A framework: make it useful to guide and improve practice of clinical trial design in smaller populations

Abstract

The increased attention to design and analysis of randomised clinical trials in small populations has triggered thinking regarding the most appropriate design methods for a particular clinical research question. Decision schemes and algorithms have been proposed, with varying starting points and foci. Parmar et al. (BMC Medicine 14:183, 2016) proposed a framework designed to assist the clinical trial team in design choices during protocol preparation. Herein, further stimulus is given regarding the extent to which a framework may help change practice for the better, the careful considerations for changing the usual error levels applied and the room for innovation in clinical trial design. Please see related article: http://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-016-0722-3 .