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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
Abstract #313351
Title: Hybrid Controlled Trials with EHR-Derived Data for Cancer Trials: What, Why, When, and How
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

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.

Authors who are presenting talks have a * after their name.

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