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We study the problem of learning to choose from m discrete treatment options (e.g., news item or medical drug) the one with best causal effect for a particular instance (e.g., user or patient) where the training data consists of passive…

Machine Learning · Statistics 2017-08-02 Nathan Kallus

Early prediction of mortality and length of stay(LOS) of a patient is vital for saving a patient's life and management of hospital resources. Availability of electronic health records(EHR) makes a huge impact on the healthcare domain and…

Machine Learning · Computer Science 2020-12-01 Batuhan Bardak , Mehmet Tan

Therapeutic advancements in oncology have shifted towards targeted therapy based on genomic aberrations. This necessitates innovative statistical approaches in clinical trials, particularly in master protocol studies. Basket trials, a type…

Applications · Statistics 2025-02-12 Antonios Daletzakis , Rutger van den Bor , Vincent van der Noort , Kit CB Roes

In precision medicine, Dynamic Treatment Regimes (DTRs) are treatment protocols that adapt over time in response to a patient's observed characteristics. A DTR is a set of decision functions that takes an individual patient's information as…

Methodology · Statistics 2022-03-17 Cong Jiang , Michael Wallace , Mary Thompson

Decision makers, such as doctors and judges, make crucial decisions such as recommending treatments to patients, and granting bails to defendants on a daily basis. Such decisions typically involve weighting the potential benefits of taking…

Machine Learning · Statistics 2016-11-24 Himabindu Lakkaraju , Cynthia Rudin

Design and forecasting of patient enrollment is among the greatest challenges that the clinical research enterprize faces today, as inefficient enrollment can be a major cause of drug development delays. Therefore, the development of the…

Methodology · Statistics 2022-12-27 Vladimir Anisimov , Matthew Austin

There is a constant need for many healthcare programs to timely address problems with infection prevention and control (IP&C). For example, pathogens can be transmitted among patients with cystic fibrosis (CF) in both the inpatient and…

Methodology · Statistics 2022-07-27 Xiaotong Jiang , William Stoudemire , Marianne S. Muhlebach , Michael R. Kosorok

Sequential multiple assignment randomized trials mimic the actual treatment processes experienced by physicians and patients in clinical settings and inform the comparative effectiveness of dynamic treatment regimes. In such trials,…

Methodology · Statistics 2025-04-07 Zi Wang , Yu Cheng , Abdus S. Wahed

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although…

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…

Methodology · Statistics 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

The ICH E9(R1) addendum (2019) proposed principal stratification (PS) as one of five strategies for dealing with intercurrent events. Therefore, understanding the strengths, limitations, and assumptions of PS is important for the broad…

The treatment effect in a specific subgroup is often of interest in randomized clinical trials. When the subgroup is characterized by the absence of certain post-randomization events, a naive analysis on the subset of patients without these…

Applications · Statistics 2018-09-12 Baldur P. Magnusson , Heinz Schmidli , Nicolas Rouyrre , Daniel O. Scharfstein

In many important applications of precision medicine, the outcome of interest is time to an event (e.g., death, relapse of disease) and the primary goal is to identify the optimal individualized decision rule (IDR) to prolong survival time.…

Methodology · Statistics 2022-04-11 Yu Zhou , Lan Wang , Rui Song , Tuoyi Zhao

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall

Longitudinal clinical reasoning over electronic health records requires tracking evolving physiological measurements, laboratory results, and interventions across extended patient trajectories. Existing LLM-based clinical reasoning systems…

Machine Learning · Computer Science 2026-05-27 Zhan Qu , Michael Färber

Phenotyping is the process of distinguishing groups of patients to identify different types of disease progression. A recent trend employs low-rank matrix and tensor factorization methods for their capability of dealing with multi-modal,…

Machine Learning · Computer Science 2025-06-26 Cristian Minoccheri , Sophia Tesic , Kayvan Najarian , Ryan Stidham

Chronic low back pain (cLBP) is a prevalent condition with profound impacts on functioning and quality of life. While multiple evidence-based treatments exist, they all have modest average treatment effects$\unicode{x2013}$potentially due…

Pilot feasibility studies play a pivotal role in the development of clinical trials for rare diseases, where small populations and slow recruitment often threaten trial viability. While such studies are commonly used to assess operational…

Applications · Statistics 2025-07-15 Lara Maleyeff , Valérie Leclair , Shirin Golchi , Marie Hudson

Conventional inclusion criteria used in osteoarthritis clinical trials are not very effective in selecting patients who would benefit from a therapy being tested. Typically majority of selected patients show no or limited disease…

Observational cohort studies are increasingly being used for comparative effectiveness research to assess the safety of therapeutics. Recently, various doubly robust methods have been proposed for average treatment effect estimation by…

Methodology · Statistics 2025-03-11 Xiaoqing Tan , Shu Yang , Wenyu Ye , Douglas E. Faries , Ilya Lipkovich , Zbigniew Kadziola