Informing single-arm clinical trials with external controls.

Abstract

Randomized controlled trials are the accepted standard for evaluating investigational therapies, but such trials are sometimes not an option for reasons of ethics or feasibility. Here, we discuss opportunities to address evidence gaps by using historical clinical trial data and real-world data in external control arms for single-arm trials, as well as the associated challenges. Randomized controlled trials are the accepted standard for evaluating investigational therapies, but such trials are sometimes not an option for reasons of ethics or feasibility. Here, we discuss opportunities to address evidence gaps by using historical clinical trial data and real-world data in external control arms for single-arm trials, as well as the associated challenges.