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The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

Comparing the survival times among two groups is a common problem in time-to-event analysis, for example if one would like to understand whether one medical treatment is superior to another. In the standard survival analysis setting, there…

Methodology · Statistics 2023-07-07 Dennis Dobler , Eni Musta

Estimating the effects of interventions on patient outcome is one of the key aspects of personalized medicine. Their inference is often challenged by the fact that the training data comprises only the outcome for the administered treatment,…

Breakthroughs in cancer biology have defined new research programs emphasizing the development of therapies that target specific pathways in tumor cells. Innovations in clinical trial design have followed with master protocols defined by…

Methodology · Statistics 2020-07-09 Alexander M. Kaizer , Joseph S. Koopmeiners , Nan Chen , Brian P. Hobbs

In this paper, we propose the use of causal inference techniques for survival function estimation and prediction for subgroups of the data, upto individual units. Tree ensemble methods, specifically random forests were modified for this…

Econometrics · Economics 2018-03-23 Vikas Ramachandra

Adaptive designs are commonly used in clinical and drug development studies for optimum utilization of available resources. In this article, we consider the problem of estimating the effect of the selected (better) treatment using a…

Statistics Theory · Mathematics 2023-01-24 Masihuddin , Neeraj Misra

With the development of novel therapies such as molecularly targeted agents and immunotherapy, the maximum tolerated dose paradigm that "more is better" does not necessarily hold anymore. In this context, doses and schedules of novel…

Oversubscribed treatments are often allocated using randomized waiting lists. Applicants are ranked randomly, and treatment offers are made following that ranking until all seats are filled. To estimate causal effects, researchers often…

Methodology · Statistics 2018-10-24 Clement de Chaisemartin , Luc Behaghel

The interpretation of randomised clinical trial results is often complicated by intercurrent events. For instance, rescue medication is sometimes given to patients in response to worsening of their disease, either in addition to the…

Methodology · Statistics 2021-02-12 Hege Michiels , Cristina Sotto , An Vandebosch , Stijn Vansteelandt

Surgery planning in patients diagnosed with brain tumor is dependent on their survival prognosis. A poor prognosis might demand for a more aggressive treatment and therapy plan, while a favorable prognosis might enable a less risky surgery…

Image and Video Processing · Electrical Eng. & Systems 2020-09-08 Sobia Yousaf , Syed Muhammad Anwar , Harish RaviPrakash , Ulas Bagci

Immunotherapy emerges as promising approach for treating cancer. Encouraging findings have validated the efficacy of immunotherapy medications in addressing tumors, resulting in prolonged survival rates and notable reductions in toxicity…

Image and Video Processing · Electrical Eng. & Systems 2023-07-28 Francesco Rundo , Concetto Spampinato , Michael Rundo

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Despite the massive costs and widespread harms of substance use, most individuals with substance use disorders (SUDs) receive no treatment at all. Digital therapeutics platforms are an emerging low-cost and low-barrier means of extending…

Systems and Control · Electrical Eng. & Systems 2025-04-03 Eric Pulick , Yonatan Mintz

This paper proposes a general framework for probabilistic certification of cancer therapies. The certification is defined in terms of two key issues which are the tumor contraction and the lower admissible bound on the circulating…

Systems and Control · Computer Science 2015-02-24 Mazen Alamir

Dynamic treatment regimes have been proposed to personalize treatment decisions by utilizing historical patient data, but they may not always improve on the current standard of care. It is thus meaningful to integrate the standard of care…

Applications · Statistics 2025-12-11 Johannes Hruza , Arvid Sjölander , Erin Gabriel , Samir Bhatt , Michael Sachs

Tumor Treating Fields (TTFields) is an FDA approved treatment for specific types of cancer and significantly extends patients life. The intensity of the TTFields within the tumor was associated with the treatment outcomes: the larger the…

Computer Vision and Pattern Recognition · Computer Science 2020-10-07 Reuben R Shamir , Zeev Bomzon

It has recently become popular to define treatment effects for subsets of the target population characterized by variables not observable at the time a treatment decision is made. Characterizing and estimating such treatment effects is…

Statistics Theory · Mathematics 2007-08-30 Marshall M. Joffe , Dylan Small , Chi-Yuan Hsu

Radiation therapy (RT) is a medical treatment to kill cancer cells or shrink tumors. To manually schedule patients for RT is a time-consuming and challenging task. By the use of optimization, patient schedules for RT can be created…

Optimization and Control · Mathematics 2023-05-04 Sara Frimodig , Per Enqvist , Mats Carlsson , Carole Mercier

In cancer epidemiology using population-based data, regression models for the excess mortality hazard is a useful method to estimate cancer survival and to describe the association between prognosis factors and excess mortality. This method…

Methodology · Statistics 2019-04-19 Francisco J. Rubio , Bernard Rachet , Roch Giorgi , Camille Maringe , Aurelien Belot

Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…

Methodology · Statistics 2021-03-24 Andrew Ying , Eric J. Tchetgen Tchetgen
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