Dunn David T, Glidden David V
MRC Clinical Trials Unit at UCL, London, UK.
Epidemiology & Biostatistics Department, University of California, San Francisco, CA, USA.
Stat Commun Infect Dis. 2019 Jan;11(1):20190006. doi: 10.1515/scid-2019-0006. Epub 2019 Jul 12.
The design and analysis of active-control trials to evaluate experimental HIV pre-exposure prophylaxis (PrEP) agents pose serious statistical challenges. We recently proposed a new outcome measure, the averted infections ratio (AIR) - the proportion of infections that would be averted by using the experimental agent rather than the control agent (compared to no intervention). The main aim of the current paper is to examine the mathematical connection between AIR and the HIV incidence rate ratio, the standard outcome measure. We also consider the sample size implications of the choice of primary outcome measure and explore the connection between effectiveness and efficacy under a simplified model of adherence.
评估实验性HIV暴露前预防（PrEP）药物的活性对照试验的设计和分析带来了严峻的统计学挑战。我们最近提出了一种新的结局指标，即避免感染率（AIR）——使用实验药物而非对照药物（与不进行干预相比）可避免的感染比例。本文的主要目的是研究AIR与HIV发病率比（标准结局指标）之间的数学联系。我们还考虑了主要结局指标选择对样本量的影响，并在一个简化的依从性模型下探讨有效性和效力之间的联系。
