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Sequential decision making significantly speeds up research and is more cost-effective compared to fixed-n methods. We present a method for sequential decision making for stratified count data that retains Type-I error guarantee or false…

Methodology · Statistics 2023-02-23 Rosanne J. Turner , Peter D. Grünwald

We propose a two-stage design for a clinical trial with an early stopping rule for safety. We use different criteria to assess early stopping and efficacy. The early stopping rule is based on a criteria that can be determined more quickly…

Methodology · Statistics 2013-07-25 Daniel Zelterman

We demonstrate the effectiveness of the categorical distribution as a neural network output for next event prediction. This is done for both discrete-time and continuous-time event sequences. To model continuous-time processes, the…

Machine Learning · Computer Science 2026-01-26 Kevin Doran , Tom Baden

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

The predictions from an accurate prognostic model can be of great interest to patients and clinicians. When predictions are reported to individuals, they may decide to take action to improve their health or they may simply be comforted by…

Quantitative Methods · Quantitative Biology 2019-09-10 Michael C Sachs , Arvid Sjölander , Erin E Gabriel

Computer experiments have become an indispensable alternative to complex physical and engineering experiments. The Kriging model is the most widely used surrogate model, with the core goal of minimizing the discrepancy between the surrogate…

Methodology · Statistics 2026-01-26 Ruonan Zheng , Min-Qian Liu , Yongdao Zhou , Xuan Chen

Applied Difference-in-Differences studies often involve outcomes that are discrete, mixed, censored, or otherwise non-continuously distributed, while policy questions frequently concern distributional effects rather than mean effects alone.…

Econometrics · Economics 2026-05-22 Nelly K. Djuazon , Emmanuel Selorm Tsyawo

In this paper we propose a new template for empirical studies intended to assess causal effects: the outcome-wide longitudinal design. The approach is an extension of what is often done to assess the causal effects of a treatment or…

Methodology · Statistics 2018-10-25 Tyler J. VanderWeele , Maya B. Mathur , Ying Chen

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

In urgent decision making applications, ensemble simulations are an important way to determine different outcome scenarios based on currently available data. In this paper, we will analyze the output of ensemble simulations by considering…

Graphics · Computer Science 2019-10-21 Max Kontak , Jules Vidal , Julien Tierny

In this paper, we consider an experimental setting where units enter the experiment sequentially. Our goal is to form stopping rules which lead to estimators of treatment effects with a given precision. We propose a fixed-width confidence…

Statistics Theory · Mathematics 2024-05-07 Mattias Nordin , Mårten Schultzberg

Scientists are attempting to use models of ever increasing complexity, especially in medicine, where gene-based diseases such as cancer require better modeling of cell regulation. Complex models suffer from uncertainty and experiments are…

Molecular Networks · Quantitative Biology 2018-06-01 Mahdi Imani , Roozbeh Dehghannasiri , Ulisses M. Braga-Neto , Edward R. Dougherty

Multivariate outcomes are common in pragmatic cluster randomized trials. While sample size calculation procedures for multivariate outcomes exist under parallel assignment, none have been developed for a stepped wedge design. In this…

Methodology · Statistics 2022-12-05 Kendra Davis-Plourde , Monica Taljaard , Fan Li

Strategic test allocation plays a major role in the control of both emerging and existing pandemics (e.g., COVID-19, HIV). Widespread testing supports effective epidemic control by (1) reducing transmission via identifying cases, and (2)…

Methodology · Statistics 2022-12-06 Ivana Malenica , Jeremy R. Coyle , Mark J. van der Laan , Maya L. Petersen

In repeated Measure Designs with multiple groups, the primary purpose is to compare different groups in various aspects. For several reasons, the number of measurements and therefore the dimension of the observation vectors can depend on…

Statistics Theory · Mathematics 2022-07-20 Paavo Sattler , Markus Pauly

Calibration of expensive simulation models involves an emulator based on simulation outputs generated across various parameter settings to replace the actual model. Noisy outputs of stochastic simulation models require many simulation…

Methodology · Statistics 2025-05-08 Özge Sürer

This paper introduces a new discrete distribution suggested by curtailed sampling rules common in early-stage clinical trials. We derive the distribution of the smallest number of independent Bernoulli(p) trials needed in order to observe…

Statistics Theory · Mathematics 2018-02-16 Michelle DeVeaux , Michael J. Kane , Daniel Zelterman

This paper studies the identification of causal effects of a continuous treatment using a new difference-in-difference strategy. Our approach allows for endogeneity of the treatment, and employs repeated cross-sections. It requires an…

Econometrics · Economics 2023-04-18 Xavier D'Haultfoeuille , Stefan Hoderlein , Yuya Sasaki

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient…

Methodology · Statistics 2017-10-11 Michael Grayling , James Wason , Adrian Mander