FDA-AACR Real-world Evidence Workshop

Date: 8 a.m. – 5 p.m. July 19, 2019

Location: Doubletree by Hilton Bethesda, 8120 Wisconsin Ave, Bethesda, MD 20814

Workshop Material

Recorded Webcast (session and individual presentation YouTube links below):

• Session I: Introduction to Real-world Evidence: This session introduced real-world evidence concepts and the utility of using real-world data sources.
  • Jacqueline Corrigan-Curay, JD, MD
  • Elad Sharon, MD, MPH
• Session II: Premarket Use Cases: This session provided examples of using real-world evidence in drug development.
  • Michael A. Kelsh, PhD, MPH
  • William Capra, PhD
  • Weili He, PhD
  • Panel discussion
• Session III: Postmarket Use Cases: This session provided examples of using real-world evidence in postmarket situations.
  • Albert L. Kraus, PhD
  • Ruthanna Davi, PhD
  • Jeff Allen, PhD
  • Panel discussion
• Session IV: Large Genomic Databases & Real-world Evidence: This session explored large genomic databases and digital data as real-world sources of information.
  • Wendy Rubinstein, MD, PhD
  • Robert Grossman, PhD
  • William Dalton, PhD, MD
  • Neal J. Meropol, MD
  • Gary Palmer, MD, JD, MBA, MPH
  • Jonathan Hirsch
  • Deborah Schrag, MD, MPH
  • Panel discussion
• Session V: Real-world Evidence – Future Directions: This session explored potential opportunities and challenges as real-world evidence develops.
  • Andrea Coravos
  • James Gulley, MD, PhD
  • Mark Shapiro, MBA, PhD
  • Panel Discussion

*Some speakers elected to withhold their slides, but all presentations are represented in the webcast recording.

Background

Real-world evidence (RWE) is clinical evidence generated from datasets including electronic health records, administrative claims data, patient-reported data, and genomics/biomarker data when collected in the routine provision of care. RWE has been lauded for its broad potential applications to improve clinical care, accelerate research, and support regulatory decision making. Stemming from policy mandates established by the 21st Century Cures Act and PDUFA VI, there has been considerable progress in using RWE to support research efforts, ranging from retrospective cohort studies to prospective randomized trials in real-world settings. Regulatory-grade RWE use cases span the drug development lifecycle, including early clinical development, clinical trials, and post-marketing and commercialization. To fulfill the mandate to further advance RWE, the U.S. Food and Drug Administration recently released the Framework for FDA’s Real-World Evidence Program. This workshop will discuss the agency’s work thus far on the implementation of that framework while exploring the current state of RWE in oncology. Speakers at the workshop will examine pre- and post- market use cases in which RWE was utilized to improve and accelerate treatment development and patient care and explore the potential of large genomic databases as real-world data sources. This event will also look to the future of RWE and consider how ongoing efforts will impact the oncology drug development landscape.

Workshop Cochairs:

Sean Khozin, MD, MPH, Associate Director, Oncology Center of Excellence, Director, Information Exchange and Data Transformation (INFORMED), U.S. Food and Drug Administration

Pallavi Mishra-Kalyani, PhD, Team Leader, Division of Biometrics V, Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Deborah Schrag, MD, MPH, Chief, Division of Population Sciences, Dana-Farber Cancer Institute