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Suppose we are interested in the effect of a treatment in a clinical trial. The efficiency of inference may be limited due to small sample size. However, external control data are often available from historical studies. Motivated by an…

Methodology · Statistics 2021-12-10 Xinyu Li , Wang Miao , Fang Lu , Xiao-Hua Zhou

In a completely randomized experiment, the variances of treatment effect estimators in the finite population are usually not identifiable and hence not estimable. Although some estimable bounds of the variances have been established in the…

Statistics Theory · Mathematics 2022-09-20 Ruoyu Wang , Qihua Wang , Wang Miao , Xiaohua Zhou

In clinical trials, it is often of interest to understand the principal causal effect (PCE), the average treatment effect for a principal stratum (a subset of patients defined by the potential outcomes of one or more post-baseline…

Methodology · Statistics 2022-10-06 Yongming Qu , Ilya Lipkovich , Stephen J. Ruberg

We study the identification and estimation of long-term treatment effects when both experimental and observational data are available. Since the long-term outcome is observed only after a long delay, it is not measured in the experimental…

Methodology · Statistics 2024-09-04 Guido Imbens , Nathan Kallus , Xiaojie Mao , Yuhao Wang

Interval analysis, when applied to the so called problem of experimental data fitting, appears to be still in its infancy. Sometimes, partly because of the unrivaled reliability of interval methods, we do not obtain any results at all.…

Data Analysis, Statistics and Probability · Physics 2009-03-03 Marek W. Gutowski

When the difference between treatments in a clinical trial is estimated by a difference in means, then it is well known that randomization ensures unbiassed estimation, even if no account is taken of important baseline covariates. However,…

Statistics Theory · Mathematics 2014-07-22 J. N. S. Matthews , Nuri H. Badi

Though platform trials have been touted for their flexibility and streamlined use of trial resources, their statistical efficiency is not well understood. We fill this gap by establishing their greater efficiency for comparing the relative…

Methodology · Statistics 2022-01-28 Tzu-Jung Huang , Alex Luedtke , the AMP Investigators Group

Variable selection for regression models plays a key role in the analysis of biomedical data. However, inference after selection is not covered by classical statistical frequentist theory which assumes a fixed set of covariates in the…

Methodology · Statistics 2021-07-21 Michael Kammer , Daniela Dunkler , Stefan Michiels , Georg Heinze

Interim analyses are vital in clinical trials for early decision-making. While frequentist implications are well-established, the consequences of repeated Bayesian interim monitoring for efficacy, specifically regarding multiplicity, remain…

Methodology · Statistics 2025-12-30 Suyu Liu , Beibei Guo , Laura Thompson , Lei Nie , Ying Yuan

In the analysis of survey data it is of interest to estimate and quantify uncertainty about means or totals for each of several non-overlapping subpopulations, or areas. When the sample size for a given area is small, standard confidence…

Methodology · Statistics 2018-09-26 Kyle Burris , Peter Hoff

Confidence intervals for a binomial parameter or for the ratio of Poisson means are commonly desired in high energy physics (HEP) applications such as measuring a detection efficiency or branching ratio. Due to the discreteness of the data,…

Data Analysis, Statistics and Probability · Physics 2009-12-23 Robert D. Cousins , Kathryn E. Hymes , Jordan Tucker

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

The analysis of platform trials can be enhanced by utilizing non-concurrent controls. Since including this data might also introduce bias in the treatment effect estimators if time trends are present, methods for incorporating…

Methodology · Statistics 2026-05-21 Pavla Krotka , Martin Posch , Marta Bofill Roig

Clinical trials often allow patients in the control arm to switch to the treatment arm if their physical conditions are worse than certain tolerance levels. For instance, treatment switching arises in the Concorde clinical trial, which aims…

Applications · Statistics 2023-09-06 Alessandra Mattei , Peng Ding , Veronica Ballerini , Fabrizia Mealli

Comparisons are carried out of the confidence intervals constructed with Neyman's frequentist method and with the \Delta L=1/2 likelihood method, using the example of low-statistics life time estimates.

Data Analysis, Statistics and Probability · Physics 2007-05-23 A. D. Bukin

The following questions are discussed: ``Why confidence intervals are a hot topic?''; ``Are confidence intervals objective?''; ``What is the usefulness of coverage?''; ``How to obtain useful information from experiment?''; ``The confidence…

High Energy Physics - Experiment · Physics 2007-05-23 C. Giunti

It is crucial to design Phase II cancer clinical trials that balance the efficiency of treatment selection with clinical practicality. Sargent and Goldberg proposed a frequentist design that allow decision-making even when the primary…

Methodology · Statistics 2025-05-15 Moka Komaki , Satoru Shinoda , Haiyan Zheng , Kouji Yamamoto

When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…

Methodology · Statistics 2023-09-08 Nicole Schnitzler , Eloise Kaizar

The difference-in-differences (DID) method identifies the average treatment effects on the treated (ATT) under mainly the so-called parallel trends (PT) assumption. The most common and widely used approach to justify the PT assumption is…

Econometrics · Economics 2023-08-23 Kyunghoon Ban , Désiré Kédagni

The purpose of this paper is to propose methodologies for statistical inference of low-dimensional parameters with high-dimensional data. We focus on constructing confidence intervals for individual coefficients and linear combinations of…

Methodology · Statistics 2012-11-05 Cun-Hui Zhang , Stephanie S. Zhang
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