Using Real-World Data in Single-Arm Clinical Trials: When It's Done, Why It's Done, How It's Done

Single-arm clinical trials—in which all enrolled patients receive active treatment and no one receives placebo control—have long been acceptable to regulatory authorities in those instances in which inclusion of a control arm is either infeasible or unethical. These situations arise particularly in rare diseases and oncology. In the wake of the 21st Century Cures Act, FDA has shown increased amenability to use of real-world data sources to identify patients for synthetic control arms in single-arm clinical trials.

This free webinar will describe the fundamentals of synthetic control arms with a focus on oncology trials, highlighting when they are done, why they are done, and how they are done. The featured speakers will frame the issues and articulate the common approaches to synthetic control arms in a manner that balances strategic aspects of research design with methodologic rigor on operationalizing the approach.

The session will end with an open question and answer discussion.

David Thompson, PhD, Senior Vice President, Real World Research, Syneos Health

David Thompson, PhD, is a health economist who has been working in the field of real-world research since 1988. During the course of his career, he has developed expertise in a wide variety of methodologic approaches, including economic modeling, retrospective database analysis, prospective real-world research, and patient-reported outcomes.  His work has been global in nature, with extensive experience in North American & European markets as well as the emerging markets of the Asia-Pacific and Latin American regions. 

Dave is Senior Vice President, Real-World Research, at Syneos Health, a position he has held since 2016. Prior to joining Syneos Health, he held leadership positions at Quintiles (2012-2016), i3 Innovus/OptumInsight (2000-2012) and PAI (1988-2000).

Dave is a long-time contributor to ISPOR, having led the launch and functioned as Editor-in-Chief of Value & Outcomes Spotlight (and, before it, ISPOR Connections) from 2008 to 2020. He is a current member of the ISPOR Board of Directors.

Among various current external affiliations, Dave is a steering committee member for the Clinical Trials Transformation Initiative (CTTI), a multistakeholder initiative of Duke University and the US Food & Drug Administration; a team leader on the CTTI initiatives on real-world evidence (RWE) and pragmatic clinical trials; and an advisory group member for the Duke-Margolis Real-World Evidence Collaborative.  

Craig White, PhD, Senior Vice President and General Manager, Outcomes Science, ConcertAI

Craig White, PhD, is a health policy researcher with approximately 20 years’ experience in healthcare data and analytics. Craig’s research is broad and has covered Value-based pharmaceutical pricing methodologies, innovative uses of RWD in clinical development and traditional health economic models and cost-effectiveness analyses. Dr White’s research has included evaluation of Thyroid and Prostate Cancer treatment during his fellowship at Massachusetts General Hospital in the Program for Cancer Outcomes Research, and methodological work as part of the NSF/NIH funded Bioinformatics Research Training Program at Harvard Medical School. He has helped develop commercialize and analyze various healthcare datasets since 2004.

Craig currently works as Senior Vice President and General Manager of ConcertAI’s Outcomes Science group, a role he has occupied since 2019. Previously, Dr. White held leadership roles at Precision Medicine Group, where he was Vice President and Managing Director of the Health Economics and Outcomes Research group. Prior to that, he spent nearly 10 years in various roles at IQVIA culminating in his leadership role on the Enterprise Solutions Team for Real World Insights where he led marquee initiatives in digital health and value-based pricing.

Dr. White received his PhD in Health Policy from Harvard University and BE in Electrical and Electronic Engineering from the University of Auckland.