Hybrid Controlled Trials with EHR-Derived Data for Cancer Trials: What, Why, When, and How

• Activity Number: 164 - Leveraging Real-World Data in the Drug Development Process
• Type: Topic Contributed
• Date/Time: Tuesday, August 4, 2020 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Brian Segal* and Melissa Curtis and Meghna Samant and Katherine Tan and Shrujal Baxi and Somnath Sarkar
• Companies: Flatiron Health and Flatiron Health and Flatiron Health and Flatiron Health and Flatiron Health and Flatiron, Inc.
• Keywords: Electronic health records; Real-world evidence

Abstract

A well designed and executed randomized controlled trial (RCT) provides the most reliable evidence for assessing treatment efficacy and safety. However, there are situations in which a well designed and executed RCT might not be feasible (rare diseases) or ethical (no effective standard therapy). In these situations, there is potential to use external data to improve the efficiency and feasibility of trials while maintaining some randomization. Hybrid controlled trials accomplish this by augmenting the control arm of an RCT with external data. In this way, hybrid controlled trials are a middle ground between full RCTs and single-arm trials with external comparators and have some qualities of each design. This talk will give an overview of hybrid controlled trials using electronic health record (EHR)-derived external data for cancer trials, focusing on what they are, why they can be beneficial to patients and sponsors, when they are a potentially viable study design (and when they are not), and how to select control patients from EHRs and use borrowing methods to incorporate them into the trial design and analysis.