Related papers: Regional consistency evaluation and sample size ca…
Multi-regional clinical trials (MRCTs) have become common practice for drug development and global registration. Once overall significance is established, demonstrating regional consistency is critical for local health authorities. Methods…
Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…
Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…
Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…
Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…
Machine learning (ML) models are increasingly used as decision-support tools in high-risk domains. Evaluating the causal impact of deploying such models can be done with a randomized controlled trial (RCT) that randomizes users to ML vs.…
Randomized controlled trials (RCTs) have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically…
Statistical methodology plays a crucial role in drug regulation. Decisions by the FDA or EMA are typically made based on multiple primary studies testing the same medical product, where the two-trials rule is the standard requirement,…
Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and…
Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…
The Dunnett procedure compares several treatment or dose groups with a control group, while controlling the familywise error rate. When deviations from the normal distribution and heterogeneous variances occur, the nominal $\alpha$ level…
Choice consistency with utility maximization, as a key assumption in economics, has been extensively used to evaluate decision quality of individuals and to predict real-world outcomes across different contexts. Here we investigate the…
There has been a growing trend that activities relating to clinical trials take place at locations other than traditional trial sites (hence decentralized clinical trials or DCTs), some of which are at settings of real-world clinical…
Equivalence testing plays a key role in several domains, such as the development of generic medical products, which are therapeutically equivalent to brand-name drugs but with reduced cost and increased accessibility. Promoting access to…
Manufacturers are required to demonstrate products meet reliability targets. A typical way to achieve this is with reliability demonstration tests (RDTs), in which a number of products are put on test and the test is passed if a target…
We argue that randomized controlled trials (RCTs) are special even among settings where average treatment effects are identified by a nonparametric unconfoundedness assumption. This claim follows from two results of Robins and Ritov (1997):…
Addressing the reproducibility crisis in artificial intelligence through the validation of reported experimental results is a challenging task. It necessitates either the reimplementation of techniques or a meticulous assessment of papers…
In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…
Micro-randomized trials (MRTs) are widely used to assess the marginal and moderated effect of mobile health (mHealth) treatments delivered via mobile devices. In many applications, the mHealth treatments are categorical with multiple levels…
This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs)…