Related papers: Improving clinical trial interpretation with ACCEP…

We propose an adaptive sequential framework for testing two simple hypotheses that analytically ensures finite exposure to the less effective treatment. Our proposed procedure employs a likelihood ratio-driven adaptive allocation rule,…

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

Large language models (LLMs) show promise for supporting clinicians in diagnostic communication by generating explanations and guidance for patients. Yet their ability to produce outputs that are both understandable and empathetic remains…

Optimal treatment rules can improve health outcomes on average by assigning a treatment associated with the most desirable outcome to each individual. Due to an unknown data generation mechanism, it is appealing to use flexible models to…

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

When evaluating medical tests or biomarkers for disease classification, the area under the receiver-operating characteristic (ROC) curve is a widely used performance metric that does not require us to commit to a specific decision…

The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

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…

Contrastive explanations clarify why an event occurred in contrast to another. They are more inherently intuitive to humans to both produce and comprehend. We propose a methodology to produce contrastive explanations for classification…

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

The use of simulation-based sensitivity analyses is fundamental to evaluate and compare candidate designs for future clinical trials. In this context, sensitivity analyses are especially useful to assess the dependence of important design…

The schemes for fault-tolerant postselected quantum computation given in [Knill, Fault-Tolerant Postselected Quantum Computation: Schemes, http://arxiv.org/abs/quant-ph/0402171] are analyzed to determine their error-tolerance. The analysis…

Clinical risk prediction is a valuable tool for guiding healthcare interventions toward those most likely to benefit. Yet, evaluating the pairing of a risk prediction model with an intervention using randomized controlled trials presents…

Clinical dataset labels are rarely certain as annotators disagree and confidence is not uniform across cases. Typical aggregation procedures, such as majority voting, obscure this variability. In simple experiments on medical imaging…

Net benefit is widely used and reported to evaluate the clinical utility of prediction models, yet its interpretation often remains difficult in practice. In this didactical note, we develop two complementary interpretations that make net…

Hybrid controlled trials (HCTs), which augment randomized controlled trials (RCTs) with external controls (ECs), are increasingly receiving attention as a way to address limited power, slow accrual, and ethical concerns in clinical…

Matching and weighting methods for observational studies involve the choice of an estimand, the causal effect with reference to a specific target population. Commonly used estimands include the average treatment effect in the treated (ATT),…

How do we know if a particular medical treatment actually works? Ideally one would consult all available evidence from relevant clinical trials. Unfortunately, such results are primarily disseminated in natural language scientific articles,…

We present methods for causally interpretable meta-analyses that combine information from multiple randomized trials to estimate potential (counterfactual) outcome means and average treatment effects in a target population. We consider…

In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at…