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Given the cost and duration of phase III and phase IV clinical trials, the development of statistical methods for go/no-go decisions is vital. In this paper, we introduce a Bayesian methodology to compute the probability of success based on…

Methodology · Statistics 2020-10-27 Ethan M. Alt , Matthew A. Psioda , Joseph G. Ibrahim

Clinical decision making regarding treatments based on personal characteristics leads to effective health improvements. Machine learning (ML) has been the primary concern of diagnosis support according to comprehensive patient information.…

Machine Learning · Computer Science 2021-06-09 Kazuki Nakamura , Ryosuke Kojima , Eiichiro Uchino , Koichi Murashita , Ken Itoh , Shigeyuki Nakaji , Yasushi Okuno

In many causal studies, outcomes are censored by death, in the sense that they are neither observed nor defined for units who die. In such studies, the focus is usually on the stratum of always survivors up to a single fixed time s.…

Methodology · Statistics 2024-01-02 Giulio Grossi , Marco Mariani , Alessandra Mattei , Fabrizia Mealli

Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events (AEs)…

Methodology · Statistics 2024-10-08 Anthony Sisti , Andrew Zullo , Roee Gutman

Most conventional risk analysis methods rely on a single best estimate of exposure per person which does not allow for adjustment for exposure-related uncertainty. Here, we propose a Bayesian model averaging method to properly quantify the…

Applications · Statistics 2020-04-07 Deukwoo Kwon , F. Owen Hoffman , Brian E. Moroz , Steven L. Simon

Bayesian nonparametric methods are a popular choice for analysing survival data due to their ability to flexibly model the distribution of survival times. These methods typically employ a nonparametric prior on the survival function that is…

Methodology · Statistics 2022-02-22 Edwin Fong , Brieuc Lehmann

The inclusion of the propensity score as a covariate in Bayesian regression trees for causal inference can reduce the bias in treatment effect estimations, which occurs due to the regularization-induced confounding phenomenon. This study…

Methodology · Statistics 2018-08-30 Pedro Henrique Filipini dos Santos , Hedibert Freitas Lopes

Disparity in suicide rates between urban and suburban/rural areas is growing, with rural areas typically witnessing higher suicide rates in the U.S. However, previous studies often ignored the effect of socio-environmental factors on the…

Applications · Statistics 2022-01-19 Sayanti Mukherjee , Zhiyuan Wei

Principal stratification analysis evaluates how causal effects of a treatment on a primary outcome vary across strata of units defined by their treatment effect on some intermediate quantity. This endeavor is substantially challenged when…

Methodology · Statistics 2024-03-21 Chanmin Kim , Corwin Zigler

In studying the marginal effect of antidepressants on body mass index using electronic health records data, we face several challenges. Patients' characteristics can affect the exposure (confounding) as well as the timing of routine visits…

Applications · Statistics 2021-06-29 Janie Coulombe , Erica E. M. Moodie , Robert W. Platt , Christel Renoux

In heterogeneous cohorts and those where censoring by non-primary risks is informative many conventional survival analysis methods are not applicable; the proportional hazards assumption is usually violated at population level and the…

This study aimed to systematically identify and quantify risks for drug-induced rhabdomyolysis (DIR) using real-world data and to propose an evidence-based risk mitigation framework. We conducted a retrospective pharmacovigilance study…

Other Quantitative Biology · Quantitative Biology 2025-11-04 Enpu Liang

Ecological Momentary Assessment provides real-time data on suicidal thoughts and behaviors, but predicting suicide attempts remains challenging due to their rarity and patient heterogeneity. We show that single models fit to all patients…

Machine Learning · Statistics 2025-11-25 Genesis Hang , Annie Chen , Hope Neveux , Matthew K. Nock , Yaniv Yacoby

The dearth of prescribing guidelines for physicians is one key driver of the current opioid epidemic in the United States. In this work, we analyze medical and pharmaceutical claims data to draw insights on characteristics of patients who…

Machine Learning · Computer Science 2020-05-01 Chirag Nagpal , Dennis Wei , Bhanukiran Vinzamuri , Monica Shekhar , Sara E. Berger , Subhro Das , Kush R. Varshney

Assessing causal effects in the presence of unobserved confounding is a challenging problem. Existing studies leveraged proxy variables or multiple treatments to adjust for the confounding bias. In particular, the latter approach attributes…

Methodology · Statistics 2023-10-17 Yong Wu , Mingzhou Liu , Jing Yan , Yanwei Fu , Shouyan Wang , Yizhou Wang , Xinwei Sun

This paper presents a novel nonlinear regression model for estimating heterogeneous treatment effects from observational data, geared specifically towards situations with small effect sizes, heterogeneous effects, and strong confounding.…

Methodology · Statistics 2019-11-14 P. Richard Hahn , Jared S. Murray , Carlos Carvalho

Statistical agencies and other institutions collect data under the promise to protect the confidentiality of respondents. When releasing microdata samples, the risk that records can be identified must be assessed. To this aim, a widely…

Applications · Statistics 2015-06-03 Cinzia Carota , Maurizio Filippone , Roberto Leombruni , Silvia Polettini

Learning the structure of Bayesian networks from data provides insights into underlying processes and the causal relationships that generate the data, but its usefulness depends on the homogeneity of the data population, a condition often…

It is often of interest in observational studies to measure the causal effect of a treatment on time-to-event outcomes. In a medical setting, observational studies commonly involve patients who initiate medication therapy and others who do…

Methodology · Statistics 2019-10-07 Reagan Mozer , Mark E. Glickman

Drug overdose deaths, including from opioids, remain a significant public health threat to the United States (US). To abate the harms of opioid misuse, understanding its prevalence at the local level is crucial for stakeholders in…

Applications · Statistics 2026-01-09 Zixuan Feng , Qiushi Chen , Paul Griffin , Le Bao
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