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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

With increasing data availability, causal effects can be evaluated across different data sets, both randomized controlled trials (RCTs) and observational studies. RCTs isolate the effect of the treatment from that of unwanted (confounding)…

Causal inference is vital for informed decision-making across fields such as biomedical research and social sciences. Randomized controlled trials (RCTs) are considered the gold standard for internal validity of inferences, whereas…

Methodology · Statistics 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Randomized controlled trials are the gold standard for causal inference and play a pivotal role in modern evidence-based medicine. However, the sample sizes they use are often too limited to draw significant causal conclusions for subgroups…

Methodology · Statistics 2024-04-26 Xi Lin , Jens Magelund Tarp , Robin J. Evans

Background: Randomized controlled trials (RCTs) are costly, time-consuming, and often infeasible, while treatment-effect estimation from observational data is limited by unobserved confounding. Methods: We developed a three-step framework…

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

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

Randomized experiments are the gold standard for causal inference, and justify simple comparisons across treatment groups. Regression adjustment provides a convenient way to incorporate covariate information for additional efficiency. This…

Methodology · Statistics 2022-10-25 Anqi Zhao , Peng Ding

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

Methodology · Statistics 2025-02-20 Peijin Wang , Hwanhee Hong , Kyungeun Jeon , Laine Elliott Thomas

Randomized experiments are the gold standard for estimating the causal effects of an intervention. In the simplest setting, each experimental unit is randomly assigned to receive treatment or control, and then the outcomes in each treatment…

Methodology · Statistics 2020-06-05 Guillaume Basse , Yi Ding , Panos Toulis

The authors propose robust adaptive strategies based on stochastic minimax optimization for a series of simulated treatments on a one-dimensional patient phantom. The plan applied during the first fractions should be able to handle…

Optimization and Control · Mathematics 2018-10-01 Michelle Böck , Anders Forsgren , Kjell Eriksson , Björn Hårdemark

Although randomized controlled trials (RCTs) are a cornerstone of comparative effectiveness, they typically have much smaller sample size than observational studies because of financial and ethical considerations. Therefore there is…

Methodology · Statistics 2023-11-16 Lauren D. Liao , Emilie Højbjerre-Frandsen , Alan E. Hubbard , Alejandro Schuler

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

Combining observational and experimental data for causal inference can improve treatment effect estimation. However, many observational data sets cannot be released due to data privacy considerations, so one researcher may not have access…

Methodology · Statistics 2024-08-26 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

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…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Randomized clinical trials eliminate confounding but impose strict exclusion criteria that limit recruitment to a subset of the population. Observational datasets are more inclusive but suffer from confounding -- often providing overly…

Machine Learning · Statistics 2022-10-31 Eric V. Strobl , Thomas A. Lasko

We consider the problem of estimating the average treatment effect (ATE) when both randomized control trial (RCT) data and external real-world data (RWD) are available. We decompose the ATE estimand as the difference between a pooled-ATE…

Methodology · Statistics 2025-01-22 Mark van der Laan , Sky Qiu , Jens Magelund Tarp , Lars van der Laan

In the online ride-hailing pricing context, companies often conduct randomized controlled trials (RCTs) and utilize uplift models to assess the effect of discounts on customer orders, which substantially influences competitive market…

Methodology · Statistics 2025-09-24 Kairong Han , Weidong Huang , Taiyang Zhou , Peng Zhen , Kun Kuang

Restricted mean survival time (RMST) offers a compelling nonparametric alternative to hazard ratios for right-censored time-to-event data, particularly when the proportional hazards assumption is violated. By capturing the total event-free…

Methodology · Statistics 2025-01-28 Jinghao Sun , Douglas E. Schaubel , Eric J. Tchetgen Tchetgen

This project considers how one might augment a limited amount of data from randomized controlled trial (RCT) with more plentiful data from an observational database (ODB), in order to estimate a causal effect. In our motivating setting, the…

Methodology · Statistics 2018-10-23 Evan Rosenman , Art B. Owen , Michael Baiocchi , Hailey Banack