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Clinicians increasingly rely on prediction models to guide treatment choices. Most prediction models, however, are developed using observational data that include some patients who have already received the treatment the prediction model is…

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

One central goal of design of observational studies is to embed non-experimental data into an approximate randomized controlled trial using statistical matching. Despite empirical researchers' best intention and effort to create…

Methodology · Statistics 2022-06-22 Kan Chen , Siyu Heng , Qi Long , Bo Zhang

In observational studies, the identification of causal estimands depends on the no unmeasured confounding (NUC) assumption. As this assumption is not testable from observed data, sensitivity analysis plays an important role in observational…

Methodology · Statistics 2023-09-28 Md Abdul Basit , Mahbub A. H. M. Latif , Abdus S Wahed

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

Machine Learning · Computer Science 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

Drawing causal inferences from observational studies (OS) requires unverifiable validity assumptions; however, one can falsify those assumptions by benchmarking the OS with experimental data from a randomized controlled trial (RCT). A major…

Cluster randomized trials are widely used when individual randomization is logistically infeasible or when correlations between observations cannot be ignored, especially in fields such as ophthalmology, infectious disease, vaccine…

Methodology · Statistics 2026-05-04 Wanying Shao , Toshimitsu Hamasaki , Scott Evans , Guoqing Diao

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

Econometrics · Economics 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

We will study the impact of adolescent sports participation on early-adulthood health using longitudinal data from the National Study of Youth and Religion. We focus on two primary outcomes measured at ages 23--28 -- self-rated health and…

When estimating causal effects using observational data, it is desirable to replicate a randomized experiment as closely as possible by obtaining treated and control groups with similar covariate distributions. This goal can often be…

Methodology · Statistics 2010-10-28 Elizabeth A. Stuart

The ability to generalize experimental results from randomized control trials (RCTs) across locations is crucial for informing policy decisions in targeted regions. Such generalization is often hindered by the lack of identifiability due to…

Econometrics · Economics 2021-12-10 Xinkun Nie , Guido Imbens , Stefan Wager

Randomized controlled trials (RCTs) yield internally valid causal effect estimates, but generalizing these results to target populations with different characteristics requires an untestable selection ignorability assumption: conditional on…

Methodology · Statistics 2026-03-31 Amir Asiaee , Samhita Pal , Jared D. Huling

The recent growth in data availability in football has increased the risk of incorrect use of data-driven models, making guidelines on their validation and application necessary. The Expected Threat (xT) model is an accessible option for…

Applications · Statistics 2026-04-28 Koen van Arem , Jakob Söhl , Mirjam Bruinsma , Geurt Jongbloed

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…

Methodology · Statistics 2025-10-07 Han Chang Chiam , Franz König , Martin Posch

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

Testing whether a variable of interest affects the outcome is one of the most fundamental problem in statistics and is often the main scientific question of interest. To tackle this problem, the conditional randomization test (CRT) is…

Methodology · Statistics 2023-05-26 Dae Woong Ham , Jiaze Qiu

Background: Randomized controlled trials are often used to inform policy and practice for broad populations. The average treatment effect (ATE) for a target population, however, may be different from the ATE observed in a trial if there are…

Clinical trials in specific indications require the administration of rescue medication in case a patient does not sufficiently respond to investigational treatment. The application of additional treatment on an as needed basis causes…

Methodology · Statistics 2016-08-30 Gerd K. Rosenkranz

In the presence of competing events, many investigators are interested in a direct treatment effect on the event of interest that does not capture treatment effects on competing events. Classical survival analysis methods that treat…

Methodology · Statistics 2025-10-29 Takuya Kawahara , Sean McGrath , Jessica G Young

We study RCTs that evaluate the impact of service interventions, for example, teachers or advisors conducting proactive outreach to at-risk students, medical providers giving medication adherence support by calling or texting, or social…

Methodology · Statistics 2025-12-22 Justin Boutilier , Jonas Oddur Jonasson , Hannah Li , Erez Yoeli
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