Related papers: A General Framework for Designing and Evaluating A…

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

Externally controlled trials are crucial in clinical development when randomized controlled trials are unethical or impractical. These trials consist of a full treatment arm with the experimental treatment and a full external control arm.…

The appearance of a new dangerous and contagious disease requires the development of a drug therapy faster than what is foreseen by usual mechanisms. Many drug therapy developments consist in investigating through different clinical trials…

A growing body of work uses the paradigm of algorithmic fairness to frame the development of techniques to anticipate and proactively mitigate the introduction or exacerbation of health inequities that may follow from the use of…

Conditional power calculations are frequently used to guide the decision whether or not to stop a trial for futility or to modify planned sample size. These ignore the information in short-term endpoints and baseline covariates, and thereby…

Most work in causal inference considers deterministic interventions that set each unit's treatment to some fixed value. However, under positivity violations these interventions can lead to non-identification, inefficiency, and effects with…

This article develops a framework for testing general hypothesis in high-dimensional models where the number of variables may far exceed the number of observations. Existing literature has considered less than a handful of hypotheses, such…

With more and better clinical data being captured outside of clinical studies and greater data sharing of clinical studies, external controls may become a more attractive alternative to randomized clinical trials. Both industry and…

Network meta-analysis (NMA) combines evidence from multiple trials to compare the effectiveness of a set of interventions. In public health research, interventions are often complex, made up of multiple components or features. This makes it…

Unmeasured confounding is a threat to causal inference in observational studies. In recent years, use of negative controls to mitigate unmeasured confounding has gained increasing recognition and popularity. Negative controls have a…

This paper presents a novel approach to active learning that takes into account the non-independent and identically distributed (non-i.i.d.) structure of a clinical trial setting. There exists two types of clinical trials: retrospective and…

Recovering and distinguishing between the strict-preference, indifference and/or indecisiveness parts of a decision maker's preferences is a challenging task but also important for testing theory and conducting welfare analysis. This paper…

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

We develop our previous works concerning the identification of the collection of significant factors determining some, in general, non-binary random response variable. Such identification is important, e.g., in biological and medical…

Attrition in survey and field experiments presents a challenge for social science research. Common approaches to deal with this problem -- such as complete case analysis, multiple imputation, and weighting methods -- rely on strong…

In recent years new cancer treatments improved survival in multiple histologies. Some of these therapeutics, and in particular treatment combinations, are often associated with severe treatment-related adverse events (AEs). It is therefore…

Instrumental variables have been widely used to estimate the causal effect of a treatment on an outcome. Existing confidence intervals for causal effects based on instrumental variables assume that all of the putative instrumental variables…

We propose a general framework for interactively learning models, such as (binary or non-binary) classifiers, orderings/rankings of items, or clusterings of data points. Our framework is based on a generalization of Angluin's equivalence…

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

In early phase drug development of combination therapy, the primary objective is to preliminarily assess whether there is additive activity from a novel agent when combined with an established monotherapy. Due to potential feasibility…