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Understanding the effects of interventions is central to scientific progress, with randomized controlled trials (RCTs) regarded as the gold standard for causal inference in many applied fields. However, RCTs are costly, time-consuming, and…
Proper citation is of great importance in academic writing for it enables knowledge accumulation and maintains academic integrity. However, citing properly is not an easy task. For published scientific entities, the ever-growing academic…
The statistical analysis of clinical trials is often complicated by missing data. Patients sometimes experience intercurrent events (ICEs), which usually (although not always) lead to missing subsequent outcome measurements for such…
Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…
Data mining and machine learning techniques such as classification and regression trees (CART) represent a promising alternative to conventional logistic regression for propensity score estimation. Whereas incomplete data preclude the…
Clinical decision support using data mining techniques offers more intelligent way to reduce the decision error in the last few years. However, clinical datasets often suffer from high missingness, which adversely impacts the quality of…
Results from Randomized Controlled Trials (RCTs) establish the comparative effectiveness of interventions, and are in turn critical inputs for evidence-based care. However, results from RCTs are presented in (often unstructured) natural…
Today, data analysts largely rely on intuition to determine whether missing or withheld rows of a dataset significantly affect their analyses. We propose a framework that can produce automatic contingency analysis, i.e., the range of values…
Randomized Controlled Trials (RCTs) represent the gold standard for causal inference yet remain a scarce resource. While large-scale observational data is often available, it is utilized only for retrospective fusion, and remains discarded…
As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…
Meta-analyses statistically aggregate the findings of different randomized controlled trials (RCTs) to assess treatment effectiveness. Because this yields robust estimates of treatment effectiveness, results from meta-analyses are…
Bayesian data analysis (BDA) is today used by a multitude of research disciplines. These disciplines use BDA as a way to embrace uncertainty by using multilevel models and making use of all available information at hand. In this chapter, we…
A core component of precision medicine research involves optimizing individualized treatment rules (ITRs) based on patient characteristics. Many studies used to estimate ITRs are longitudinal in nature, collecting outcomes over time. Yet,…
Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective…
There has been an increasing interest in using cell and gene therapy (CGT) to treat/cure difficult diseases. The hallmark of CGT trials are the small sample size and extremely high efficacy. Due to the innovation and novelty of such…
Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…
Data-driven decision making has been a common task in today's big data era, from simple choices such as finding a fast way to drive home, to complex decisions on medical treatment. It is often supported by visual analytics. For various…
Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…
Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and…
Causal effect estimation (CEE) provides a crucial tool for predicting the unobserved counterfactual outcome for an entity. As CEE relaxes the requirement for ``perfect'' counterfactual samples (e.g., patients with identical attributes and…