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When evaluating the effectiveness of a drug, a Randomized Controlled Trial (RCT) is often considered the gold standard due to its perfect randomization. While RCT assures strong internal validity, its restricted external validity poses…

Applications · Statistics 2024-06-07 Kuan Jiang , Xin-xing Lai , Shu Yang , Ying Gao , Xiao-Hua Zhou

In traditional dose-finding studies, dose-limiting toxicity (DLT) is determined within a fixed time observation window where DLT is often defined as a binary outcome. In the setting of oncology dose-finding trials, often patients in…

Applications · Statistics 2019-09-09 Lucie Biard , Bin Cheng , Gulam A. Manji , Shing M. Lee

Background: Randomized controlled trials (RCTs) are costly, time-consuming, and often infeasible, while treatment-effect estimation from observational data is limited by unobserved confounding. Methods: We developed a three-step framework…

We propose a deep learning model - Probabilistic Prognostic Estimates of Survival in Metastatic Cancer Patients (PPES-Met) for estimating short-term life expectancy (3 months) of the patients by analyzing free-text clinical notes in the…

Artificial Intelligence · Computer Science 2018-07-17 Imon Banerjee , Michael Francis Gensheimer , Douglas J. Wood , Solomon Henry , Daniel Chang , Daniel L. Rubin

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

Methodology · Statistics 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

The ISCHEMIA Trial randomly assigned patients with ischemic heart disease to an invasive treatment strategy centered on revascularization with a control group assigned non-invasive medical therapy. As is common in such ``strategy trials,''…

Econometrics · Economics 2025-01-06 Joshua D. Angrist , Bruno Ferman , Carol Gao , Peter Hull , Otavio L. Tecchio , Robert W. Yeh

We present a general approach to synthesizing evidence of time-to-event endpoints in meta-analyses of aggregate data (AD). Our work goes beyond most previous meta-analytic research by using reconstructed survival data as a source of…

Massive research efforts are now underway to develop a cure for HIV infection, allowing patients to discontinue lifelong combination antiretroviral therapy (ART). New latency-reversing agents (LRAs) may be able to purge the persistent…

Populations and Evolution · Quantitative Biology 2014-08-07 Alison L. Hill , Daniel I. S. Rosenbloom , Feng Fu , Martin A. Nowak , Robert F. Siliciano

BACKGROUND: Atrial fibrillation (AF), the most common arrhythmia, is linked to high morbidity and mortality. In a fast-evolving AF rhythm control treatment era, predicting AF recurrence after its onset may be crucial to achieve the optimal…

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

Applications · Statistics 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

Randomized experiments are the gold standard for causal inference, and justify simple comparisons across treatment groups. Regression adjustment provides a convenient way to incorporate covariate information for additional efficiency. This…

Methodology · Statistics 2022-10-25 Anqi Zhao , Peng Ding

Objective: Subcutaneous Immunotherapy (SCIT) is the long-lasting causal treatment of allergic rhinitis (AR). How to enhance the adherence of patients to maximize the benefit of allergen immunotherapy (AIT) plays a crucial role in the…

Machine Learning · Computer Science 2024-07-22 Yin Li , Yu Xiong , Wenxin Fan , Kai Wang , Qingqing Yu , Liping Si , Patrick van der Smagt , Jun Tang , Nutan Chen

We investigate estimation of causal effects of multiple competing (multi-valued) treatments in the absence of randomization. Our work is motivated by an intention-to-treat study of the relative cardiometabolic risk of assignment to one of…

Readmissions among Medicare beneficiaries are a major problem for the US healthcare system from a perspective of both healthcare operations and patient caregiving outcomes. Our study analyzes Medicare hospital readmissions using LSTM…

Machine Learning · Computer Science 2024-10-24 Xintao Li , Sibei Liu , Dezhi Yu , Yang Zhang , Xiaoyu Liu

Managing physiological variables within clinically safe target zones is a central challenge in healthcare, particularly for chronic conditions such as Type 1 Diabetes Mellitus (T1DM). Reinforcement learning (RL) offers promise for…

Machine Learning · Computer Science 2025-08-07 David H. Mguni , Jing Dong , Wanrong Yang , Ziquan Liu , Muhammad Salman Haleem , Baoxiang Wang

The paper investigates the escalating concerns surrounding the surge in diabetes cases, exacerbated by the COVID-19 pandemic, and the subsequent strain on medical resources. The research aims to construct a predictive model quantifying…

Applications · Statistics 2025-02-04 Jorden Lam , Kunpeng Xu

Clinical trials are common in medical research where multiple non-Gaussian responses and time-dependent observations are frequent. The analysis of data from these studies requires statistical modeling techniques that take these…

In Randomised Controlled Trials (RCT) with treatment non-compliance, instrumental variable approaches are used to estimate complier average causal effects. We extend these approaches to cost-effectiveness analyses, where methods need to…

Methodology · Statistics 2016-12-02 Karla DiazOrdaz , Angelo Franchini , Richard Grieve

The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

Signal Processing · Electrical Eng. & Systems 2023-05-10 Sayeri Lala , Niraj K. Jha