Causal Inference in a Placebo-Controlled Clinical Trial with Binary Outcome and Ordered Compliance
作者:
Els Goetghebeur,
Geert Molenberghs,
期刊:
Journal of the American Statistical Association
(Taylor Available online 1996)
卷期:
Volume 91,
issue 435
页码: 928-934
ISSN:0162-1459
年代: 1996
DOI:10.1080/01621459.1996.10476962
出版商: Taylor & Francis Group
关键词: Coarsening;Counterfactual framework;Efficacy;Intention to treat;Interval censoring;Ordinal data
数据来源: Taylor
摘要:
We propose a likelihood-based method to analyze the causal effect of partial compliance (i.e., unplanned partial exposure to treatment or placebo) in the LRC-CPPT data, a prevention trial with long term follow-up previously analyzed by Efron and Feldman. Initially, we construct ordered compliance categories and dichotomize response. Assuming increased exposure to cholestyramine does not increase cholesterol, we estimate exposure—response curves in different compliance subsets. Subjects in different arms with similar levels of compliance to the assignment may have a different placebo prognosis (i.e., success probability under a possible zero exposure level). The sole assumption that the placebo group reflects response to zero exposure for the treatment group as a whole allows estimation of a causal parameter in a special case only. When a single parameter represents the association between responses to possible treatment exposures and treatment compliance, simple estimates are derived for a set of causal parameters. The example is analyzed in detail, and more general applicability and extensions of the method are discussed.
点击下载:
PDF (1110KB)
返 回