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Activity Number: 155 - Research Reproducibility for Precision Medicine: From Controlled Experiments to Real-World Evidence
Type: Topic Contributed
Date/Time: Monday, July 29, 2019 : 10:30 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract #305249 Presentation
Title: On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Gene Therapy Trials
Author(s): Qing Liu*
Companies: Quantitative and Regulatory Medical Science, LLC
Keywords: Real World Evidence; Gene Therapy; Rare Disease; Bayesian Analysis; Power; Type 1 Error Rates

For gene therapy trials, we consider the setting where clinical development program consists of a prospective observation study real-world evidence (RWE) of patients receiving a standard of care (SOC), and a phase 3 randomized and blinded study to establish effectiveness and safety of a new drug compared with an integrated RWE of SOC. We propose an efficient inter-group (EIG) analysis approach where RWE from both the perspective observational study and the concurrent control of the parallel group design are combined via a Bayesian approach. The resulting design is 50%-65% more efficient than a traditional parallel group design. The analysis procedure controls the type 1 error rates and maintains high statistical power. This makes it feasible to conduct trials where it is difficult to enroll patients.

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

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