Synthetic control arms and other uses of external data in clinical trials for hematological malignancies

Abstract

Clinical trialists have been debating the use of data external to the study for decades. In this review, we develop concepts for interested readers to consider the use of external data in clinical trials, and we provide perspectives on the real-world usage of external data in phase 2 and 3 clinical trials for hematological malignancies across US academic cancer clinical trial networks. Only a minority of these studies have included external data (beyond use in establishing outcome benchmarks) in recent years, but improvements in data sharing and advances in methodologies may lead to more sources of potentially high-quality data for the broader clinical trials community. Identifying appropriate external data specific to the clinical trial and developing plans to adequately address differences between study and external data will remain key considerations for including external data in clinical trials.

Keywords: Clinical trials; External data; Hematological malignancies; Historical controls; Synthetic control arms; cancer.