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Estimating heterogeneous treatment effects has become increasingly important in many fields and life and death decisions are now based on these estimates: for example, selecting a personalized course of medical treatment. Recently, a…

统计方法学 · 统计学 2019-04-01 Sören R. Künzel , Simon J. S. Walter , Jasjeet S. Sekhon

In clinical trials, principal stratification analysis is commonly employed to address the issue of truncation by death, where a subject dies before the outcome can be measured. However, in practice, many survivor outcomes may remain…

统计方法学 · 统计学 2025-07-08 Wei Li , Yuan Liu , Shanshan Luo , Zhi Geng

In clinical trials, patients may discontinue treatments prematurely, breaking the initial randomization and, thus, challenging inference. Stakeholders in drug development are generally interested in going beyond the Intention-To-Treat (ITT)…

Clinical trials are notorious for their high failure rates and steep costs, leading to wasted time and resources spend, prolonged development timelines, and delayed patient access to new therapies. A key contributor to these failures is…

量子物理 · 物理学 2026-01-19 Laia Domingo , Christine Johnson

Recent trends in planning research have led to empirical comparison becoming commonplace. The field has started to settle into a methodology for such comparisons, which for obvious practical reasons requires running a subset of planners on…

人工智能 · 计算机科学 2011-06-10 E. Dahlman , A. E. Howe

What is the ideal regression (if any) for estimating average causal effects? We study this question in the setting of discrete covariates, deriving expressions for the finite-sample variance of various stratification estimators. This…

统计方法学 · 统计学 2022-09-26 P. Richard Hahn , Andrew Herren

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

统计方法学 · 统计学 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

Principal stratification analysis evaluates how causal effects of a treatment on a primary outcome vary across strata of units defined by their treatment effect on some intermediate quantity. This endeavor is substantially challenged when…

统计方法学 · 统计学 2024-03-21 Chanmin Kim , Corwin Zigler

Over time, clinical trials have increasingly incorporated complex design and analysis elements such as interim analyses, adaptations, multiple endpoints, and sophisticated multiplicity schemes for multiple endpoints and/or treatment arms…

We study estimation and inference for heterogeneous principal causal effects with binary treatments and binary intermediate variables. Principal causal effects are subgroup effects within strata defined by potential values of an…

统计方法学 · 统计学 2026-03-11 Rui Zhang , Charles R. Doss , Jared D. Huling

In neoadjuvant trials on early-stage breast cancer, patients are usually randomized into a control group and a treatment group with an additional target therapy. Early efficacy of the new regimen is assessed via the binary pathological…

统计方法学 · 统计学 2022-04-04 Xiaoqing Tan , Judah Abberbock , Priya Rastogi , Gong Tang

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable…

统计方法学 · 统计学 2025-09-01 Jiaqi Tong , Chao Cheng , Guangyu Tong , Michael O. Harhay , Fan Li

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

统计计算 · 统计学 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

Extending (generalizing or transporting) causal inferences from a randomized trial to a target population requires ``generalizability'' or ``transportability'' assumptions, which state that randomized and non-randomized individuals are…

We investigate how to exploit structural similarities of an individual's potential outcomes (POs) under different treatments to obtain better estimates of conditional average treatment effects in finite samples. Especially when it is…

机器学习 · 统计学 2021-10-26 Alicia Curth , Mihaela van der Schaar

Comparisons of different treatments or production processes are the goals of a significant fraction of applied research. Unsurprisingly, two-sample problems play a main role in Statistics through natural questions such as `Is the the new…

统计方法学 · 统计学 2017-09-05 P. C. Álvarez-Esteban , E. del Barrio , J. A. Cuesta-Albertos , C. Matrán

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

统计方法学 · 统计学 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Randomized trials balance all covariates on average and provide the gold standard for estimating treatment effects. Chance imbalances nevertheless exist more or less in realized treatment allocations and intrigue an important question: what…

统计方法学 · 统计学 2023-07-18 Anqi Zhao , Peng Ding

The concept of Probability of Causation (PC) is critically important in legal contexts and can help in many other domains. While it has been around since 1986, current operationalizations can obtain only the minimum and maximum values of…

统计方法学 · 统计学 2018-08-14 Tapajit Dey , Audris Mockus

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

机器学习 · 统计学 2022-06-08 Susan Athey , Guido Imbens