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相关论文: Protocols for Observational Studies: Methods and O…

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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

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

统计方法学 · 统计学 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

机器学习 · 计算机科学 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

In this paper we present tools for applied researchers that re-purpose off-the-shelf methods from the computer-science field of machine learning to create a "discovery engine" for data from randomized controlled trials (RCTs). The applied…

机器学习 · 统计学 2019-05-13 Jens Ludwig , Sendhil Mullainathan , Jann Spiess

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

统计方法学 · 统计学 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

统计方法学 · 统计学 2019-02-27 Anthony D. Scotina , Roee Gutman

Assessing the causal effects of interventions on ordinal outcomes is an important objective of many educational and behavioral studies. Under the potential outcomes framework, we can define causal effects as comparisons between the…

统计方法学 · 统计学 2018-04-06 Jiannan Lu , Peng Ding , Tirthankar Dasgupta

There has been an increasing use of master protocols in oncology clinical trials because of its efficiency and flexibility to accelerate cancer drug development. Depending on the study objective and design, a master protocol trial can be a…

应用统计 · 统计学 2023-11-13 Jie Chen , Xiaoyun , Li , Chengxing , Lu , Sammy Yuan , Godwin Yung , Jingjing Ye , Hong Tian , Jianchang Lin

Time plays a fundamental role in causal analyses, where the goal is to quantify the effect of a specific treatment on future outcomes. In a randomized experiment, times of treatment, and when outcomes are observed, are typically well…

统计方法学 · 统计学 2016-01-19 Guillaume W. Basse , Alexander Volfovsky , Edoardo M. Airoldi

American football is the most popular high school sport and is among the leading cause of injury among adolescents. While there has been considerable recent attention on the link between football and cognitive decline, there is also…

应用统计 · 统计学 2019-02-27 Timothy G. Gaulton , Sameer K. Deshpande , Dylan S. Small , Mark D. Neuman

Clinical trials usually target average treatment effects, but treatment decisions are made for individuals. This tension motivates a common criticism of evidence-based medicine: a treatment that is beneficial on average may be inappropriate…

应用统计 · 统计学 2026-05-29 Zach Shahn , Mats Stensrud

This project considers how one might augment a limited amount of data from randomized controlled trial (RCT) with more plentiful data from an observational database (ODB), in order to estimate a causal effect. In our motivating setting, the…

统计方法学 · 统计学 2018-10-23 Evan Rosenman , Art B. Owen , Michael Baiocchi , Hailey Banack

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

统计方法学 · 统计学 2022-02-08 Melody Huang

A randomized trial allows estimation of the causal effect of an intervention compared to a control in the overall population and in subpopulations defined by baseline characteristics. Often, however, clinical questions also arise regarding…

Randomized clinical trials are considered the gold standard for informing treatment guidelines, but results may not generalize to real-world populations. Generalizability is hindered by distributional differences in baseline covariates and…

统计方法学 · 统计学 2025-06-03 Rachael K. Ross , Ivan Diaz , Amy J. Pitts , Elizabeth A. Stuart , Kara E. Rudolph

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

统计方法学 · 统计学 2022-11-10 Wenqi Shi , Xi Lin

Inferring causal effects from an observational study is challenging because participants are not randomized to treatment. Observational studies in infectious disease research present the additional challenge that one participant's treatment…

统计方法学 · 统计学 2020-12-25 Brian G. Barkley , Michael G. Hudgens , John D. Clemens , Mohammad Ali , Michael E. Emch

When longitudinal outcomes are evaluated in mortal populations, their non-existence after death complicates the analysis and its causal interpretation. Where popular methods often merge longitudinal outcome and survival into one scale or…

Randomized Controlled Trials (RCTs) are the gold standard for evaluating the effect of new medical treatments. Treatments must pass stringent regulatory conditions in order to be approved for widespread use, yet even after the regulatory…

机器学习 · 统计学 2025-03-13 Omer Noy Klein , Alihan Hüyük , Ron Shamir , Uri Shalit , Mihaela van der Schaar

We study causal inference under case-control and case-population sampling. Specifically, we focus on the binary-outcome and binary-treatment case, where the parameters of interest are causal relative and attributable risks defined via the…

计量经济学 · 经济学 2023-10-24 Sung Jae Jun , Sokbae Lee