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Traditionally, the major objective in phase I trials is to identify a working-dose for subsequent studies, whereas the major endpoint in phase II and III trials is treatment efficacy. The dose sought is typically referred to as the maximum…

Methodology · Statistics 2016-08-14 Mourad Tighiouart , André Rogatko

If explicit, formal consideration of clinical pharmacology at all informs the design and conduct of modern oncology dose-finding trials, the designs themselves hardly attest to this. Yet in conducting a trial, investigators affirm that they…

Methodology · Statistics 2020-12-25 David C. Norris

Targeted maximum likelihood estimation (TMLE) is a general method for estimating parameters in semiparametric and nonparametric models. Each iteration of TMLE involves fitting a parametric submodel that targets the parameter of interest. We…

Methodology · Statistics 2014-06-03 Iván Díaz , Michael Rosenblum

Contours are used in radiotherapy treatment planning to identify regions to be irradiated with high dose and regions to be spared. Therefore, any contouring uncertainty influences the whole treatment. Even though this is the biggest…

Medical Physics · Physics 2022-04-22 Eliana Vásquez Osorio , Jane Shortall , Jennifer Robbins , Marcel van Herk

Dual agent dose-finding trials study the effect of a combination of more than one agent, where the objective is to find the Maximum Tolerated Dose Combination (MTC), the combination of doses of the two agents that is associated with a…

Applications · Statistics 2025-02-10 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

In the first stage of a two-stage study, the researcher uses a statistical model to impute the unobserved exposures. In the second stage, imputed exposures serve as covariates in epidemiological models. Imputation error in the first stage…

Applications · Statistics 2021-07-19 Ron Sarafian , Itai Kloog , Jonathan D. Rosenblatt

A major bottleneck of standard auto-regressive large language models is that their inference process is inherently sequential, resulting in very long and costly inference times. To circumvent this, practitioners proposed a class of language…

Machine Learning · Computer Science 2025-11-11 Sitan Chen , Kevin Cong , Jerry Li

Long-Short-Term-Memory (LSTM) networks have shown great promise in artificial intelligence (AI) based language modeling. Recently, LSTM networks have also become popular for designing AI-based Intrusion Detection Systems (IDS). However, its…

Cryptography and Security · Computer Science 2021-09-24 Mohit Sewak , Sanjay K. Sahay , Hemant Rathore

We identify three properties of the standard oncology phase I trial design or 3 + 3 design. We show that the standard design implicitly uses isotonic regression to estimate a maximum tolerated dose. We next illustrate the relationship…

Applications · Statistics 2009-06-08 Gregory J. Hather , Howard Mackey

A common problem faced in clinical studies is that of estimating the effect of the most effective (e.g., the one having the largest mean) treatment among $k~(\geq2)$ available treatments. The most effective treatment is adjudged based on…

Statistics Theory · Mathematics 2022-09-20 Masihuddin , Neeraj Misra

Nonconvex and nonsmooth optimization problems are frequently encountered in much of statistics, business, science and engineering, but they are not yet widely recognized as a technology in the sense of scalability. A reason for this…

Optimization and Control · Mathematics 2018-01-19 Bo Jiang , Tianyi Lin , Shiqian Ma , Shuzhong Zhang

Regression discontinuity design (RDD) is a quasi-experimental approach to study the causal effects of an intervention/treatment on later health outcomes. It exploits a continuously measured assignment variable with a clearly defined cut-off…

Applications · Statistics 2024-06-28 Maja Popovic , Daniela Zugna , Lorenzo Richiardi

We consider designs for cancer trials which allow each medical centre to treat only a limited number of cancer types with only a limited number of drugs. We specify desirable properties of these designs, and prove some consequences. Then we…

Applications · Statistics 2019-05-31 R. A. Bailey , Peter J. Cameron

Discovery problems often require deciding whether additional sampling is needed to detect all categories whose prevalence exceeds a prespecified threshold. We study this question under a Bernoulli product (incidence) model, where categories…

Methodology · Statistics 2026-01-29 Alessandro Colombi , Mario Beraha , Amichai Painsky , Stefano Favaro

The regression discontinuity design (RDD) is a quasi-experimental design that can be used to identify and estimate the causal effect of a treatment using observational data. In an RDD, a pre-specified rule is used for treatment assignment,…

Methodology · Statistics 2016-01-05 Panayiota Constantinou , Aidan G. O'Keeffe

We derive conditions for posterior consistency when the responses are independent but not identically distributed ($i.n.i.d$) and the model is "misspecified" to be a family of densities parametrized by a possibly infinite dimensional…

Statistics Theory · Mathematics 2014-08-27 Karthik Sriram , R. V. Ramamoorthi

We consider an experiment with at least two stages or batches and $O(N)$ subjects per batch. First, we propose a semiparametric treatment effect estimator that efficiently pools information across the batches, and show it asymptotically…

Methodology · Statistics 2023-09-28 Harrison H. Li , Art B. Owen

We consider the problem of constructing optimal designs for population pharmacokinetics which use random effect models. It is common practice in the design of experiments in such studies to assume uncorrelated errors for each subject. In…

Applications · Statistics 2010-11-16 Holger Dette , Andrey Pepelyshev , Tim Holland-Letz

Suppose one has a collection of parameters indexed by a (possibly infinite dimensional) set. Given data generated from some distribution, the objective is to estimate the maximal parameter in this collection evaluated at this distribution.…

Methodology · Statistics 2016-05-26 Alexander R. Luedtke , Mark J. van der Laan

The Survivable Network Design problem (SNDP) is a well-studied problem, motivated by the design of networks that are robust to faults under the assumption that any subset of edges up to a specific number can fail. We consider non-uniform…

Data Structures and Algorithms · Computer Science 2024-03-26 Chandra Chekuri , Rhea Jain