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Machine learning (ML) models are increasingly used as decision-support tools in high-risk domains. Evaluating the causal impact of deploying such models can be done with a randomized controlled trial (RCT) that randomizes users to ML vs.…
Multivariate linear mixed models (mvLMMs) have been widely used in many areas of genetics, and have attracted considerable recent interest in genome-wide association studies (GWASs). However, fitting mvLMMs is computationally non-trivial,…
The generative large language models (LLMs) are increasingly used for data augmentation tasks, where text samples are paraphrased (or generated anew) and then used for classifier fine-tuning. Existing works on augmentation leverage the…
We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling…
Linear mixed models (LMMs) are used as an important tool in the data analysis of repeated measures and longitudinal studies. The most common form of LMMs utilize a normal distribution to model the random effects. Such assumptions can often…
Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…
Randomized experiments are the gold standard for causal inference, and justify simple comparisons across treatment groups. Regression adjustment provides a convenient way to incorporate covariate information for additional efficiency. This…
Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…
Adjustment for ``super'' or ``prognostic'' composite covariates has become more popular in randomized trials recently. These prognostic covariates are often constructed from historical data by fitting a predictive model of the outcome on…
Many statistical estimands can expressed as continuous linear functionals of a conditional expectation function. This includes the average treatment effect under unconfoundedness and generalizations for continuous-valued and personalized…
In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…
Large language models (LLMs) have become increasingly capable of following instructions and complex reasoning, making prompting a flexible interface for adapting models without parameter updates. Yet prompt design remains labor-intensive…
Large Language Models (LLMs) have demonstrated promise in medical knowledge assessments, yet their practical utility in real-world clinical decision-making remains underexplored. In this study, we evaluated the performance of three…
What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…
While Large Language Models (LLMs) are being quickly adapted to many domains, including healthcare, their strengths and pitfalls remain under-explored. In our study, we examine the effects of prompt engineering to guide Large Language…
Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…
We develop a method for hybrid analyses that uses external controls to augment internal control arms in randomized controlled trials (RCT) where the degree of borrowing is determined based on similarity between RCT and external control…
In randomized trials, appropriately adjusting for baseline variables and short-term outcomes can lead to increased precision and reduced sample size. We examine the impact of such adjustment in group sequential designs, i.e., designs with…
Generalized linear models (GLMs) -- such as logistic regression, Poisson regression, and robust regression -- provide interpretable models for diverse data types. Probabilistic approaches, particularly Bayesian ones, allow coherent…
The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…