Randomised trials at the level of the individual
Jay Park, Nathan Ford, Denis Xavier, Per Ashorn, Rebecca F. Grais, Zulfiqar A Bhutta, Herman Goossens, Kristian Thorlund, M. Eugenia Socías, Edward J. Mills
Abstract
In global health research, short-term, small-scale clinical trials with fixed, two-arm trial designs that generally do not allow for major changes throughout the trial are the most common study design. Building on the introductory paper of this Series, this paper discusses data-driven approaches to clinical trial research across several adaptive trial designs, as well as the master protocol framework that can help to harmonise clinical trial research efforts in global health research. We provide a general framework for more efficient trial research, and we discuss the importance of considering different study designs in the planning stage with statistical simulations. We conclude this second Series paper by discussing the methodological and operational complexity of adaptive trial designs and master protocols and the current funding challenges that could limit uptake of these approaches in global health research.