Robust estimation of encouragement design intervention effects transported across sites

• 成果类型: Article
• 署名作者: Rudolph, Kara E.; van der Laan, Mark J.
• 署名单位: University of California System; University of California Berkeley; University of California System; University of California San Francisco
• 刊物名称: JOURNAL OF THE ROYAL STATISTICAL SOCIETY SERIES B-STATISTICAL METHODOLOGY
• ISSN/ISSBN: 1369-7412
• DOI: 10.1111/rssb.12226
• 发表日期: 2017
• 页码: 1509-1525
• 关键词: clinical-trials

摘要

We develop robust targeted maximum likelihood estimators (TMLEs) for transporting intervention effects from one population to another. Specifically, we develop TMLEs for three transported estimands: the intent-to-treat average treatment effect (ATE) and complier ATE, which are relevant for encouragement design interventions and instrumental variable analyses, and the ATE of the exposure on the outcome, which is applicable to any randomized or observational study. We demonstrate finite sample performance of these TMLEs by using simulation, including in the presence of practical violations of the positivity assumption. We then apply these methods to the Moving to opportunity' trial: a multisite, encouragement design intervention in which families in public housing were randomized to receive housing vouchers and logistical support to move to low poverty neighbourhoods. This application sheds light on whether effect differences across sites can be explained by differences in population composition.