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Our paper discovers a new trade-off of using regression adjustments (RAs) in causal inference under covariate-adaptive randomizations (CARs). On one hand, RAs can improve the efficiency of causal estimators by incorporating information from…

Econometrics · Economics 2025-02-19 Liang Jiang , Liyao Li , Ke Miao , Yichong Zhang

Nonlinear longitudinal proportional effect models have been proposed to improve power and provide direct estimates of the proportional treatment effect in randomized clinical trials. These models assume a fixed proportional treatment effect…

Methodology · Statistics 2026-01-23 Michael C. Donohue , Philip S. Insel , Oliver Langford

Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

Applications · Statistics 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Utilizing non-concurrent control data (NCC) in the analysis of late-entering arms in platform trials has recently received considerable attention. While incorporating NCC can lead to increased power and lower sample sizes, it might…

Methodology · Statistics 2025-09-12 Pavla Krotka , Martin Posch , Mohamed Gewily , Günter Höglinger , Marta Bofill Roig

There is a growing interest in leveraging the prevalence of mobile technology to improve health by delivering momentary, contextualized interventions to individuals' smartphones. A just-in-time adaptive intervention (JITAI) adjusts to an…

Other Statistics · Statistics 2018-12-31 Nicholas J. Seewald , Shawna N. Smith , Andy Jinseok Lee , Predrag Klasnja , Susan A. Murphy

Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

Methodology · Statistics 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

Reinforcement learning (RL) is a type of artificial intelligence for making optimal choices. In healthcare, researchers generally use offline RL (ORL), where models are trained and evaluated from retrospective observational data. To…

Machine Learning · Computer Science 2026-04-30 Thomas Frost , Hrisheekesh Vaidya , Steve Harris

Click-through rate (CTR) prediction plays an indispensable role in online platforms. Numerous models have been proposed to capture users' shifting preferences by leveraging user behavior sequences. However, these historical sequences often…

Information Retrieval · Computer Science 2024-04-16 Junjie Huang , Guohao Cai , Jieming Zhu , Zhenhua Dong , Ruiming Tang , Weinan Zhang , Yong Yu

When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

Methodology · Statistics 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

Binary endpoints are common in clinical trials and conditional odds ratios have traditionally been used to assess treatment effects. However, the interpretation of odds ratios is difficult, they are non-collapsible and rely on strong…

Methodology · Statistics 2026-05-20 Martin Schnuerch , Alex Ocampo , Klaus Kähler Holst , Christian Stock

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

The present paper deals with the problem of allocating patients to two competing treatments in the presence of covariates or prognostic factors in order to achieve a good trade-off among ethical concerns, inferential precision and…

Methodology · Statistics 2012-08-17 Alessandro Baldi Antognini , Maroussa Zagoraiou

Randomized Controlled Trials (RCTs) are the gold standard for evaluating the effect of new medical treatments. Treatments must pass stringent regulatory conditions in order to be approved for widespread use, yet even after the regulatory…

Machine Learning · Statistics 2025-03-13 Omer Noy Klein , Alihan Hüyük , Ron Shamir , Uri Shalit , Mihaela van der Schaar

This paper deals with a new design methodology for stratified comparative experiments based on interacting reinforced urn systems. The key idea is to model the interaction between urns for borrowing information across strata and to use it…

This paper studies inference in a randomized controlled trial (RCT) with covariate-adaptive randomization (CAR) and imperfect compliance of a binary treatment. In this context, we study inference on the LATE. As in Bugni et al. (2018,2019),…

Econometrics · Economics 2023-07-25 Federico A. Bugni , Mengsi Gao

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants…

Methodology · Statistics 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

Bayesian clinical trials can benefit of available historical information through the elicitation of informative prior distributions. Concerns are however often raised about the potential for prior-data conflict and the impact of Bayes test…

Methodology · Statistics 2022-12-01 Silvia Calderazzo , Annette Kopp-Schneider

As predictive models are increasingly being deployed in high-stakes decision making (e.g., loan approvals), there has been growing interest in post hoc techniques which provide recourse to affected individuals. These techniques generate…

Machine Learning · Computer Science 2021-07-14 Sohini Upadhyay , Shalmali Joshi , Himabindu Lakkaraju