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This paper addresses patient heterogeneity associated with prediction problems in biomedical applications. We propose a systematic hypothesis testing approach to determine the existence of patient subgroup structure and the number of…

Methodology · Statistics 2021-01-08 Xu Gao , Weining Shen , Jing Ning , Ziding Feng , Jianhua Hu

Modern causal inference methods allow machine learning to be used to weaken parametric modeling assumptions. However, the use of machine learning may result in complications for inference. Doubly-robust cross-fit estimators have been…

Methodology · Statistics 2022-03-11 Paul N Zivich , Alexander Breskin

Treatment effect heterogeneity occurs when individual characteristics influence the effect of a treatment. We propose a novel approach that combines prognostic score matching and conditional inference trees to characterize effect…

Selection bias is pervasive in observational studies. For example, large scale biobanks data can exhibit ``healthy volunteer bias'' when respondents are healthier and of higher socio-economic status than the population they are meant to…

Methodology · Statistics 2026-05-14 Yiwen Qiu , Filip Kovacevic , Shimeng Huang , Peter Spirtes , Francesco Locatello

The principal stratification has become a popular tool to address a broad class of causal inference questions, particularly in dealing with non-compliance and truncation-by-death problems. The causal effects within principal strata which…

Methodology · Statistics 2022-06-20 Shanshan Luo , Wei Li , Wang Miao , Yangbo He

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Confounding bias and selection bias bring two significant challenges to the validity of conclusions drawn from applied causal inference. The latter can stem from informative missingness, such as in cases of attrition. We introduce the…

Methodology · Statistics 2025-03-25 Johan de Aguas , Johan Pensar , Tomás Varnet Pérez , Guido Biele

Tomasetti and Vogelstein (2015) collected data on 31 different tissue types and found a correlation of 0.8 between the logarithms of the incidence of cancer (LCI), and the estimated number of stem cell divisions in those tissues (LSCD).…

Quantitative Methods · Quantitative Biology 2015-01-20 Lee Altenberg

Physically, disordered ensembles of non-homopolymeric polypeptides are expected to be heterogeneous; i.e., they should differ from those homogeneous ensembles of homopolymers that harbor an essentially unique relationship between average…

Biomolecules · Quantitative Biology 2021-06-25 Jianhui Song , Jichen Li , Hue Sun Chan

We present BayesPIM, a Bayesian prevalence-incidence mixture model for estimating time- and covariate-dependent disease incidence from screening and surveillance data. The method is particularly suited to settings where some individuals may…

Methodology · Statistics 2026-01-12 Thomas Klausch , Birgit I. Lissenberg-Witte , Veerle M. Coupé

The participants in randomized trials and other studies used for causal inference are often not representative of the populations seen by clinical decision-makers. To account for differences between populations, researchers may consider…

Methodology · Statistics 2022-07-12 Anders Huitfeldt , Sonja A. Swanson , Mats Julius Stensrud , Etsuji Suzuki

Causal inference in the presence of intermediate variables is a challenging problem in many applications. Principal stratification (PS) provides a framework to estimate principal causal effects (PCE) in such settings. However, existing PS…

Methodology · Statistics 2026-02-20 Lei Yang , Michael J. Daniels , Fan Li

Environmental epidemiologists are increasingly interested in establishing causality between exposures and health outcomes. A popular model for causal inference is the Rubin Causal Model (RCM), which typically seeks to estimate the average…

Applications · Statistics 2021-01-26 Keith W. Zirkle , Marie-Abele Bind , Jenise L. Swall , David C. Wheeler

Several explanations can be found in the literature about the origin of colorectal cancer. There is however some agreement on the fact that the carcinogenic process is a result of several genetic mutations of normal cells. The colon…

Analysis of PDEs · Mathematics 2016-03-08 Isabel N. Figueiredo , Carlos Leal , Giuseppe Romanazzi , Bjorn Engquist

The heterogeneous treatment effect plays a crucial role in precision medicine.There is evidence that real-world data, even subject to biases, can be employed as supplementary evidence for randomized clinical trials to improve the…

Methodology · Statistics 2025-09-04 Guangcai Mao , Shu Yang , Xiaofei Wang

Cancer is a complex disease with significant social and economic impact. Advancements in high-throughput molecular assays and the reduced cost for performing high-quality multi-omics measurements have fuelled insights through machine…

Machine Learning · Computer Science 2022-06-28 Pedro Henrique da Costa Avelar , Roman Laddach , Sophia Karagiannis , Min Wu , Sophia Tsoka

Unobserved individual heterogeneity is a common challenge in population cancer survival studies. This heterogeneity is usually associated with the combination of model misspecification and the failure to record truly relevant variables. We…

Methodology · Statistics 2023-01-06 F. J. Rubio , H. Putter , A. Belot

Recent studies of cancer somatic mutation profiles seek to identify mutations for targeted therapy in personalized medicine. Analysis of profiles, however, is not trivial, as each profile is heterogeneous and there are multiple confounding…

Machine Learning · Statistics 2020-05-25 Rahul Mehta , Muge Karaman

While we once thought of cancer as single monolithic diseases affecting a specific organ site, we now understand that there are many subtypes of cancer defined by unique patterns of gene mutations. These gene mutational data, which can be…

Quantitative Methods · Quantitative Biology 2017-03-07 Jipeng Qiang , Wei Ding , John Quackenbush , Ping Chen

Clinical studies sometimes encounter truncation by death, rendering outcomes undefined. Statistical analysis based solely on observed survivors may give biased results because the characteristics of survivors differ between treatment…

Methodology · Statistics 2022-11-23 Yuhao Deng , Yingjun Chang , Xiao-Hua Zhou
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