Placebo Response as a Latent Characteristic: Application to Analysis of Sequential Parallel Comparison Design Studies
提出将安慰剂反应视为潜在特征的新方法，通过似然估计校正序贯平行比较设计中的治疗效果，帮助更准确推断药物疗效。
In clinical trials, placebo response can affect the inference about efficacy of the studied treatment. It is important to have a robust way to classify trial subjects with respect to their response to placebo. Simple, criterion-based classification may lead to classification error and bias the inference. The uncertainty about placebo response characteristic has to be factored into the treatment effect estimation. We propose a novel approach that views the placebo response as a latent characteristic and the study sample as an unlabeled mixture of “placebo responders” and “placebo nonresponders.” The likelihood-based methodology is used to estimate the treatment effect corrected for placebo response as defined within sequential parallel comparison design.