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Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

We consider methods for transporting a prediction model and assessing its performance for use in a new target population, when outcome and covariate information for model development is available from a simple random sample from the source…

Applications · Statistics 2021-04-15 Jon A. Steingrimsson , Constantine Gatsonis , Issa J. Dahabreh

We calibrate a Natural History Model, which is a class of computer simulator used in the health industry, and here has been used to characterise bowel cancer incidence for the UK. The simulator tracks the development of bowel cancer in a…

Methodology · Statistics 2014-10-29 Jeremy E. Oakley , Benjamin D. Youngman

Weighting estimators based on propensity scores are widely used for causal estimation in a variety of contexts, such as observational studies, marginal structural models and interference. They enjoy appealing theoretical properties such as…

Methodology · Statistics 2021-10-06 Linbo Wang , Yuexia Zhang , Thomas S. Richardson , Xiao-Hua Zhou

Predictive risk scores estimating probabilities for a binary outcome on the basis of observed covariates are common across the sciences. They are frequently developed with the intent of avoiding the outcome in question by intervening in…

Methodology · Statistics 2022-08-26 James Liley

Matching a nonprobability sample to a probability sample is one strategy both for selecting the nonprobability units and for weighting them. This approach has been employed in the past to select subsamples of persons from a large panel of…

Methodology · Statistics 2021-12-03 Zhan Liu , Richard Valliant

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

Cumulative prospect theory (CPT) is known to model human decisions well, with substantial empirical evidence supporting this claim. CPT works by distorting probabilities and is more general than the classic expected utility and coherent…

Machine Learning · Computer Science 2016-03-01 Prashanth L. A. , Cheng Jie , Michael Fu , Steve Marcus , Csaba Szepesvári

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,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman

The case-cohort design allows analysis of multiple endpoints and only requires covariates to be measured for cases and non-cases in a random subcohort from the cohort. Stratification of subcohort sampling and weight calibration increase…

Applications · Statistics 2024-02-15 Lola Etievant , Mitchell H. Gail

Recently developed survival analysis methods improve upon existing approaches by predicting the probability of event occurrence in each of a number pre-specified (discrete) time intervals. By avoiding placing strong parametric assumptions…

Machine Learning · Statistics 2023-10-25 Jimmy Hickey , Ricardo Henao , Daniel Wojdyla , Michael Pencina , Matthew M. Engelhard

Estimating the causal effect of a treatment or health policy with observational data can be challenging due to an imbalance of and a lack of overlap between treated and control covariate distributions. In the presence of limited overlap,…

Methodology · Statistics 2025-03-24 Martha Barnard , Jared D. Huling , Julian Wolfson

The attributable risk, often called the population attributable risk, is in many epidemiological contexts a more relevant measure of exposure-disease association than the excess risk, relative risk, or odds ratio. When estimating…

Statistics Theory · Mathematics 2008-12-31 Daniel B. Rubin

This paper introduces a new basic risk model that could also be utilized by Covid-19 warning apps a priori, before an action is performed. Today the common warning apps estimate risk a posteriori and give no advice on particular scenarios.…

Social and Information Networks · Computer Science 2021-08-06 Jens Braband , Hendrik Schäbe

There is tremendous interest in precision medicine as a means to improve patient outcomes by tailoring treatment to individual characteristics. An individualized treatment rule formalizes precision medicine as a map from patient information…

Machine Learning · Statistics 2020-05-28 Daniel J. Luckett , Eric B. Laber , Michael R. Kosorok

Uncertainty quantification is essential in decision-making, especially when joint distributions of random variables are involved. While conformal prediction provides distribution-free prediction sets with valid coverage guarantees, it…

Machine Learning · Computer Science 2025-01-03 Rui Luo , Zhixin Zhou

Risk prediction models are a crucial tool in healthcare. Risk prediction models with a binary outcome (i.e., binary classification models) are often constructed using methodology which assumes the costs of different classification errors…

In empirical studies with time-to-event outcomes, investigators often leverage observational data to conduct causal inference on the effect of exposure when randomized controlled trial data is unavailable. Model misspecification and lack of…

Methodology · Statistics 2023-05-05 Shenbo Xu , Bang Zheng , Bowen Su , Stan Finkelstein , Roy Welsch , Kenney Ng , Ioanna Tzoulaki , Zach Shahn

Distributional forecasts are important for a wide variety of applications, including forecasting epidemics. Often, forecasts are miscalibrated, or unreliable in assigning uncertainty to future events. We present a recalibration method that…

Machine Learning · Computer Science 2023-01-11 Aaron Rumack , Ryan J. Tibshirani , Roni Rosenfeld

Recurrent events are common and important clinical trial endpoints in many disease areas, e.g., cardiovascular hospitalizations in heart failure, relapses in multiple sclerosis, or exacerbations in asthma. During a trial, patients may…

Methodology · Statistics 2026-04-08 Jiren Sun , Tobias Mutze , Richard Cook , Tianmeng Lyu