Placebo Response as a Latent Characteristic: Application to Analysis of Sequential Parallel Comparison Design Studies

2018
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.
JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION
页码:1411-1430|卷号:113|期号:524
ISSN:0162-1459
收录类型
SSCI
发表日期
2018
学科领域
循证社会科学-方法
国家
美国
语种
英语
DOI
10.1080/01621459.2017.1375930
其他关键词
CLINICAL-TRIALS; MAXIMUM-LIKELIHOOD; ENRICHMENT; DISORDERS; TIME
EISSN
1537-274X
被引频次(WOS)
4
被引更新日期
2022-01
来源机构
Boston University Harvard University Harvard Medical School Massachusetts General Hospital
关键词
Clinical trials EM algorithm Latent variable Placebo effect SPCD