@article{664b893f10f34e39973149d83f94cc36,
title = "Randomised trials at the level of the individual",
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.",
author = "Park, {Jay J.H.} and Nathan Ford and Denis Xavier and Per Ashorn and Grais, {Rebecca F.} and Bhutta, {Zulfiqar A.} and Herman Goossens and Kristian Thorlund and Socias, {Maria Eugenia} and Mills, {Edward J.}",
note = "Funding Information: In global health research, clinical trial funding pre-dominantly relies on grants from government funding agencies and non-profit organisations, and the demand for funding by far exceeds supply. The funding value from the US National Institutes of Health, for example, is usually fixed for all applications that might vary in scope. In practice, clinical trial sample size and trial duration are limited due to budget constraints. Optimistic assumptions about the treatment effects and baseline event rates are picked to justify the sample size that is suitable for the budget, to stay competitive during the application stage. For illustrative purposes, sample size and cost-savings are shown in figure 2 . Master protocols and adaptive clinical trials might require more resources than the budget that is typically allowed by the funder. Publisher Copyright: {\textcopyright} 2021 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license",
year = "2021",
month = may,
doi = "10.1016/S2214-109X(20)30540-4",
language = "English",
volume = "9",
pages = "e691--e700",
journal = "The Lancet Global Health",
issn = "2214-109X",
publisher = "Elsevier BV",
number = "5",
}