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Given the cost and duration of phase III and phase IV clinical trials, the development of statistical methods for go/no-go decisions is vital. In this paper, we introduce a Bayesian methodology to compute the probability of success based on…

Methodology · Statistics 2020-10-27 Ethan M. Alt , Matthew A. Psioda , Joseph G. Ibrahim

The inclusion of the propensity score as a covariate in Bayesian regression trees for causal inference can reduce the bias in treatment effect estimations, which occurs due to the regularization-induced confounding phenomenon. This study…

Methodology · Statistics 2018-08-30 Pedro Henrique Filipini dos Santos , Hedibert Freitas Lopes

Hybrid clinical trials, that borrow real-world data (RWD), are gaining interest, especially for rare diseases. They assume RWD and randomized control arm be exchangeable, but violations can bias results, inflate type I error, or reduce…

Bayesian doubly robust (DR) causal inference faces a fundamental dilemma: joint modeling of outcome and propensity score suffers from the feedback problem where outcome information contaminates propensity score estimation, while two-step…

Methodology · Statistics 2026-01-05 Shunichiro Orihara , Tomotaka Momozaki , Shonosuke Sugasawa

Power priors are used for incorporating historical data in Bayesian analyses by taking the likelihood of the historical data raised to the power $\alpha$ as the prior distribution for the model parameters. The power parameter $\alpha$ is…

Methodology · Statistics 2023-06-27 Samuel Pawel , Frederik Aust , Leonhard Held , Eric-Jan Wagenmakers

Covariate adjustment and methods of incorporating historical data in randomized clinical trials (RCTs) each provide opportunities to increase trial power. We unite these approaches for the analysis of RCTs with binary outcomes based on the…

Methodology · Statistics 2022-12-21 Alyssa M. Vanderbeek , Jessica L. Ross , David P. Miller , Alejandro Schuler

The question of how individual patient data from cohort studies or historical clinical trials can be leveraged for designing more powerful, or smaller yet equally powerful, clinical trials becomes increasingly important in the era of…

Methodology · Statistics 2022-09-01 Sandra Siegfried , Stephen Senn , Torsten Hothorn

External information borrowing is often considered in order to improve a clinical trial's efficiency. The Bayesian approach borrows such external information by specifying an informative prior distribution. A potential issue with this…

Methodology · Statistics 2025-08-25 Silvia Calderazzo , Manuel Wiesenfarth , Vivienn Weru , Annette Kopp-Schneider

Polygenic risk scores (PRS) developed from genome-wide association studies (GWAS) can be used for risk stratification by quantifying the genetic contribution to disease, and many clinical applications have been proposed. Bayesian methods…

Methodology · Statistics 2026-03-11 Yuzheng Dun , Nilanjan Chatterjee , Jin Jin , Akihiko Nishimura

We propose a novel adaptive importance sampling scheme for Bayesian inversion problems where the inference of the variables of interest and the power of the data noise is split. More specifically, we consider a Bayesian analysis for the…

Computation · Statistics 2021-07-27 L. Martino , F. Llorente , E. Curbelo , J. Lopez-Santiago , J. Miguez

Randomized controlled clinical trials provide the gold standard for evidence generation in relation to the efficacy of a new treatment in medical research. Relevant information from previous studies may be desirable to incorporate in the…

Methodology · Statistics 2024-05-29 Lou E. Whitehead , James M. S. Wason , Oliver Sailer , Haiyan Zheng

In preference-based reinforcement learning (RL), an agent interacts with the environment while receiving preferences instead of absolute feedback. While there is increasing research activity in preference-based RL, the design of formal…

Machine Learning · Computer Science 2020-06-30 Ellen R. Novoseller , Yibing Wei , Yanan Sui , Yisong Yue , Joel W. Burdick

Parameter estimates for associated genetic variants, report ed in the initial discovery samples, are often grossly inflated compared to the values observed in the follow-up replication samples. This type of bias is a consequence of the…

Applications · Statistics 2011-04-15 Lizhen Xu , Radu V. Craiu , Lei Sun

Randomized controlled trials (RCTs) are the gold standard for causal inference, yet practical constraints often limit the size of the concurrent control arm. Borrowing control data from previous trials offers a potential efficiency gain,…

Methodology · Statistics 2026-03-17 Linying Yang , Xing Liu , Robin J. Evans

It is generally believed that bias is minimized in well-controlled randomized clinical trials. However, bias can arise in active controlled noninferiority trials because the inference relies on a previously estimated effect size obtained…

Applications · Statistics 2013-12-02 Lei Nie , Zhiwei Zhang , Daniel Rubin , Jianxiong Chu

Bayesian design of experiments and sample size calculations usually rely on complex Monte Carlo simulations in practice. Obtaining bounds on Bayesian notions of the false-positive rate and power therefore often lack closed-form or…

Methodology · Statistics 2025-02-06 Riko Kelter , Samuel Pawel

Important objectives in cancer research are the prediction of a patient's risk based on molecular measurements such as gene expression data and the identification of new prognostic biomarkers (e.g. genes). In clinical practice, this is…

Applications · Statistics 2020-04-17 Katrin Madjar , Manuela Zucknick , Katja Ickstadt , Jörg Rahnenführer

With the increasing availability of data from historical studies and real-world data sources, hybrid control designs that incorporate external data into the evaluation of current studies are being increasingly adopted. In these designs, it…

Methodology · Statistics 2025-06-23 Masahiro Kojima , Shunichiro Orihara , Keisuke Hanada , Tomohiro Ohigashi

Hypothesis testing of structure in correlation and covariance matrices is of broad interest in many application areas. In high dimensions and/or small to moderate sample sizes, high error rates in testing is a substantial concern. This…

Methodology · Statistics 2026-01-07 Ziyang Ding , David Dunson

In clinical trials, there often exist multiple historical studies for the same or related treatment investigated in the current trial. Incorporating historical data in the analysis of the current study is of great importance, as it can help…

Methodology · Statistics 2021-02-02 Huaqing Jin , Guosheng Yin