Related papers: A meta-analytic framework to adjust for bias in ex…
Background: Advanced adaptive randomised clinical trials are increasingly used. Compared to their conventional counterparts, their flexibility may make them more efficient, increase the probability of obtaining conclusive results without…
It is increasingly common to augment randomized controlled trial with external controls from observational data, to evaluate the treatment effect of an intervention. Traditional approaches to treatment effect estimation involve ambiguous…
Randomized control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their…
We aim to generalize the results of a randomized controlled trial (RCT) to a target population with the help of some observational data. This is a problem of causal effect identification with multiple data sources. Challenges arise when the…
Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly…
The most dangerous error in clinical trial interpretation is equating p > 0.05 with no effect. This review provides a practical, algorithm-based framework for classifying randomized controlled trial (RCT) results into six distinct…
In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the…
We recently developed a new method riAFT-BART to draw causal inferences about population treatment effect on patient survival from clustered and censored survival data while accounting for the multilevel data structure. The practical…
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.…
Observed events in recommendation are consequence of the decisions made by a policy, thus they are usually selectively labeled, namely the data are Missing Not At Random (MNAR), which often causes large bias to the estimate of true outcomes…
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…
The random-effects or normal-normal hierarchical model is commonly utilized in a wide range of meta-analysis applications. A Bayesian approach to inference is very attractive in this context, especially when a meta-analysis is based only on…
We propose a prognostic stratum matching framework that addresses the deficiencies of Randomized trial data subgroup analysis and transforms ObservAtional Data to be used as if they were randomized, thus paving the road for precision…
Background: Non-inferiority studies based on non-randomised data are increasingly used in clinical research but remain prone to unmeasured confounding. The classical E-value offers a simple way to quantify such bias but has been applied…
Clinical trials often collect data on multiple outcomes, such as overall survival (OS), progression-free survival (PFS), and response to treatment (RT). In most cases, however, study designs only use primary outcome data for interim and…
A central obstacle in the objective assessment of treatment effect (TE) estimators in randomized control trials (RCTs) is the lack of ground truth (or validation set) to test their performance. In this paper, we propose a novel…
Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…
Estimation of conditional average treatment effects (CATEs) plays an essential role in modern medicine by informing treatment decision-making at a patient level. Several metalearners have been proposed recently to estimate CATEs in an…
Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…
In meta-analysis, the random-effects models are standard tools to address between-study heterogeneity in evidence synthesis analyses. For the random-effects distribution models, the normal distribution model has been adopted in most…