Individual participant data (IPD) meta-analyses of randomised trials are considered a reliable way to assess participant-level treatment effect modifiers but may not make the best use of the available data. Traditionally, effect modifiers are explored one covariate at a time, which gives rise to the possibility that evidence of treatment-covariate…

We consider a class of multiple-group individually-randomized group trials (IRGTs) that introduces a (partially) cross-classified structure in the treatment condition (only). The novel feature of this design is that the nature of the treatment induces a clustering structure that involves two or more non-nested groups among individuals in the…

Purpose: Multisite mediation studies are a cornerstone in mapping out developmental processes because they probe the mechanisms of a treatment while creating key opportunities to learn from and about variation in those mechanisms across sites. Despite the prevalence of multisite studies, a significant gap in the literature is how to plan such…

Cluster-randomized trials have been widely used to evaluate the treatment effects of interventions on student outcomes. When interventions are implemented by teachers, researchers need to account for the nested structure in schools (i.e., students are nested within teachers nested within schools). Schools usually have a very limited number of…

Background: Power analyses for a cluster-randomized trial (CRT) require estimates of additional design parameters beyond those needed for an individually randomized trial. In a 2-level CRT, there are two sample sizes, the number of clusters and the number of individuals per cluster. The intraclass correlation (ICC), or the proportion of variance…

Random encouragement designs evaluate treatments that aim to increase participation in a program or activity. These randomized controlled trials (RCTs) can also assess the mediated effects of participation itself on longer term outcomes using a complier average causal effect (CACE) estimation framework. This article considers power analysis…

Multisite cluster randomized trials (MCRTs), in which, the intermediate-level clusters (e.g., classrooms) are randomly assigned to the treatment or control condition within each site (e.g., school), are among the most commonly used experimental designs across a broad range of disciplines. MCRTs often align with the theory that programs are…

Multisite trials that randomize individuals (e.g., students) within sites (e.g., schools) or clusters (e.g., teachers/classrooms) within sites (e.g., schools) are commonly used for program evaluation because they provide opportunities to learn about treatment effects as well as their heterogeneity across sites and subgroups (defined by moderating…

Background: Randomized controlled trials (RCTs), or experiments, are the gold standard for intervention evaluation. However, the main appeal of RCTs--the clean identification of causal effects--can be compromised by interference, when one subject's actions can influence another subject's behavior or outcomes. In this paper, we formalize and study…

Network meta-analysis (NMA) is an extension of pairwise meta-analysis (PMA) which combines evidence from trials on multiple treatments in connected networks. NMA delivers internally consistent estimates of relative treatment efficacy, needed for rational decision making. Over its first 20 years NMA's use has grown exponentially, with applications…

Combining observational and experimental data for causal inference can improve treatment effect estimation. However, many observational data sets cannot be released due to data privacy considerations, so one researcher may not have access to both experimental and observational data. Nonetheless, a small amount of risk of disclosing sensitive…

For randomized trials that use text as an outcome, traditional approaches for assessing treatment impact require that each document first be manually coded for constructs of interest by trained human raters. This process, the current standard, is both time-consuming and limiting: even the largest human coding efforts are typically constrained to…

Random encouragement designs are randomized controlled trials (RCTs) that test interventions aimed at increasing participation in a program or activity whose take up is not universal. In these RCTs, instead of randomizing individuals or clusters directly into treatment and control groups to participate in a program or activity, the randomization…

Optimal design of multisite randomized trials leverages sampling costs to optimize sampling ratios and ultimately identify more efficient and powerful designs. Past implementations of the optimal design framework have assumed that costs of sampling units are equal across treatment conditions. In this study, we developed a more flexible optimal…

The purpose of this dissertation is to develop principles and strategies for and identify limitations of multisite cluster randomized trials in the context of partially and fully nested designs. In the first study, I develop principles of estimation, sampling variability, and inference for studies that leverage multisite designs within the context…