Informing single-arm clinical trials with external controls

Nature Reviews Drug Discovery
Volume 19 Issue 12, December 2020


Comment | 18 August 2020
Informing single-arm clinical trials with external controls
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.
Ruthie Davi , Nirosha Mahendraratnam […]  & Rachel Sherman