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In the presence of treatment effect heterogeneity, the average treatment effect (ATE) in a randomized controlled trial (RCT) may differ from the average effect of the same treatment if applied to a target population of interest. If all…

Methodology · Statistics 2017-05-02 Trang Quynh Nguyen , Cyrus Ebnesajjad , Stephen R. Cole , Elizabeth A. Stuart

The randomization inference literature studying randomized controlled trials (RCTs) assumes that units' potential outcomes are deterministic. This assumption is unlikely to hold, as stochastic shocks may take place during the experiment. In…

Econometrics · Economics 2022-12-15 Antoine Deeb , Clément de Chaisemartin

Randomized Controlled Trials (RCTs) represent the gold standard for causal inference yet remain a scarce resource. While large-scale observational data is often available, it is utilized only for retrospective fusion, and remains discarded…

Machine Learning · Statistics 2026-03-05 Erdun Gao , Liang Zhang , Jake Fawkes , Aoqi Zuo , Wenqin Liu , Haoxuan Li , Mingming Gong , Dino Sejdinovic

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…

Regression experts consistently recommend plotting residuals for model diagnosis, despite the availability of many numerical hypothesis test procedures designed to use residuals to assess problems with a model fit. Here we provide evidence…

Applications · Statistics 2024-11-04 Weihao Li , Dianne Cook , Emi Tanaka , Susan VanderPlas

The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

Signal Processing · Electrical Eng. & Systems 2023-05-10 Sayeri Lala , Niraj K. Jha

Observational causal inference is useful for decision making in medicine when randomized clinical trials (RCT) are infeasible or non generalizable. However, traditional approaches fail to deliver unconfounded causal conclusions in practice.…

We study the problem of learning to choose from m discrete treatment options (e.g., news item or medical drug) the one with best causal effect for a particular instance (e.g., user or patient) where the training data consists of passive…

Machine Learning · Statistics 2017-08-02 Nathan Kallus

Increasing the success rate of a process, i.e. the percentage of cases that end in a positive outcome, is a recurrent process improvement goal. At runtime, there are often certain actions (a.k.a. treatments) that workers may execute to lift…

Machine Learning · Computer Science 2023-03-08 Zahra Dasht Bozorgi , Marlon Dumas , Marcello La Rosa , Artem Polyvyanyy , Mahmoud Shoush , Irene Teinemaa

In clinical trials, an experimental treatment is sometimes added on to a standard of care or control therapy in multiple treatment phases (e.g., concomitant and maintenance phases) to improve patient outcomes. When the new regimen provides…

Methodology · Statistics 2020-11-19 Sudipta Bhattacharya , Jyotirmoy Dey

In Randomised Controlled Trials (RCT) with treatment non-compliance, instrumental variable approaches are used to estimate complier average causal effects. We extend these approaches to cost-effectiveness analyses, where methods need to…

Methodology · Statistics 2016-12-02 Karla DiazOrdaz , Angelo Franchini , Richard Grieve

Despite the importance of emphasizing the right psychotherapy treatment for an individual patient, assessing the outcome of the therapy session is equally crucial. Evidence showed that continuous monitoring patient's progress can…

Computation and Language · Computer Science 2022-12-19 Noor Fazilla Abd Yusof , Chenghua Lin

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

As a natural extension to the standard conformal prediction method, several conformal risk control methods have been recently developed and applied to various learning problems. In this work, we seek to control the conformal risk in…

Machine Learning · Computer Science 2024-05-02 Yunpeng Xu , Wenge Guo , Zhi Wei

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Effective decision making from randomised controlled clinical trials relies on robust interpretation of the numerical results. However, the language we use to describe clinical trials can cause confusion both in trial design and in…

Observational studies are valuable for estimating the effects of various medical interventions, but are notoriously difficult to evaluate because the methods used in observational studies require many untestable assumptions. This lack of…

Applications · Statistics 2022-09-14 Ethan Steinberg , Nikolaos Ignatiadis , Steve Yadlowsky , Yizhe Xu , Nigam H. Shah

A Randomized Control Trial (RCT) is considered as the gold standard for evaluating the effect of any intervention or treatment. However, its feasibility is often hindered by ethical, economical, and legal considerations, making…

Machine Learning · Computer Science 2024-03-13 Md Saiful Islam , Sahil Shikalgar , Md. Noor-E-Alam

A sensitivity analysis in an observational study assesses the robustness of significant findings to unmeasured confounding. While sensitivity analyses in matched observational studies have been well addressed when there is a single outcome…

Methodology · Statistics 2015-11-05 Colin B. Fogarty , Dylan S. Small
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