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Treatment policy estimands are frequently favored by regulators, as they assess the effect of treatment assignment regardless of post-randomization events. Despite best efforts, missing data due to study discontinuation cannot be fully…

Methodology · Statistics 2026-05-13 Ajmal Oodally , Craig Wang , Zheng Li , Tim Morris , Tobias Mütze , Arunava Chakravartty

The statistical analysis of clinical trials is often complicated by missing data. Patients sometimes experience intercurrent events (ICEs), which usually (although not always) lead to missing subsequent outcome measurements for such…

Methodology · Statistics 2025-07-03 Brendah Nansereko , Marcel Wolbers , James Carpenter , Jonathan Bartlett

The goal of causal inference is to understand the outcome of alternative courses of action. However, all causal inference requires assumptions. Such assumptions can be more influential than in typical tasks for probabilistic modeling, and…

Methodology · Statistics 2016-10-31 Dustin Tran , Francisco J. R. Ruiz , Susan Athey , David M. Blei

When treatment policy estimands are of interest, clinical trials often attempt to collect patient data after intercurrent events (ICEs), although such data are often limited. Retrieved dropout imputation methods, which use pre-ICE and…

Methodology · Statistics 2026-03-31 Brendah Nansereko , Marcel Wolbers , James R. Carpenter , Jonathan W. Bartlett

The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires…

Methodology · Statistics 2023-08-28 Suzie Cro , James H Roger , James R Carpenter

Propensity score trimming, which discards subjects with propensity scores below a threshold, is a common way to address positivity violations that complicate causal effect estimation. However, most works on trimming assume treatment is…

Methodology · Statistics 2024-07-31 Zach Branson , Edward H. Kennedy , Sivaraman Balakrishnan , Larry Wasserman

Causal inference is a critical research topic across many domains, such as statistics, computer science, education, public policy and economics, for decades. Nowadays, estimating causal effect from observational data has become an appealing…

Methodology · Statistics 2020-02-10 Liuyi Yao , Zhixuan Chu , Sheng Li , Yaliang Li , Jing Gao , Aidong Zhang

Inferring causal effects of treatments is a central goal in many disciplines. The potential outcomes framework is a main statistical approach to causal inference, in which a causal effect is defined as a comparison of the potential outcomes…

Methodology · Statistics 2018-01-04 Peng Ding , Fan Li

The estimands framework outlined in ICH E9 (R1) describes the components needed to precisely define the effects to be estimated in clinical trials, which includes how post-baseline "intercurrent" events (IEs) are to be handled. In…

Methodology · Statistics 2023-08-28 Thomas Drury , Juan J Abellan , Nicky Best , Ian R. White

The proximal causal inference framework enables the identification and estimation of causal effects in the presence of unmeasured confounding by leveraging two disjoint sets of observed strong proxies: negative control treatments and…

Methodology · Statistics 2025-12-16 Antonio Olivas-Martinez , Peter B. Gilbert , Andrea Rotnitzky

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

Causal inference has recently garnered significant interest among recommender system (RS) researchers due to its ability to dissect cause-and-effect relationships and its broad applicability across multiple fields. It offers a framework to…

Information Retrieval · Computer Science 2024-07-09 Huishi Luo , Fuzhen Zhuang , Ruobing Xie , Hengshu Zhu , Deqing Wang , Zhulin An , Yongjun Xu

Clinical trials with longitudinal outcomes typically include missing data due to missed assessments or structural missingness of outcomes after intercurrent events handled with a hypothetical strategy. Approaches based on Bayesian random…

Hill's specificity criterion has been highly influential in biomedical and epidemiological research. However, it remains controversial and its application often relies on subjective and qualitative analysis without a comprehensive and…

Methodology · Statistics 2025-06-24 Wang Miao

In biomedical research, repeated measurements within each subject are often processed to remove artifacts and unwanted sources of variation. The resulting data are used to construct derived outcomes that act as proxies for scientific…

Methodology · Statistics 2026-02-03 Zihang Wang , Razieh Nabi , Benjamin B. Risk

We consider the challenges associated with causal inference in settings where data from a randomized trial is augmented with control data from an external source to improve efficiency in estimating the average treatment effect (ATE).…

Causal inference analysis is the estimation of the effects of actions on outcomes. In the context of healthcare data this means estimating the outcome of counter-factual treatments (i.e. including treatments that were not observed) on a…

Methodology · Statistics 2018-03-21 Yishai Shimoni , Chen Yanover , Ehud Karavani , Yaara Goldschmnidt

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

In the quest to make defensible causal claims from observational data, it is sometimes possible to leverage information from "placebo treatments" and "placebo outcomes". Existing approaches employing such information focus largely on point…

Methodology · Statistics 2024-11-26 Adam Rohde , Chad Hazlett

Estimating causal effects is particularly challenging when outcomes arise in complex, non-Euclidean spaces, where conventional methods often fail to capture meaningful structural variation. We develop a framework for topological causal…

Methodology · Statistics 2026-03-04 Kwangho Kim , Hajin Lee
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