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Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

统计计算 · 统计学 2016-06-28 Marius Thomas , Björn Bornkamp

Large-scale models require substantial computational resources for analysis and studying treatment conditions. Specifically, estimating treatment effects using simulations may require a lot of infeasible resources to allocate at every…

多智能体系统 · 计算机科学 2023-08-28 Abdulrahman A. Ahmed , M. Amin Rahimian , Mark S. Roberts

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…

统计方法学 · 统计学 2023-01-23 Lars Lau Raket

Empirical work often uses treatment assigned following geographic boundaries. When the effects of treatment cross over borders, classical difference-in-differences estimation produces biased estimates for the average treatment effect. In…

计量经济学 · 经济学 2023-06-13 Kyle Butts

In observational studies, weighting methods that directly optimize the balance between treatment and covariates have received much attention lately; however these have mainly focused on binary treatments. Inspired by domain adaptation, we…

统计理论 · 数学 2020-02-27 Guillaume Martinet

In many medical and business applications, researchers are interested in estimating individualized treatment effects using data from a randomized experiment. For example in medical applications, doctors learn the treatment effects from…

统计方法学 · 统计学 2022-03-01 Kevin Wu Han , Han Wu

In observational studies, balancing covariates in different treatment groups is essential to estimate treatment effects. One of the most commonly used methods for such purposes is weighting. The performance of this class of methods usually…

统计方法学 · 统计学 2021-07-07 Ruoqi Yu , Shulei Wang

Multivalued treatment models have typically been studied under restrictive assumptions: ordered choice, and more recently unordered monotonicity. We show how treatment effects can be identified in a more general class of models that allows…

计量经济学 · 经济学 2018-05-02 Sokbae Lee , Bernard Salanié

We introduce causal inference reasoning to cross-over trials, with a focus on Thorough QT (TQT) studies. For such trials, we propose different sets of assumptions and consider their impact on the modelling strategy and estimation procedure.…

统计方法学 · 统计学 2022-05-31 Jeppe Ekstrand Halkjær Madsen , Thomas Scheike , Christian Pipper

As cancer patient survival improves, late effects from treatment are becoming the next clinical challenge. Chemotherapy and radiotherapy, for example, potentially increase the risk of both morbidity and mortality from second malignancies…

This paper develops a unified theoretical framework for detecting and estimating boundaries in treatment effects across both spatial and temporal dimensions. We formalize the concept of treatment effect boundaries as structural parameters…

计量经济学 · 经济学 2025-10-28 Tatsuru Kikuchi

Crossover designs randomly assign each unit to receive a sequence of treatments. By comparing outcomes within the same unit, these designs can effectively eliminate between-unit variation and facilitate the identification of both…

统计方法学 · 统计学 2026-04-21 Zhichao Jiang , Peng Ding

Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…

统计方法学 · 统计学 2021-03-24 Andrew Ying , Eric J. Tchetgen Tchetgen

A multistate cure model is a statistical framework used to analyze and represent the transitions individuals undergo between different states over time, accounting for the possibility of being cured by initial treatment. This model is…

统计方法学 · 统计学 2024-12-02 Yilin Jiang , Harm van Tinteren , Marta Fiocco

Intensity-based multistate models provide a useful framework for characterizing disease processes, the introduction of interventions, loss to follow-up, and other complications arising in the conduct of randomized trials studying complex…

统计方法学 · 统计学 2022-09-29 Alexandra Bühler , Richard J. Cook , Jerald F. Lawless

Many applications in medical statistics as well as in other fields can be described by transitions between multiple states (e.g. from health to disease) experienced by individuals over time. In this context, multi-state models are a popular…

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

This work proposes a statistical model for crossover trials with multiple skewed responses measured in each period. A 3 $\times$ 3 crossover trial data where different drug doses were administered to subjects with a history of seasonal…

统计方法学 · 统计学 2026-04-09 Savita Pareek , Kalyan Das , Siuli Mukhopadhyay

Clinical trials involving novel immuno-oncology (IO) therapies frequently exhibit survival profiles which violate the proportional hazards assumption due to a delay in treatment effect, and in such settings, the survival curves in the two…

统计方法学 · 统计学 2021-02-02 Nicholas C. Henderson , Kijoeng Nam , Dai Feng

We study a continuous treatment effect model in the presence of treatment spillovers through social networks. We assume that one's outcome is affected not only by his/her own treatment but also by a (weighted) average of his/her neighbors'…

计量经济学 · 经济学 2023-01-12 Tadao Hoshino
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