Related papers: A Hybrid Prior Bayesian Method for Combining Domes…
Use of historical data in clinical trial design and analysis has shown various advantages such as reduction of within-study placebo-treated number of subjects and increase of study power. The meta-analytic-predictive (MAP) approach accounts…
We propose a novel empirical Bayes robust MAP (EB-rMAP) prior to adaptively leverage external/historical data. Built on Box's prior predictive p-value, the EB-rMAP prior framework balances between model parsimony and flexibility through a…
Bayesian dynamic borrowing has become an increasingly important tool for evaluating the consistency of regional treatment effects which is a key requirement for local regulatory approval of a new drug. It helps increase the precision of…
Hybrid randomized controlled trials (hybrid RCTs) integrate external control data, such as historical or concurrent data, with data from randomized trials. While numerous frequentist and Bayesian methods, such as the test-then-pool 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…
Integrating multiple observational studies for meta-analysis has sparked much interest. The presented R package WMAP (Weighted Meta-Analysis with Pseudo-Population) addresses a critical gap in the implementation of integrative weighting…
Bridging the gap between internal and external validity is crucial for heterogeneous treatment effect estimation. Randomised controlled trials (RCTs), favoured for their internal validity due to randomisation, often encounter challenges in…
The use of information from real world to assess the effectiveness of medical products is becoming increasingly popular and more acceptable by regulatory agencies. According to a strategic real-world evidence framework published by U.S.…
It is highly desirable to borrow information from external data to augment a control arm in a randomized clinical trial, especially in settings where the sample size for the control arm is limited. However, a main challenge in borrowing…
Introduction: Increasing interest in real-world evidence has fueled the development of study designs incorporating real-world data (RWD). Using the Causal Roadmap, we specify three designs to evaluate the difference in risk of major adverse…
Obtaining causally interpretable meta-analysis results is challenging when there are differences in the distribution of effect modifiers between eligible trials. To overcome this, recent work on transportability methods has considered…
Quantitative Systems Pharmacology (QSP) promises to accelerate drug development, enable personalized medicine, and improve the predictability of clinical outcomes. Realizing this potential requires effectively managing the complexity of…
Early-phase clinical trials face the challenge of selecting optimal drug doses that balance safety and efficacy due to uncertain dose-response relationships and varied participant characteristics. Traditional randomized dose allocation…
In early phase drug development of combination therapy, the primary objective is to preliminarily assess whether there is additive activity from a novel agent when combined with an established monotherapy. Due to potential feasibility…
The integration of external data using Bayesian mixture priors has become a powerful approach in clinical trials, offering significant potential to improve trial efficiency. Despite their strengths in analytical tractability and practical…
Phase Ib/II oncology trials, despite their small sample sizes, aim to provide information for optimal internal company decision-making concerning novel drug development. Hybrid controls (a combination of the current control arm and controls…
The objective of this work is to develop an Electronic Medical Record (EMR) data processing tool that confers clinical context to Machine Learning (ML) algorithms for error handling, bias mitigation and interpretability. We present…
A platform trial is an innovative clinical trial design that enables simultaneous and continuous evaluation of multiple treatments within a single master protocol. Existing robust methods restrict analyses to concurrently randomized…
Prior information is often incorporated informally when planning a clinical trial. Here, we present an approach on how to incorporate prior information, such as data from historical clinical trials, into the nuisance parameter based sample…
There is growing interest in a hybrid control design for treatment evaluation, where a randomized controlled trial is augmented with external control data from a previous trial or a real world data source. The hybrid control design has the…