Related papers: Power prior models for treatment effect estimation…
Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…
Estimation and inference of treatment effects under unconfounded treatment assignments often suffer from bias and the `curse of dimensionality' due to the nonparametric estimation of nuisance parameters for high-dimensional confounders.…
Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…
When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…
Sequential multiple assignment randomized trials (SMARTs) are used to construct data-driven optimal intervention strategies for subjects based on their intervention and covariate histories in different branches of health and behavioral…
Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…
Accurate sample classification using transcriptomics data is crucial for advancing personalized medicine. Achieving this goal necessitates determining a suitable sample size that ensures adequate statistical power without undue resource…
Discrete random probability measures are central to Bayesian inference, particularly as priors for mixture modeling and clustering. A broad and unifying class is that of proper species sampling processes (SSPs), encompassing many Bayesian…
In many biological applications, the primary objective of study is to quantify the magnitude of treatment effect between two groups. Cohens'd or strictly standardized mean difference (SSMD) can be used to measure effect size however, it is…
The manuscript discusses how to incorporate random effects for quantile regression models for clustered data with focus on settings with many but small clusters. The paper has three contributions: (i) documenting that existing methods may…
An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…
We present a unified three-state model (TSM) framework for evaluating treatment effects in clinical trials in the presence of treatment crossover. Researchers have proposed diverse methodologies to estimate the treatment effect that would…
Determination of posterior probability for go-no-go decision and predictive power are becoming increasingly common for resource optimization in clinical investigation. There are vast published literature on these topics; however, the…
In this work we provide a couple of contributions to the analysis of longitudinal data collected by smartphones in mobile health applications. First, we propose a novel statistical approach to disentangle personalized treatment and…
Sum-product networks (SPNs) have recently emerged as a novel deep learning architecture enabling highly efficient probabilistic inference. Since their introduction, SPNs have been applied to a wide range of data modalities and extended to…
The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…
The restricted mean survival time (RMST) model has been garnering attention as a way to provide a clinically intuitive measure: the mean survival time. RMST models, which use methods based on pseudo time-to-event values and inverse…
Applied researchers are increasingly interested in whether and how treatment effects vary in randomized evaluations, especially variation not explained by observed covariates. We propose a model-free approach for testing for the presence of…
Bayesian Additive Regression Trees(BART) is a Bayesian nonparametric approach which has been shown to be competitive with the best modern predictive methods such as random forest and Gradient Boosting Decision Tree.The sum of trees…
Stepped wedge cluster randomized trials (SW-CRTs) have historically been analyzed using immediate treatment (IT) models, which assume the effect of the treatment is immediate after treatment initiation and subsequently remains constant over…