23 papers · ranked by Valyu relevance
Pavlos Msaouel, Juhee Lee, Peter F. Thall, Alan Hutson
Simple Summary We provide an extensive review of the fundamental principles of statistical science that are needed to accurately interpret randomized controlled trials (RCTs). We use these principles to explain how RCTs are motivated by the powerful but strange idea that flipping a coin to choose each patient’s…
Kerstine Carter, Olga Kuznetsova, Volodymyr Anisimov, Johannes Krisam + 3 more
Background When running a randomized controlled trial (RCT), a clinical site may face a situation when an eligible trial participant is to be randomized to the treatment that is not available at the site. In this case, there are two options: not to enroll the participant, or, without disclosing to the site, allocate…
Yao Zhang, Qingyuan Zhao
The meaning of randomization tests has become obscure in statistics education and practice over the last century. This article makes a fresh attempt at rectifying this core concept of statistics. A new term—"quasi-randomization test"—is introduced to define significance tests based on theoretical models and distinguish…
Yevgen Ryeznik, Oleksandr Sverdlov
Designs Authors: ['Yevgen Ryeznik' 'Oleksandr Sverdlov'] In this paper, we present Insertus.jl, the Julia package that can help the user generate a randomization sequence of a given length for a multi-arm trial with a pre-specified target allocation ratio and assess the operating characteristics of the chosen…
Issa J Dahabreh, Sarah E. Robertson, Miguel A. Hernán
We discuss the identifiability of causal estimands for generalizability and transportability analyses, both under perfect and imperfect adherence to treatment assignment. We consider a setting where the trial data contain information on baseline covariates, assignment at baseline, intervention at baseline (point…
Robert Mash, Bolatito B. Fatusin, Edith Madela-Mntla, Christopher Butler
'Christopher Butler'] This article outlines the essential features of clinical trials for doctoral or early career researchers. The World Health Organization has recently emphasised the need for higher quality clinical trials, more trials from low- and middle-income countries, as well as primary care, more engagement…
Sheng Chen, Wei Wang
Background Randomized Controlled Trial is the most rigorous study design to test the efficacy and effectiveness of an intervention. Patient preference may negatively affect patient performance and decrease the generalizability of a trial to clinical population. Patient preference trial have particular implications in…
Yi Qian, Yifan Yi, Jun Shao, Yanyao Yi + 3 more
Trials Authors: ['Yi Qian' 'Yifan Yi' 'Jun Shao' 'Yanyao Yi' 'Nicole Mayer-Hamblett' 'Patrick J. Heagerty' 'Ting Ye'] A platform trial is an innovative clinical trial design that uses a master protocol (i.e., one overarching protocol) to evaluate multiple treatments in an ongoing manner and can accelerate the…
Isabel Chien, Nina Deliu, Richard E. Turner, Adrian Weller + 2 more
'Sofía S. Villar' 'Niki Kilbertus'] While interest in the application of machine learning to improve healthcare has grown tremendously in recent years, a number of barriers prevent deployment in medical practice. A notable concern is the potential to exacerbate entrenched biases and existing health disparities in…
Joshua Trebach, Ali Graebner, Mark K. Su
“The only way to test a hypothesis is to look for all the information that disagrees with it.” -Karl Popper The word “hypothesis” originates from Greek hypo (“under”) and thesis (“placing”), and its ancient usage was as “a summary of the plot of a classical drama.” Here we will delve into the modern scientific dramas…
Difan Song, Simon Mak, C. F. Jeff Wu
Experiments are the gold standard for causal inference. In many applications, experimental units can often be recruited or chosen sequentially, and the adaptive execution of such experiments may offer greatly improved inference of causal quantities over nonadaptive approaches, particularly when experiments are…
Oleksandr Sverdlov, Yevgen Ryeznik, Volodymyr Anisimov, Olga M. Kuznetsova + 4 more
'Olga M. Kuznetsova' 'Ruth Knight' 'Kerstine Carter' 'Sonja Drescher' 'Wenle Zhao'] Background The design of a multi-center randomized controlled trial (RCT) involves multiple considerations, such as the choice of the sample size, the number of centers and their geographic location, the strategy for recruitment of…
Guijing Zhang, Li-Xin Zhang
Response-adaptive randomization designs have attracted much attention in clinical trials. This paper proposes a new class of response-adaptive design which built on the efficient randomized-adaptive design (ERADE) proposed by Hu, Zhang, and He. The original ERADE uses a discrete allocation probability function and…
E. Chasseloup, X. Li, M. O. Karlsson
Recent investigations assessed two non-linear mixed effect (NLME) model based approaches to test for drug effect on real data in the context of balanced two-arms designs. The standard approach (STD) showed type I error inflation and biased drug effect estimates contrary to the proposed alternative, individual model…
Anja Collazo, Hans-Georg Kuhn, Tobias Kurth, Marco Piccininni + 1 more
Animal attrition in preclinical experiments can introduce bias in the estimation of causal treatment effects, as surviving animals may not be representative of the entire study population. This can compromise the internal validity of the study, despite randomization at the outset. Directed Acyclic Graphs (DAGs) are…
Alexandre Cretton, Kate Schipper, Mahmoud Hassan, Paolo Ruggeri + 1 more
Contextual interference (CI) enhances motor learning by practicing skill variations in a random rather than blocked order. It has been demonstrated that performing aiming distances in a random order increased electrophysiological (EEG) markers of perceptual, attentional, and working memory processes. However, only the…
Yushi Tang, Irineo Cabreros, John D. Storey
The process by which genes are transmitted from parent to child provides a source of randomization preceding all other factors that may causally influence any particular child phenotype. Because of this, it is natural to consider genetic transmission as a source of experimental randomization. In this work, we show how…
Anita Bandrowski, Amit Namburi, Adam Ferguson, Candace L. Floyd + 1 more
Preclinical research in traumatic brain injury (TBI) continues to significantly increase knowledge and yield a large number of peer-reviewed studies, but translation of these results to the clinical setting has been minimal. Rigor and transparency factors such as concealment of group allocation (e.g., “blinding’’) or…
Authors not listed
With the ever-increasing demand for atomistic structures representative of real-life systems as well as the ad-vent of exascale computers, it has now become necessary and possible to use advanced global optimization (GO) techniques to intelligently sample the potential energy surface (PES). Given the previous studies…
Prashanth Athri, Vidhya Murali, Pradyumna Y Muralidhar, Cassandra Königs + 4 more
- 1. Department of Computer Science and Engineering, Amrita School of Engineering, Amrita Vishwa Vidyapeetham, Bengaluru, India - 2. PES Center for Pattern Recognition, Department of Computer Science and Engineering, PES University, Bengaluru, India - 3. Bioinformatics and Medical Informatics, Bielefeld University…
Long Qian, Xin Lu, Parvez Haris, Jianyong Zhu + 2 more
Clinical trials are crucial for drug development, but they require significant time and financial resources. Additionally, uncertainties may arise during these trials concerning their results due to concerns surrounding effectiveness, safety, or the enrollment of participants. If robust AI (artificial intelligence)…
Sophie C.M.J. Willemsen, Leonie Oostwoud Wijdenes, Robert J. van Beers, Mathieu Koppen + 1 more
Path integration, the process of updating one’s position using successive self-motion signals, has previously been studied using visual distance reproduction tasks in which optic flow patterns provide information about traveled distance. These studies have reported that reproduced distances show two types of systematic…
Authors not listed
Integrating machine learning (ML) into drug discovery has ushered in a new era of innovation, dramatically enhancing the efficiency and precision of identifying and developing new therapeutics. This review provides a comprehensive analysis of the current applications of machine learning in drug discovery, focusing on…