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个体层面的随机试验。

Randomised trials at the level of the individual.

机构信息

Department of Experimental Medicine, University of British Columbia, Vancouver, BC, Canada.

Centre for Infectious Disease Epidemiology and Research, School of Public Health and Family Medicine, University of Cape Town, Cape Town, South Africa.

出版信息

Lancet Glob Health. 2021 May;9(5):e691-e700. doi: 10.1016/S2214-109X(20)30540-4.

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.

摘要

在全球健康研究中,最常见的研究设计是短期、小规模的临床试验,采用固定的、双臂试验设计,通常不允许在整个试验过程中进行重大更改。本文是本系列介绍性论文的续篇,讨论了几种适应性试验设计中基于数据驱动的方法,以及有助于协调全球健康研究中临床试验研究工作的主方案框架。我们为更有效的试验研究提供了一个通用框架,并通过统计模拟讨论了在规划阶段考虑不同研究设计的重要性。本文最后讨论了适应性试验设计和主方案的方法学和操作复杂性,以及当前可能限制这些方法在全球健康研究中应用的资金挑战。

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