82 FR 35534 - Developing a Framework for Regulatory Use of Real-World Evidence; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 82, Issue 145 (July 31, 2017)

Page Range		35534-35535
FR Document		2017-16021

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.

Federal Register, Volume 82 Issue 145 (Monday, July 31, 2017)

[Federal Register Volume 82, Number 145 (Monday, July 31, 2017)]
[Notices]
[Pages 35534-35535]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2017-16021]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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

Federal Register / Vol. 82, No. 145 / Monday, July 31, 2017 / Notices 35535

the development of RWE and its Meeting Materials: All event materials several health data policy topics and
applicability within specific regulatory will be provided to registered attendees begin to formulate its work plan for
decision-making contexts. This will via email prior to the workshop and 2018. To inform the work plan, the
include, but not be limited to, publicly available at the Duke-Margolis Committee will be briefed by the
challenges related to RWD collection Web site (https://healthpolicy.duke.edu/ Commission on Evidence-based
and quality, innovative methods for events/public-workshop-developing- Policymaking (CEP) regarding the
developing RWE from RWD, and framework-regulatory-use-real-world- release of its report and
promising areas for RWE pilot evidence). recommendations as well as hear from
demonstrations. Transcripts: Please be advised that HHS leadership regarding data needs
III. Participating in the Public transcripts will not be available. and gaps. A panel will be held to
Workshop Dated: July 25, 2017. discuss the new topic ‘‘Beyond HIPAA,’’
Registration: To register for the public Leslie Kux, an exploration of challenges that extend
workshop, please visit the following Associate Commissioner for Policy. beyond HIPAA and the range of policy
Web site before September 12, 2017: [FR Doc. 2017–16021 Filed 7–28–17; 8:45 am] options that may be available to the
https://healthpolicy.duke.edu/events/ BILLING CODE 4164–01–P Department related to privacy, security
public-workshop-developing- and access measures to protect
framework-regulatory-use-real-world- individually identifiable health
evidence. There will be no onsite DEPARTMENT OF HEALTH AND information in an environment of
registration. Please provide complete HUMAN SERVICES electronic networking and multiple uses
contact information for each attendee, of data. Additional discussions are
including name, title, affiliation, National Committee on Vital and Health
planned on the Predictability Roadmap
address, email, and telephone. Statistics: Meeting
project in follow up to a Standards
Registration is free and based on Pursuant to the Federal Advisory Subcommittee workshop focused on
space availability, with priority given to Committee Act, the Department of possible approaches to improve the
early registrants. Early registration is Health and Human Services (HHS) predictability and improvements in the
recommended because seating is announces the following advisory adoption and processes related to
limited; therefore, FDA may limit the committee meeting.
number of participants from each updating standards and operating rules
Name: National Committee on Vital for electronic administrative
organization. A 1-hour lunch break is and Health Statistics (NCVHS), Full
scheduled, but food will not be transactions (e.g. claims, eligibility,
Committee and Population Health electronic funds transfer); and on
provided. There are multiple restaurants Subcommittee Meetings.
within walking distance of the terminology & vocabulary development,
Dates and Times: maintenance, and dissemination
Conference Center.
If you need special accommodations Monday, September 11, 2017: 9:00 a.m.– processes. The Committee also plans to
due to a disability, please contact 5:45 p.m. finalize the update to its strategic plan
Joanna Higgison at the Duke-Margolis Tuesday, September 12, 2017: 8:30 and selection criteria for undertaking
Center for Health Policy, 908–432–4872, a.m.–5:00 p.m. new Committee projects. The times and
joanna.higgison@duke.edu, no later Wednesday, September 13, 2017: 8:45 topics are subject to change. Please refer
than September 6, 2017. a.m.–5:30 p.m. to the posted agenda for any updates.
Streaming Webcast of the Public Thursday, September 14, 2017: 8:30
a.m.–3:15 p.m. Contact person for more information:
Workshop: This public workshop will
Place: U.S. Department of Health and Substantive program information may
also be webcast and archived video
Human Services, Hubert H. Humphrey be obtained from Rebecca Hines, MHS,
footage will be available at the Duke-
Margolis Web site (https:// Building, 200 Independence Avenue Executive Secretary, NCVHS, National
healthpolicy.duke.edu/events/public- SW., Room 705A, Washington, DC Center for Health Statistics, Centers for
workshop-developing-framework- 20201. Disease Control and Prevention, 3311
regulatory-use-real-world-evidence) Status: Open. Toledo Road, Hyattsville, Maryland
following the workshop. Persons Purpose: At the September 11–12, 20782, telephone (301) 458–4715.
interested in viewing the live webcast 2017 hearing, the Population Health Summaries of meetings and a roster of
must register online by September 12, Subcommittee will focus on Next Committee members are available on the
2017, at 5 p.m. Eastern Time (see Generation Vital Statistics. The purpose home page of the NCVHS Web site:
Registration). Early registration is of the hearing is to assess the current http://www.ncvhs.hhs.gov/, where
recommended because webcast state of the national vital statistics further information including an agenda
connections are limited. Organizations system (NVSS) to address concerns and instructions to access the audio
are requested to register all participants, regarding sustainability and viability of broadcast of the meetings will also be
but to view using one connection per the system infrastructure. The focus will posted. Should you require reasonable
location whenever possible. Webcast be on the system’s capacity to provide accommodation, please contact the CDC
participants will be sent technical timely, high quality, secure vital Office of Equal Employment
system requirements in advance of the administrative and statistical data for Opportunity on (770) 488–3210 as soon
event. Prior to joining the streaming identity establishment and protection, as possible.
sradovich on DSKBCFCHB2PROD with NOTICES

webcast of the public workshop, it is identification of trends in disease and
Dated: July 25, 2017.
recommended that you review these epidemics, e.g., the recent surge in
technical system requirements. opioid-related deaths, and a host of Laina Bush,
FDA has verified the Web site critical uses for research, finance, Deputy Assistant Secretary for Planning and
addresses in this document, as of the planning, public records and services. Evaluation, Office of the Assistant Secretary
date this document publishes in the At the September 13–14, 2017 full for Planning and Evaluation.
Federal Register, but Web sites are meeting, the Committee will hear [FR Doc. 2017–16036 Filed 7–28–17; 8:45 am]
subject to change over time. presentations, hold discussions on BILLING CODE 4151–05–P

VerDate Sep<11>2014 17:38 Jul 28, 2017 Jkt 241001 PO 00000 Frm 00029 Fmt 4703 Sfmt 9990 E:\FR\FM\31JYN1.SGM 31JYN1

Document Created: 2017-07-29 00:21:35
Document Modified: 2017-07-29 00:21:35

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 September 13, 2017, from 9 a.m. to 4:30 p.m., Eastern Time.
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]
FR Citation		82 FR 35534

82 FR 35534 - Developing a Framework for Regulatory Use of Real-World Evidence; Public Workshop

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 82, Issue 145 (July 31, 2017)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration