相关论文: Two-stage matching-adjusted indirect comparison

In matched observational studies with continuous treatments, individuals with different treatment doses but the same or similar covariate values are paired for causal inference. While inexact covariate matching (i.e., covariate imbalance…

Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Covariate adjustment is widely recommended to improve statistical efficiency in randomized clinical trials (RCTs), yet empirical evidence comparing available strategies remains limited. This lack of real-world evaluation leaves unresolved…

Adaptive experiments such as multi-armed bandits offer efficiency gains over traditional randomized experiments but pose two major challenges: invalid inference on the Average Treatment Effect (ATE) due to adaptive sampling and low…

Clinical trials with a hybrid control arm (a control arm constructed from a combination of randomized patients and real-world data on patients receiving usual care in standard clinical practice) have the potential to decrease the cost of…

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…

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

Matching on covariates is a well-established framework for estimating causal effects in observational studies. The principal challenge stems from the often high-dimensional structure of the problem. Many methods have been introduced to…

When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

In many scientific fields such as biology, psychology and sociology, there is an increasing interest in estimating the causal effect of a matrix exposure on an outcome. Covariate balancing is crucial in causal inference and both exact…

An efficient computational approach for imaging binary-type physical properties suitable for various models in biomedical applications is developed and validated. The proposed methodology includes gradient-based multiscale optimization with…

In randomized clinical trials, adjustments for baseline covariates at both design and analysis stages are highly encouraged by regulatory agencies. A recent trend is to use a model-assisted approach for covariate adjustment to gain…

This paper studies inference on the average treatment effect in experiments in which treatment status is determined according to "matched pairs" and it is additionally desired to adjust for observed, baseline covariates to gain further…

Purpose: Two-stage single-arm trial designs are commonly used in phase II oncology to infer treatment effects for a binary primary outcome (e.g., tumour response). It is imperative that such studies be designed, analysed, and reported…

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose (MTD), followed by a randomization stage where patients are assigned to two or more doses to…

Two-phase sampling designs are frequently employed in epidemiological studies and large-scale health surveys. In such designs, certain variables are exclusively collected within a second-phase random subsample of the initial first-phase…

The statistical efficiency of randomized clinical trials can be improved by incorporating information from baseline covariates (i.e., pre-treatment patient characteristics). This can be done in the design stage using stratified (permutated…

Randomized controlled trials (RCTs) in oncology often allow control group participants to crossover to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment…