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Censored survival data are common in clinical trials, but small control groups can pose challenges, particularly in rare diseases or where balanced randomization is impractical. Recent approaches leverage external controls from historical…

统计方法学 · 统计学 2025-05-16 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Wendy Ye , Ilya Lipkovich , Douglas Faries

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Skepticism about the assumption of no unmeasured confounding, also known as exchangeability, is often warranted in making causal inferences from observational data; because exchangeability hinges on an investigator's ability to accurately…

统计方法学 · 统计学 2023-02-22 Yifan Cui , Hongming Pu , Xu Shi , Wang Miao , Eric Tchetgen Tchetgen

In observational studies, treatments are typically not randomized and therefore estimated treatment effects may be subject to confounding bias. The instrumental variable (IV) design plays the role of a quasi-experimental handle since the IV…

统计方法学 · 统计学 2016-08-30 Lan Liu , Wang Miao , Baoluo Sun , James Robins , Eric Tchetgen Tchetgen

A platform trial is an innovative clinical trial design that enables simultaneous and continuous evaluation of multiple treatments within a single master protocol. Existing robust methods restrict analyses to concurrently randomized…

统计方法学 · 统计学 2026-05-15 Yuhan Qian , Yu Du , Jingning Zhang , Yanyao Yi , Patrick J. Heagerty , Ting Ye

We study nonparametric inference for the causal dose-response (or treatment effect) curve when the treatment variable is continuous rather than binary or discrete. We do this by developing doubly robust confidence intervals for the…

统计方法学 · 统计学 2025-08-13 Charles R. Doss

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

统计方法学 · 统计学 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Recent work has focused on the potential and pitfalls of causal identification in observational studies with multiple simultaneous treatments. Building on previous work, we show that even if the conditional distribution of unmeasured…

统计方法学 · 统计学 2025-03-28 Jiajing Zheng , Alexander D'Amour , Alexander Franks

Many trials are designed to collect outcomes at or around pre-specified times after randomization. If there is variability in the times when participants are actually assessed, this can pose a challenge to learning the effect of treatment,…

统计方法学 · 统计学 2024-10-28 Bonnie B. Smith , Yujing Gao , Shu Yang , Ravi Varadhan , Andrea J. Apter , Daniel O. Scharfstein

In experiments that study social phenomena, such as peer influence or herd immunity, the treatment of one unit may influence the outcomes of others. Such "interference between units" violates traditional approaches for causal inference, so…

统计方法学 · 统计学 2023-08-30 David Choi

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…

统计方法学 · 统计学 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data…

统计理论 · 数学 2026-05-15 Ziyang Liu , Niwen Zhou , Peng Wu , Xu Guo

In randomized trials and observational studies, it is often necessary to evaluate the extent to which an intervention affects a time-to-event outcome, which is only partially observed due to right censoring. For instance, in infectious…

统计方法学 · 统计学 2024-12-16 Yutong Jin , Peter B. Gilbert , Aaron Hudson

Study populations are typically sampled from limited points in space and time, and marginalized groups are underrepresented. To assess the external validity of randomized and observational studies, we propose and evaluate the worst-case…

机器学习 · 统计学 2022-02-04 Sookyo Jeong , Hongseok Namkoong

Causal inference is a critical research area with multi-disciplinary origins and applications, ranging from statistics, computer science, economics, psychology to public health. In many scientific research, randomized experiments provide a…

统计方法学 · 统计学 2022-07-26 Jingying Zeng

Classical semiparametric inference with missing outcome data is not robust to contamination of the observed data and a single observation can have arbitrarily large influence on estimation of a parameter of interest. This sensitivity is…

统计方法学 · 统计学 2021-03-02 Eva Cantoni , Xavier de Luna

This paper examines the identification and estimation of heterogeneous treatment effects in event studies, emphasizing the importance of both lagged dependent variables and treatment effect heterogeneity. We show that omitting lagged…

计量经济学 · 经济学 2025-09-18 Irene Botosaru , Laura Liu

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

统计方法学 · 统计学 2021-12-01 David Cheng , Tianxi Cai

Conditional effect estimation has great scientific and policy importance because interventions may impact subjects differently depending on their characteristics. Most research has focused on estimating the conditional average treatment…

统计方法学 · 统计学 2023-04-25 Alec McClean , Zach Branson , Edward H. Kennedy

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