and supported by a
cooperative agreement with FDA. The
purpose of the public workshop is to
bring rare disease stakeholders together
to discuss the challenges associated
with the development and regulatory
decision-making for rare disease
treatments and to also discuss
promising study designs and analytical
methods that can help overcome these
challenges.
DATES: The public workshop will be
held on March 19, 2018, from 9 a.m. to
5 p.m. Eastern Daylight Time (EDT). See
the SUPPLEMENTARY INFORMATION section
for registration date and information.
ADDRESSES: The public workshop will
be held at the DoubleTree by Hilton
Hotel Washington DC-Silver Spring,
8727 Colesville Rd., Silver Spring, MD
20910. For additional travel and hotel
information, please refer to the Duke
Margolis Center for Health Policy
website at: https://
healthpolicy.duke.edu/events/
innovative-tools-and-statisticalmethods-treatment-development-raredisease-settings.
FOR FURTHER INFORMATION CONTACT:
Robyn Bent, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993, 240–
402–2572, Robyn.Bent@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
SUMMARY:
I. Background
Rare disease settings pose several
significant challenges for clinical
E:\FR\FM\14FEN1.SGM
14FEN1
Federal Register / Vol. 83, No. 31 / Wednesday, February 14, 2018 / Notices
research, drug development, and
regulatory review. Small population
sizes, possible limited scientific
understanding of the disease of interest,
and a lack of market incentives often
preclude more traditional clinical trial
or analytical approaches from being
pursued. To help collaboratively
address these barriers, FDA is working
with stakeholders to solicit feedback on
promising designs and methodologies
for use in the development of rare
disease treatments that can form the
basis of formal guidance documents.
daltland on DSKBBV9HB2PROD with NOTICES
II. Topics for Discussion at the Public
Workshop
During the public workshop, speakers
and participants will discuss a range of
tools and methods that can be used in
the development of treatments for rare
diseases and small patient populations.
The meeting will include both
presentations by panelists and
dedicated time for questions and
comments from attendees. Topics will
include: Master protocols, use of
external controls in single-arm trials,
analytical tools for trials with multiple
or novel endpoints, and best practices
for leveraging Bayesian statistics and
adaptive study designs.
III. Participating in the Public
Workshop
Registration: To register for the public
workshop, visit the following website:
https://healthpolicy.duke.edu/events/
innovative-tools-and-statisticalmethods-treatment-development-raredisease-settings. If you are unable to
attend the meeting in person, you can
register to view a live webcast of the
meeting. There will be no onsite
registration. Please provide complete
contact information for each attendee,
including name, title, affiliation,
address, email, and telephone.
Registration is free and based on
space availability, with priority given to
early registrants. Persons interested in
attending this public workshop must
register by 5 p.m. EDT on Thursday,
March 15, 2018. Early registration is
recommended because seating is
limited; therefore, FDA may limit the
number of participants from each
organization.
If you need special accommodations
due to a disability, please contact Sarah
Supsiri at the Duke-Margolis Center for
Health Policy (phone: 202–791–9561,
email: sarah.supsiri@duke.edu) no later
than March 12, 2018.
Streaming webcast of the public
workshop: This public workshop will
also be webcast. Archived video footage
will also be available at the DukeMargolis website following the
VerDate Sep<11>2014
22:07 Feb 13, 2018
Jkt 244001
workshop (https://
healthpolicy.duke.edu/events/
innovative-tools-and-statisticalmethods-treatment-development-raredisease-settings). Persons interested in
viewing the live webcast must register
online before 5 p.m. EDT on March 18,
2018 (see Registration). Early
registration is recommended because
webcast connections are limited.
Organizations are requested to register
all participants, but to view using one
connection per location whenever
possible. Webcast participants will be
sent technical system requirements in
advance of the event. Prior to joining the
streaming webcast of the public
workshop, it is recommended that you
review these technical system
requirements.
Transcripts: Please be advised that
transcripts will not be available.
Other Issues for Consideration: A 1hour lunch break is scheduled, but food
will not be provided. There are multiple
restaurants within walking distance of
the DoubleTree by Hilton Hotel, 8727
Colesville Rd., Silver Spring, MD 20910.
All event materials will be provided
to registered attendees via email prior to
the workshop and will be publicly
available at the Duke-Margolis Center
for Health Policy website (https://
healthpolicy.duke.edu/events/
innovative-tools-and-statisticalmethods-treatment-development-raredisease-settings).
Dated: February 7, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–02990 Filed 2–13–18; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2015–E–2576]
Determination of Regulatory Review
Period for Purposes of Patent
Extension; JARDIANCE
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA or Agency) has
determined the regulatory review period
for JARDIANCE and is publishing this
notice of that determination as required
by law. FDA has made the
determination because of the
submission of an application to the
Director of the U.S. Patent and
Trademark Office (USPTO), Department
SUMMARY:
PO 00000
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6583
of Commerce, for the extension of a
patent which claims that human drug
product.
Anyone with knowledge that any
of the dates as published (in the
SUPPLEMENTARY INFORMATION section) are
incorrect may submit either electronic
or written comments and ask for a
redetermination by April 16, 2018. See
‘‘Petitions’’ in the SUPPLEMENTARY
INFORMATION section for more
information.
DATES:
You may submit comments
as follows. Please note that late,
untimely filed comments will not be
considered. Electronic comments must
be submitted on or before April 16,
2018. The https://www.regulations.gov
electronic filing system will accept
comments until midnight Eastern Time
at the end of April 16, 2018. Comments
received by mail/hand delivery/courier
(for written/paper submissions) will be
considered timely if they are
postmarked or the delivery service
acceptance receipt is on or before that
date. Furthermore, any interested
person may petition FDA for a
determination regarding whether the
applicant for extension acted with due
diligence during the regulatory review
period by August 13, 2018. See
‘‘Petitions’’ in the SUPPLEMENTARY
INFORMATION section for more
information.
ADDRESSES:
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
instructions for submitting comments.
Comments submitted electronically,
including attachments, to https://
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 https://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’’).
E:\FR\FM\14FEN1.SGM
14FEN1
]]>Agencies
[Federal Register Volume 83, Number 31 (Wednesday, February 14, 2018)]
[Notices]
[Pages 6582-6583]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-02990]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-N-0001]
Utilizing Innovative Statistical Methods and Trial Designs in
Rare Disease Settings; Public Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing the
following 1-day public workshop entitled ``Utilizing Innovative
Statistical Methods and Trial Designs in Rare Disease Settings.'' This
workshop is convened by the Duke-Robert J. Margolis, MD, Center for
Health Policy at Duke University and supported by a cooperative
agreement with FDA. The purpose of the public workshop is to bring rare
disease stakeholders together to discuss the challenges associated with
the development and regulatory decision-making for rare disease
treatments and to also discuss promising study designs and analytical
methods that can help overcome these challenges.
DATES: The public workshop will be held on March 19, 2018, from 9 a.m.
to 5 p.m. Eastern Daylight Time (EDT). See the SUPPLEMENTARY
INFORMATION section for registration date and information.
ADDRESSES: The public workshop will be held at the DoubleTree by Hilton
Hotel Washington DC-Silver Spring, 8727 Colesville Rd., Silver Spring,
MD 20910. For additional travel and hotel information, please refer to
the Duke Margolis Center for Health Policy website at: https://healthpolicy.duke.edu/events/innovative-tools-and-statistical-methods-treatment-development-rare-disease-settings.
FOR FURTHER INFORMATION CONTACT: Robyn Bent, Center for Drug Evaluation
and Research, Food and Drug Administration, 10903 New Hampshire Ave.,
Silver Spring, MD 20993, 240-402-2572, [email protected].
SUPPLEMENTARY INFORMATION:
I. Background
Rare disease settings pose several significant challenges for
clinical
[[Page 6583]]
research, drug development, and regulatory review. Small population
sizes, possible limited scientific understanding of the disease of
interest, and a lack of market incentives often preclude more
traditional clinical trial or analytical approaches from being pursued.
To help collaboratively address these barriers, FDA is working with
stakeholders to solicit feedback on promising designs and methodologies
for use in the development of rare disease treatments that can form the
basis of formal guidance documents.
II. Topics for Discussion at the Public Workshop
During the public workshop, speakers and participants will discuss
a range of tools and methods that can be used in the development of
treatments for rare diseases and small patient populations. The meeting
will include both presentations by panelists and dedicated time for
questions and comments from attendees. Topics will include: Master
protocols, use of external controls in single-arm trials, analytical
tools for trials with multiple or novel endpoints, and best practices
for leveraging Bayesian statistics and adaptive study designs.
III. Participating in the Public Workshop
Registration: To register for the public workshop, visit the
following website: https://healthpolicy.duke.edu/events/innovative-tools-and-statistical-methods-treatment-development-rare-disease-settings. If you are unable to attend the meeting in person, you can
register to view a live webcast of the meeting. There will be no onsite
registration. Please provide complete contact information for each
attendee, including name, title, affiliation, address, email, and
telephone.
Registration is free and based on space availability, with priority
given to early registrants. Persons interested in attending this public
workshop must register by 5 p.m. EDT on Thursday, March 15, 2018. Early
registration is recommended because seating is limited; therefore, FDA
may limit the number of participants from each organization.
If you need special accommodations due to a disability, please
contact Sarah Supsiri at the Duke-Margolis Center for Health Policy
(phone: 202-791-9561, email: [email protected]) no later than
March 12, 2018.
Streaming webcast of the public workshop: This public workshop will
also be webcast. Archived video footage will also be available at the
Duke-Margolis website following the workshop (https://healthpolicy.duke.edu/events/innovative-tools-and-statistical-methods-treatment-development-rare-disease-settings). Persons interested in
viewing the live webcast must register online before 5 p.m. EDT on
March 18, 2018 (see Registration). Early registration is recommended
because webcast connections are limited. Organizations are requested to
register all participants, but to view using one connection per
location whenever possible. Webcast participants will be sent technical
system requirements in advance of the event. Prior to joining the
streaming webcast of the public workshop, it is recommended that you
review these technical system requirements.
Transcripts: Please be advised that transcripts will not be
available.
Other Issues for Consideration: A 1-hour lunch break is scheduled,
but food will not be provided. There are multiple restaurants within
walking distance of the DoubleTree by Hilton Hotel, 8727 Colesville
Rd., Silver Spring, MD 20910.
All event materials will be provided to registered attendees via
email prior to the workshop and will be publicly available at the Duke-
Margolis Center for Health Policy website (https://healthpolicy.duke.edu/events/innovative-tools-and-statistical-methods-treatment-development-rare-disease-settings).
Dated: February 7, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-02990 Filed 2-13-18; 8:45 am]
BILLING CODE 4164-01-P
