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Both cluster randomized trials and quasi-experimental designs are used to evaluate the impact of health and social policies and interventions. Stepped-wedge cluster randomized trials randomize a staggered adoption approach, while recent…

Methodology · Statistics 2026-04-15 Haidong Lu , Gregg S. Gonsalves , Fan Li , Guanyu Tong , Lee Kennedy-Shaffer

The conventional more-is-better dose selection paradigm, which targets the maximum tolerated dose (MTD), is not suitable for the development of targeted therapies and immunotherapies as the efficacy of these novel therapies may not increase…

Methodology · Statistics 2023-08-31 Peng Yang , Daniel Li , Ruitao Lin , Bo Huang , Ying Yuan

Cell heterogeneity plays an important role in patient responses to drug treatments. In many cancers, it is associated with poor treatment outcomes. Many modern drug combination therapies aim to exploit cell heterogeneity, but determining…

Quantitative Methods · Quantitative Biology 2025-02-18 Simon F. Martina-Perez , Samuel W. S. Johnson , Rebecca M. Crossley , Jennifer C. Kasemeier , Paul M. Kulesa , Ruth E. Baker

Analyses of heterogeneous treatment effects (HTE) are common in applied causal inference research. However, when outcomes are latent variables assessed via psychometric instruments such as educational tests, standard methods ignore the…

In clinical trials, the response of a given subject often depends on the selected treatment as well as on some covariates. We study optimal approximate designs of experiments in the models with treatment and covariate effects. We allow for…

Statistics Theory · Mathematics 2019-07-10 Samuel Rosa

Understanding treatment effect heterogeneity has become an increasingly popular task in various fields, as it helps design personalized advertisements in e-commerce or targeted treatment in biomedical studies. However, most of the existing…

Methodology · Statistics 2024-07-12 Waverly Wei , Xinwei Ma , Jingshen Wang

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

We developed a study design for rare disease clinical trials (RDTs) that efficiently evaluate treatments, promotes access to new treatments during treatment development, and optimizes healthcare resource utilization for future treatment…

Applications · Statistics 2016-07-04 Jian Yong , Sohaib H. Mohammad , Yan Yuan

State-level policy studies often conduct heterogeneity analyses that quantify how treatment effects vary across state characteristics. These analyses may be used to inform state-specific policy decisions, or to infer how the effect of a…

Empirical studies using Regression Discontinuity (RD) designs often explore heterogeneous treatment effects based on pretreatment covariates, even though no formal statistical methods exist for such analyses. This has led to the widespread…

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

The global average treatment effect (GATE) is a primary quantity of interest in the study of causal inference under network interference. With a correctly specified exposure model of the interference, the Horvitz-Thompson (HT) and H\'ajek…

Methodology · Statistics 2020-09-07 Johan Ugander , Hao Yin

In cluster randomized trials, the average treatment effect among individuals (i-ATE) can be different from the cluster average treatment effect (c-ATE) when informative cluster size is present, i.e., when treatment effects or participant…

Methodology · Statistics 2025-10-02 Bryan S. Blette , Zhe Chen , Brennan C. Kahan , Andrew Forbes , Michael O. Harhay , Fan Li

Background: Stepped wedge cluster randomized trials (SW-CRTs) involve sequential measurements within clusters over time. Initially, all clusters start in the control condition before crossing over to the intervention on a staggered…

Methodology · Statistics 2026-01-21 Jale Basten , Katja Ickstadt , Nina Timmesfeld

One primary goal of precision medicine is to estimate the individualized treatment rules (ITRs) that optimize patients' health outcomes based on individual characteristics. Health studies with multiple treatments are commonly seen in…

Methodology · Statistics 2025-05-07 Xuqiao Li , Qiuyan Zhou , Ying Wu , Ying Yan

Recently, coding has been a useful technique to mitigate the effect of stragglers in distributed computing. However, coding in this context has been mainly explored under the assumption of homogeneous workers, although the real-world…

Distributed, Parallel, and Cluster Computing · Computer Science 2020-02-18 DaeJin Kim , Hyegyeong Park , Junkyun Choi

Deep learning models have demonstrated promising results in estimating treatment effects (TEE). However, most of them overlook the variations in treatment outcomes among subgroups with distinct characteristics. This limitation hinders their…

Machine Learning · Computer Science 2024-01-31 Seungyeon Lee , Ruoqi Liu , Wenyu Song , Ping Zhang

The credibility revolution advances the use of research designs that permit identification and estimation of causal effects. However, understanding which mechanisms produce measured causal effects remains a challenge. The dominant current…

Econometrics · Economics 2026-04-08 Jiawei Fu , Tara Slough

Many policy evaluations occur in settings where treatment is randomized at the cluster level, and there is treatment noncompliance within each cluster. For example, villages might be assigned to treatment and control, but residents in each…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

When treatments are non-randomly assigned, continuous, and yield heterogeneous effects at the same intensity, causal identification becomes particularly challenging. In such contexts, existing approaches often fail to provide…

Econometrics · Economics 2026-03-05 Augusto Cerqua , Roberta Di Stefano , Raffaele Mattera