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

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Food and Drug Administration, HHS.


Notice of 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).


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.


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:​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).

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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,

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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 (​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:​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: 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 (​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 (​events/​leveraging-randomized-clinical-trials-and-real-world-data-generate-real-world-evidence).

Transcripts: Transcripts of the public workshop will not be available.

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Dated: June 4, 2019.

Lowell J. Schiller,

Principal Associate Commissioner for Policy.

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[FR Doc. 2019-12113 Filed 6-7-19; 8:45 am]