English
Related papers

Related papers: Adjustment for Inconsistency in Adaptive Phase 2/3…

200 papers

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Purpose: Two-stage single-arm trial designs are commonly used in phase II oncology to infer treatment effects for a binary primary outcome (e.g., tumour response). It is imperative that such studies be designed, analysed, and reported…

Applications · Statistics 2020-07-09 Michael J. Grayling , Adrian P. Mander

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

We consider the effects of parameter uncertainty on the optimal radiation schedule in the context of the linear-quadratic model. Our interest arises from the observation that if inter-patient variations in OAR and tumor sensitivities to…

Tissues and Organs · Quantitative Biology 2015-06-05 Hamidreza Badri , Yoichi Watanabe , Kevin Leder

The analysis of screening experiments is often done in two stages, starting with factor selection via an analysis under a main effects model. The success of this first stage is influenced by three components: (1) main effect estimators'…

Methodology · Statistics 2024-03-19 Jonathan W. Stallrich , Michael McKibben

This paper studies a two-stage model of experimentation, where the researcher first samples representative units from an eligible pool, then assigns each sampled unit to treatment or control. To implement balanced sampling and assignment,…

Econometrics · Economics 2023-08-22 Max Cytrynbaum

Nowadays, more and more clinical trials choose combinational agents as the intervention to achieve better therapeutic responses. However, dose-finding for combinational agents is much more complicated than single agent as the full order of…

Applications · Statistics 2022-08-05 Shu Wang , Ji-Hyun Lee

Antimicrobial resistance is a threat to public health with millions of deaths linked to drug resistant infections every year. To mitigate resistance, common strategies that are used are combination therapies and therapy switching. However,…

Populations and Evolution · Quantitative Biology 2025-06-02 Juan Magalang , Javier Aguilar , Jose Perico Esguerra , Édgar Roldán , Daniel Sanchez-Taltavull

In observational studies of treatment effects, estimates may be biased by unmeasured confounders, which can potentially affect the validity of the results. Understanding sensitivity to such biases helps assess how unmeasured confounding…

Methodology · Statistics 2026-02-02 Qishuo Yin , Dylan S. Small

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

Methodology · Statistics 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Medical image analysis relies on accurate segmentation, and benefits from controllable synthesis (of new training images). Yet both tasks of the cyclical pipeline face spatial imbalance: lesions occupy small regions against vast…

Computer Vision and Pattern Recognition · Computer Science 2026-03-06 Anugunj Naman , Ayushman Singh , Gaibo Zhang , Yaguang Zhang

We construct optimal designs for group testing experiments where the goal is to estimate the prevalence of a trait by using a test with uncertain sensitivity and specificity. Using optimal design theory for approximate designs, we show that…

Statistics Theory · Mathematics 2017-01-05 Shih-Hao Huang , Mong-Na Lo Huang , Kerby Shedden , Weng Kee Wong

In many applied optimization settings, parameters that define the constraints may not guarantee the best possible solution, and superior solutions might exist that are infeasible for the given parameter values. Removing such constraints,…

Optimization and Control · Mathematics 2024-07-22 Farzin Ahmadi , Todd R. McNutt , Kimia Ghobadi

Transitioning from Phase 2 to Phase 3 in drug development, at a rate of $\approx$40%, is the most stringent among phase transitions (Hay et al. (2014)). Yet, success rate at Phase 3 leading to approval is only $\approx$50% (Arrowsmith…

Methodology · Statistics 2025-10-29 Yujia Sun , Yang Han , Xingya Wang , Szu-Yu Tang , Yushi Liu , Jason C. Hsu

Given a fixed-sample-size test that controls the error probabilities under two specific, but arbitrary, distributions, a 3-stage and two 4-stage tests are proposed and analyzed. For each of them, a novel, concrete, non-asymptotic,…

Statistics Theory · Mathematics 2022-06-27 Yiming Xing , Georgios Fellouris

One of the primary drivers for self-adaptation is ensuring that systems achieve their goals regardless of the uncertainties they face during operation. Nevertheless, the concept of uncertainty in self-adaptive systems is still…

Software Engineering · Computer Science 2021-03-05 Sara M. Hezavehi , Danny Weyns , Paris Avgeriou , Radu Calinescu , Raffaela Mirandola , Diego Perez-Palacin

Large-scale classification of data where classes are structurally organized in a hierarchy is an important area of research. Top-down approaches that exploit the hierarchy during the learning and prediction phase are efficient for large…

Machine Learning · Computer Science 2017-06-06 Azad Naik , Huzefa Rangwala

The dose delivered to the planning target volume by proton beams is highly conformal, sparing organs at risk and normal tissues. New treatment planning systems adapted to spot scanning techniques have been recently proposed to…

Medical Physics · Physics 2022-05-18 François Smekens , Nicolas Freud , Bruno Sixou , Guillaume Beslon , Jean M Létang

Efforts to develop biomarker-targeted anti-cancer therapies have progressed rapidly in recent years. Six antibodies acting on programmed death ligand 1 or programmed death 1 pathways were approved in 75 cancer indications between 2015 and…

Methodology · Statistics 2022-10-07 Emily C. Zabor , Alexander M. Kaizer , Nathan A. Pennell , Brian P. Hobbs

Group sequential designs in clinical trials allow for interim efficacy and futility monitoring. Adjustment for baseline covariates can increase power and precision of estimated effects. However, inconsistently applying covariate adjustment…

Methodology · Statistics 2023-08-11 Marlena S. Bannick , Sonya L. Heltshe , Noah Simon