Everyone Counts - Regulating the Evidence from Clinical Trial Data to Real-World Evidence for the Public Good: The Past, Present, and Future

• Activity Number: 440 - Let’s Make Everyone and Everything Count! Benefit-Risk Assessment Challenges, Lessons and Impacts in the Age of Big Data from Clinical Trials to Real-World Evidence
• Type: Topic Contributed
• Date/Time: Thursday, August 6, 2020 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Lisa LaVange*
• Companies: UNC-CH
• Keywords: Subtantial evidence; Real-world data; Complex Innovative Trial Designs

Abstract

Statisticians traditionally contribute to study design, conduct, analysis, and reporting. More recently, attention has turned to the need to understand the evidence provided by study results, whether that evidence is generated in support of a regulatory action about an intervention or a change in medical practice. Evaluating evidence involves weighing the benefit and risk provided, and both qualitative and quantitate approaches are available for this purpose. Initiatives undertaken by the US FDA beginning in 2016 focus on real-world evidence generation using real-world data in both the pre- and post-market settings. Patient advocacy groups and medical researchers alike view the rich data streams that are becoming increasingly available as too big to ignore in terms of providing insight into a therapy's safety and effectiveness. Similarly, the use of innovative or otherwise non-traditional trial designs to generate substantial evidence holds promise to stakeholders in terms of efficiently addressing important research questions. I will discuss the statistician's role in evaluating evidence in today's exciting but changing research landscape.