84 FR 26882 - Leveraging Randomized Clinical Trials To Generate Real-World Evidence for Regulatory Purposes; Public Workshop

The Food and Drug Administration (FDA, the Agency, or we) is announcing the following public workshop entitled ``Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes.'' Convened by Duke University's Robert J. Margolis, MD, Center for Health Policy (Duke Margolis) and supported by a cooperative agreement with FDA, the purpose of the public workshop is to bring the stakeholder community together to explore key considerations for using randomized designs, such as large simple trials or those that incorporate pragmatic elements to generate real- world evidence (RWE).

[Federal Register Volume 84, Number 111 (Monday, June 10, 2019)]
[Notices]
[Pages 26882-26883]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2019-12113]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-N-0001]


Leveraging Randomized Clinical Trials To Generate Real-World 
Evidence for Regulatory Purposes; 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 ``Leveraging 
Randomized Clinical Trials to Generate Real-World Evidence for 
Regulatory Purposes.'' Convened by Duke University's Robert J. 
Margolis, MD, Center for Health Policy (Duke Margolis) and supported by 
a cooperative agreement with FDA, the purpose of the public workshop is 
to bring the stakeholder community together to explore key 
considerations for using randomized designs, such as large simple 
trials or those that incorporate pragmatic elements to generate real-
world evidence (RWE).

DATES: The public workshop will be held on July 11, 2019, from 8:30 
a.m. to 5 p.m., Eastern Time and July 12, 2019, from 9 a.m. to 1 p.m., 
Eastern Time. See the SUPPLEMENTARY INFORMATION section for 
registration date and information.

ADDRESSES: The public workshop will be held at The Westin City Center, 
1400 M St. NW, Washington, DC 20005. For additional travel and hotel 
information, please refer to the following website: https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-and-real-world-data-generate-real-world-evidence. There will also be a live 
webcast for those unable to attend the meeting in person (see Streaming 
Webcast of the Public Workshop).

FOR FURTHER INFORMATION CONTACT: Dianne Paraoan, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 3326, Silver Spring, MD 20993, 301-796-
2500, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    Section 3022 of the 21st Century Cures Act (Cures Act) amended the 
Federal Food, Drug, and Cosmetic Act (FD&C Act) to add section 505F, 
Utilizing real world evidence (21 U.S.C. 355g). This section requires 
the establishment of a program to evaluate the potential use of RWE to 
help support the approval of a new indication for a drug approved under 
section 505(c) of the FD&C Act (21 U.S.C. 355(c)) and to help to 
support or satisfy postapproval study requirements. In December 2018, 
FDA published the Framework for the RWE program (https://www.fda.gov/media/120060/download). To inform FDA's RWE Framework, on September 13, 
2017, through its cooperative agreement with Duke Margolis, FDA 
convened a public meeting that explored the use of RWE for regulatory 
decisions.
    The RWE Framework includes information describing sources of RWE, 
gaps in data collection activities, standards and methodologies for 
collecting and analyzing RWE, and priority areas, remaining challenges, 
and potential pilot opportunities to address the overarching Cures Act 
requirements. The RWE Framework also discusses the integration of 
clinical trials into clinical care settings and FDA's intent to issue 
guidance on this subject. The public workshop announced in this notice 
is a part of FDA's ongoing efforts to implement the RWE Framework by 
exploring the utility of RWE for regulatory decision making. This 
workshop will focus on how randomized clinical trial designs can use 
real-world data (RWD) to generate RWE, particularly in clinical care 
settings.

II. Topics for Discussion at the Public Workshop

    This workshop will explore key considerations for using randomized 
clinical trial designs and RWD to generate RWE, particularly in 
clinical care settings. Considerations for discussion include: (1) 
Selection of interventions appropriate in clinical care settings, (2) 
study design elements and study populations, (3) capturing outcomes in 
clinical care settings, and (4) addressing potential challenges around 
blinding, randomization, and bias. The workshop will also explore 
regulatory considerations for randomized clinical trials using RWD, 
such as safety and product monitoring and maintaining data integrity.

III. Participating in the Public Workshop

    Registration: To register for the public workshop, please visit the 
following website: https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-and-real-world-data-generate-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. Persons interested in attending this public 
workshop must register by July 10, 2019, 11:59 p.m. Eastern Time. Early 
registration is recommended because seating is limited; therefore, FDA 
may limit the number of participants from each organization. 
Registrants will receive confirmation when they have been registered. 
We will let registrants know if registration closes before the day of 
the public workshop.
    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 July 
5, 2019.
    Streaming Webcast of the Public Workshop: This public workshop will 
also be webcast, and archived video footage will be available at the 
Duke-Margolis website (https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-and-real-world-data-generate-real-world-evidence) following the workshop. Persons interested in viewing the 
live webcast are encouraged to register in advance (see Registration). 
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 website addresses in this document, 
as of the date this document publishes in the Federal Register, but 
websites 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 website (https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-and-real-world-data-generate-real-world-evidence).
    Transcripts: Transcripts of the public workshop will not be 
available.


[[Page 26883]]


    Dated: June 4, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-12113 Filed 6-7-19; 8:45 am]
BILLING CODE 4164-01-P