[Federal Register Volume 82, Number 145 (Monday, July 31, 2017)]
[Notices]
[Pages 35534-35535]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-16021]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0001]


Developing a Framework for Regulatory Use of Real-World Evidence; 
Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is 
announcing the following public workshop entitled ``Developing a 
Framework for Regulatory Use of Real-World Evidence.'' 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 the stakeholder community 
together to discuss a variety of topics related to the use of real-
world data (RWD) and real-world evidence (RWE) in drug development and 
regulatory decision making. Topics will include an update on FDA's 
activities to address the use of RWE in regulatory decisions and the 
development of a framework for tackling challenges related to RWE's 
regulatory acceptability. In addition, panelists will discuss 
opportunities to improve data development activities, study designs, 
and analytical methods used to create robust RWE.

DATES: The public workshop will be held on September 13, 2017, from 9 
a.m. to 4:30 p.m., Eastern Time.

ADDRESSES: The public workshop will be held at the Conference Center at 
1777 F Street NW., Washington, DC 20006. For additional travel and 
hotel information, please refer to the following Web site: https://healthpolicy.duke.edu/events/public-workshop-developing-framework-regulatory-use-real-world-evidence. There will also be a live webcast 
for those unable to attend the meeting in person (see Streaming Webcast 
of Public Workshop).

FOR FURTHER INFORMATION CONTACT: Kayla Garvin, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 6314, Silver Spring, MD 20993, (301) 796-
7578, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    RWD (data relating to patient health status and/or the delivery of 
health care routinely collected from a variety of sources) and RWE 
(clinical evidence regarding the usage and potential benefits or risks 
of a drug derived from analysis of RWD) are increasingly being used by 
multiple stakeholders within the health care system. Payers may rely on 
RWD and RWE to refine formularies or assist in coverage decisions. 
Physicians and professional societies can utilize RWE to further tailor 
clinical practice guidelines and decision-support tools. Medical 
product developers can use RWE to further develop a product's benefit-
risk profile, monitor postmarket safety and adverse events, or generate 
additional hypotheses for continued clinical development.
    The 21st Century Cures Act, section 3022 (Pub. L. 114-255), enacted 
on December 13, 2016, directed FDA to establish a program to evaluate 
the potential use of RWE. The framework of the program was to include 
information describing the sources of RWE, the gaps in data collection, 
standards and methods for collection and analysis, and the priority 
areas and challenges.
    To date, RWD and RWE have been used in very specific regulatory 
contexts. Some treatments for rare diseases, for example, have utilized 
RWE as part of the historical controls used for clinical study and, 
ultimately, regulatory submission. Postmarket safety surveillance has 
also relied heavily on RWD-generating networks. As part of exploring 
the opportunities for enhanced use of these types of data and evidence 
in additional regulatory decision-making contexts, FDA is seeking input 
on the opportunities and challenges in using RWE to support the 
approval of a new indication for an already approved drug, and to help 
support or satisfy postapproval study requirements.
    This public workshop is being held to engage external stakeholders 
in discussions around the current state of RWE development and 
potential challenge areas for using RWE in regulatory decisions beyond 
postmarket safety surveillance.

II. Topics for Discussion at the Public Workshop

    During the course of the public workshop, speakers and participants 
will cover a range of issues related to

[[Page 35535]]

the development of RWE and its applicability within specific regulatory 
decision-making contexts. This will include, but not be limited to, 
challenges related to RWD collection and quality, innovative methods 
for developing RWE from RWD, and promising areas for RWE pilot 
demonstrations.

III. Participating in the Public Workshop

    Registration: To register for the public workshop, please visit the 
following Web site before September 12, 2017: https://healthpolicy.duke.edu/events/public-workshop-developing-framework-regulatory-use-real-world-evidence. 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. Early registration is recommended because 
seating is limited; therefore, FDA may limit the number of participants 
from each organization. A 1-hour lunch break is scheduled, but food 
will not be provided. There are multiple restaurants within walking 
distance of the Conference Center.
    If you need special accommodations due to a disability, please 
contact Joanna Higgison at the Duke-Margolis Center for Health Policy, 
908-432-4872, [email protected], no later than September 6, 
2017.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be webcast and archived video footage will be available at the 
Duke-Margolis Web site (https://healthpolicy.duke.edu/events/public-workshop-developing-framework-regulatory-use-real-world-evidence) 
following the workshop. Persons interested in viewing the live webcast 
must register online by September 12, 2017, at 5 p.m. Eastern Time (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.
    FDA has verified the Web site addresses in this document, as of the 
date this document publishes in the Federal Register, but Web sites are 
subject to change over time.
    Meeting Materials: All event materials will be provided to 
registered attendees via email prior to the workshop and publicly 
available at the Duke-Margolis Web site (https://healthpolicy.duke.edu/events/public-workshop-developing-framework-regulatory-use-real-world-evidence).
    Transcripts: Please be advised that transcripts will not be 
available.

    Dated: July 25, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-16021 Filed 7-28-17; 8:45 am]
 BILLING CODE 4164-01-P