相关论文: Testing Prioritized Composite Endpoint with Multip…
Phenotyping is the process of distinguishing groups of patients to identify different types of disease progression. A recent trend employs low-rank matrix and tensor factorization methods for their capability of dealing with multi-modal,…
Nested nonparametric processes are vectors of random probability measures widely used in the Bayesian literature to model the dependence across distinct, though related, groups of observations. These processes allow a two-level clustering,…
The incorporation of "real-world data" to supplement the analysis of trials and improve decision-making has spurred the development of statistical techniques to account for introduced confounding. Recently, "hybrid" methods have been…
Diffusion models have recently shown considerable potential in solving Bayesian inverse problems when used as priors. However, sampling from the resulting denoising posterior distributions remains a challenge as it involves intractable…
Randomized controlled trials are the gold standard for evaluating the efficacy of an intervention. However, there is often a trade-off between selecting the most scientifically relevant primary endpoint versus a less relevant, but more…
Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…
Major advances have been made regarding the utilization of artificial intelligence in health care. In particular, deep learning approaches have been successfully applied for automated and assisted disease diagnosis and prognosis based on…
In comparative studies of progressive diseases, such as randomized controlled trials (RCTs), the mean Change From Baseline (CFB) of a continuous outcome at a pre-specified follow-up time across subjects in the target population is a…
Prediction performance of a risk scoring system needs to be carefully assessed before its adoption in clinical practice. Clinical preventive care often uses risk scores to screen asymptomatic population. The primary clinical interest is to…
Mixed outcome endpoints that combine multiple continuous and discrete components to form co-primary, multiple primary or composite endpoints are often employed as primary outcome measures in clinical trials. There are many advantages to…
As alternatives to the time-to-first-event analysis of composite endpoints, the {\it net benefit} (NB) and the {\it win ratio} (WR) -- which assess treatment effects using prioritized component outcomes based on clinical importance -- have…
Period-prevalent cohorts are often used for their cost-saving potential in epidemiological studies of survival outcomes. Under this design, prevalent patients allow for evaluations of long-term survival outcomes without the need for long…
The propensity score analysis is one of the most widely used methods for studying the causal treatment effect in observational studies. This paper studies treatment effect estimation with the method of matching weights. This method…
Most statistical tests for treatment effects used in randomized clinical trials with survival outcomes are based on the proportional hazards assumption, which often fails in practice. Data from early exploratory studies may provide evidence…
Accurate and early prediction of a disease allows to plan and improve a patient's quality of future life. During pandemic situations, the medical decision becomes a speed challenge in which physicians have to act fast to diagnose and…
Measuring disease progression in clinical trials for testing novel treatments for multifaceted diseases as Progressive Supranuclear Palsy (PSP), remains challenging. In this study we assess a range of statistical approaches to compare…
Simultaneous tests of superiority and non-inferiority hypotheses on multiple endpoints are often performed in clinical trials to demonstrate that a new treatment is superior over a control on at least one endpoint and non-inferior on the…
In oncological clinical trials, overall survival (OS) is the gold-standard endpoint, but long follow-up and treatment switching can delay or dilute detectable effects. Progression-free survival (PFS) often provides earlier evidence and is…
The analysis of multiple time-to-event outcomes in a randomised controlled clinical trial can be accomplished with exisiting methods. However, depending on the characteristics of the disease under investigation and the circumstances in…
A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the…