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)

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, 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.

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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