YSPH Biostatistics Seminar: “The Design of Hybrid Controlled Trials that Leverage External Data and Randomization"

NOTE: BIS 525 students are required to attend in person. Others are invited to attend in person, but may also attend via Zoom.

SPEAKER: Lorenzo Trippa, PhD, Associate Professor, Department of Biostatistics, Harvard T.H. Chan School
of Public Health

TITLE: “The Design of Hybrid Controlled Trials that Leverage External Data and Randomization"

ABSTRACT:  Patient-level data from completed clinical studies or electronic health records can be used in the design and analysis of clinical trials. However, these external data can bias the evaluation of the experimental treatment when the statistical design does not appropriately account for potential confounders. In this work, we introduce a hybrid clinical trial design that combines the use of external control datasets and randomization to experimental and control arms, with the aim of producing efficient inference on the experimental treatment effects. Our analysis of the hybrid trial design includes scenarios where the distributions of measured and unmeasured prognostic patient characteristics differ across studies. Using simulations and datasets from clinical studies in extensive-stage small cell lung cancer and glioblastoma, we illustrate the potential advantages of hybrid trial designs compared to externally controlled trials and randomized trial designs.