83 FR 6582 - Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 31 (February 14, 2018)

Page Range		6582-6583
FR Document		2018-02990

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.

Federal Register, Volume 83 Issue 31 (Wednesday, February 14, 2018)

[Federal Register Volume 83, Number 31 (Wednesday, February 14, 2018)]
[Notices]
[Pages 6582-6583]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-02990]


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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

6582 Federal Register / Vol. 83, No. 31 / Wednesday, February 14, 2018 / Notices

for determining the amount of extension 1. The date an exemption under Dated: February 8, 2018.
an applicant may receive. section 505(i) of the Federal Food, Drug, Leslie Kux,
A regulatory review period consists of and Cosmetic Act (FD&C Act) (21 U.S.C. Associate Commissioner for Policy.
two periods of time: A testing phase and 355(i)) became effective: April 9, 2004. [FR Doc. 2018–03029 Filed 2–13–18; 8:45 am]
an approval phase. For human drug FDA has verified the Warner-Lambert BILLING CODE 4164–01–P
products, the testing phase begins when Company, LLC, claim that April 9, 2004,
the exemption to permit the clinical is the date the investigational new drug
investigations of the drug becomes application (IND) became effective. DEPARTMENT OF HEALTH AND
effective and runs until the approval HUMAN SERVICES
2. The date the application was
phase begins. The approval phase starts
with the initial submission of an initially submitted with respect to the Food and Drug Administration
application to market the human drug human drug product under section
505(b) of the FD&C Act: August 13, [Docket No. FDA–2018–N–0001]
product and continues until FDA grants
permission to market the drug product. 2014. FDA has verified the applicant’s Utilizing Innovative Statistical Methods
Although only a portion of a regulatory claim that the new drug application and Trial Designs in Rare Disease
review period may count toward the (NDA) for IBRANCE (NDA 207103) was Settings; Public Workshop
actual amount of extension that the initially submitted on August 13, 2014.
Director of USPTO may award (for AGENCY: Food and Drug Administration,
3. The date the application was
example, half the testing phase must be HHS.
approved: February 3, 2015. FDA has
subtracted as well as any time that may verified the applicant’s claim that NDA ACTION: Notice of public workshop.
have occurred before the patent was 207103 was approved on February 3,
issued), FDA’s determination of the SUMMARY: The Food and Drug
2015. Administration (FDA) is announcing the
length of a regulatory review period for
a human drug product will include all This determination of the regulatory following 1-day public workshop
of the testing phase and approval phase review period establishes the maximum entitled ‘‘Utilizing Innovative Statistical
as specified in 35 U.S.C. 156(g)(1)(B). potential length of a patent extension. Methods and Trial Designs in Rare
However, the USPTO applies several Disease Settings.’’ This workshop is
FDA has approved for marketing the
statutory limitations in its calculations convened by the Duke-Robert J.
human drug product IBRANCE
of the actual period for patent extension. Margolis, MD, Center for Health Policy
(palbociclib). IBRANCE is indicated for
In its applications for patent extension, at Duke University and supported by a
the treatment of hormone receptor-
this applicant seeks 1,810 days or 1,509 cooperative agreement with FDA. The
positive, human epidermal growth
days of patent term extension. purpose of the public workshop is to
factor receptor 2-negative advanced or
bring rare disease stakeholders together
metastatic breast cancer in combination
III. Petitions to discuss the challenges associated
with:
with the development and regulatory
• An aromatase inhibitor as initial Anyone with knowledge that any of decision-making for rare disease
endocrine based therapy in the dates as published are incorrect may treatments and to also discuss
postmenopausal women; or submit either electronic or written promising study designs and analytical
• fulvestrant in women with disease comments and, under 21 CFR 60.24, ask methods that can help overcome these
progression following endocrine for a redetermination (see DATES). challenges.
therapy. Furthermore, as specified in § 60.30 (21 DATES: The public workshop will be
Subsequent to this approval, the CFR 60.30), any interested person may held on March 19, 2018, from 9 a.m. to
USPTO received patent term restoration petition FDA for a determination 5 p.m. Eastern Daylight Time (EDT). See
applications for IBRANCE (U.S. Patent regarding whether the applicant for the SUPPLEMENTARY INFORMATION section
Nos. 6,936,612 and 7,208,489) from extension acted with due diligence for registration date and information.
Warner-Lambert Company, LLC, and the
during the regulatory review period. To ADDRESSES: The public workshop will
USPTO requested FDA’s assistance in
meet its burden, the petition must be held at the DoubleTree by Hilton
determining the patents’ eligibility for
comply with all the requirements of Hotel Washington DC-Silver Spring,
patent term restoration. In a letter dated
July 12, 2016, FDA advised the USPTO § 60.30, including but not limited to: 8727 Colesville Rd., Silver Spring, MD
that this human drug product had Must be timely (see DATES), must be 20910. For additional travel and hotel
undergone a regulatory review period filed in accordance with § 10.20, must information, please refer to the Duke
and that the approval of IBRANCE contain sufficient facts to merit an FDA Margolis Center for Health Policy
represented the first permitted investigation, and must certify that a website at: https://
commercial marketing or use of the true and complete copy of the petition healthpolicy.duke.edu/events/
product. Thereafter, the USPTO has been served upon the patent innovative-tools-and-statistical-
requested that FDA determine the applicant. (See H. Rept. 857, part 1, 98th methods-treatment-development-rare-
product’s regulatory review period. Cong., 2d sess., pp. 41–42, 1984.) disease-settings.
Petitions should be in the format FOR FURTHER INFORMATION CONTACT:
II. Determination of Regulatory Review Robyn Bent, Center for Drug Evaluation
specified in 21 CFR 10.30.
Period and Research, Food and Drug
Submit petitions electronically to
FDA has determined that the https://www.regulations.gov at Docket Administration, 10903 New Hampshire
daltland on DSKBBV9HB2PROD with NOTICES

applicable regulatory review period for Ave., Silver Spring, MD 20993, 240–
No. FDA–2013–S–0610. Submit written
IBRANCE is 3,954 days. Of this time, 402–2572, Robyn.Bent@fda.hhs.gov.
petitions (two copies are required) to the
3,779 days occurred during the testing SUPPLEMENTARY INFORMATION:
Dockets Management Staff (HFA–305),
phase of the regulatory review period,
Food and Drug Administration, 5630 I. Background
while 175 days occurred during the
approval phase. These periods of time Fishers Lane, Rm. 1061, Rockville, MD Rare disease settings pose several
were derived from the following dates: 20852. significant challenges for clinical

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Document Created: 2018-02-14 03:59:28
Document Modified: 2018-02-14 03:59:28

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 public workshop.
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.
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]
FR Citation		83 FR 6582

83 FR 6582 - Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 83, Issue 31 (February 14, 2018)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration