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Single-arm studies in the early development phases of new treatments are not uncommon in the context of rare diseases or in paediatrics. If an assessment of efficacy is to be made at the end of such a study, the observed endpoints can be…

Methodology · Statistics 2026-03-02 Moritz Fabian Danzer , Rene Schmidt

Cross-validation under sample selection bias can, in principle, be done by importance-weighting the empirical risk. However, the importance-weighted risk estimator produces sub-optimal hyperparameter estimates in problem settings where…

Machine Learning · Computer Science 2019-08-28 Wouter M. Kouw , Jesse H. Krijthe , Marco Loog

Scholars frequently use covariate balance tests to test the validity of natural experiments and related designs. Unfortunately, when measured covariates are unrelated to potential outcomes, balance is uninformative about key identification…

Methodology · Statistics 2025-10-15 Clara Bicalho , Adam Bouyamourn , Thad Dunning

The (relevance) weighted likelihood was introduced to formally embrace a variety of statistical procedures that trade bias for precision. Unlike its classical counterpart, the weighted likelihood combines all relevant information while…

Statistics Theory · Mathematics 2007-06-13 Xiaogang Wang , James V. Zidek

Background: For RCTs with time-to-event endpoints, proportional hazard (PH) models are typically used to estimate treatment effects and logrank tests are commonly used for hypothesis testing. There is growing support for replacing this…

Methodology · Statistics 2024-12-10 Dominic Magirr , Craig Wang , Xinlei Deng , Tim Morris , Mark Baillie

In this paper we consider how to evaluate survival distribution predictions with measures of discrimination. This is a non-trivial problem as discrimination measures are the most commonly used in survival analysis and yet there is no clear…

Machine Learning · Statistics 2022-03-10 Raphael Sonabend , Andreas Bender , Sebastian Vollmer

The maximum likelihood estimation of the left-truncated log-logistic distribution with a given truncation point is analyzed in detail from both mathematical and numerical perspectives. These maximum likelihood equations often do not possess…

Non-proportional hazards data are routinely encountered in randomized clinical trials. In such cases, classic Cox proportional hazards model can suffer from severe power loss, with difficulty in interpretation of the estimated hazard ratio…

Methodology · Statistics 2023-12-29 Hong Zhang , Qing Li , Devan V. Mehrotra , Judong Shen

For the analysis of time-to-event data, frequently used methods such as the log-rank test or the Cox proportional hazards model are based on the proportional hazards assumption, which is often debatable. Although a wide range of parametric…

This paper develops a framework for incorporating prior information into sequential multiple testing procedures while maintaining asymptotic optimality. We define a weighted log-likelihood ratio (WLLR) as an additive modification of the…

Methodology · Statistics 2026-02-24 Soumyabrata Bose , Jay Bartroff

Imbalance in covariate distributions leads to biased estimates of causal effects. Weighting methods attempt to correct this imbalance but rely on specifying models for the treatment assignment mechanism, which is unknown in observational…

Methodology · Statistics 2022-05-13 Eric Dunipace

A crossover trial is an efficient trial design when there is no carry-over effect. To reduce the impact of the biological carry-over effect, a washout period is often designed. However, the carry-over effect remains an outstanding concern…

Applications · Statistics 2024-03-06 Danni Shi , Ting Ye

Cross-validation is a statistical tool that can be used to improve large covariance matrix estimation. Although its efficiency is observed in practical applications and a convergence result towards the error of the non linear shrinkage is…

Statistics Theory · Mathematics 2025-09-18 Lamia Lamrani , Christian Bongiorno , Marc Potters

Rare disease trials face unique statistical challenges due to limited patient populations and heterogeneous clinical manifestations among patients. Multiple endpoints are often necessary to comprehensively capture treatment benefits. A…

Methodology · Statistics 2026-05-05 Tianyue Zhou , Susan Gruber , Hana Lee , Wonyul Lee , Lei Nie , Mark van der Laan

The logrank test is a well-known nonparametric test which is often used to compare the survival distributions of two samples including right censored observations, it is also known as the Mantel-Haenszel test. The $G^{\rho}$ family of…

Methodology · Statistics 2020-08-04 Tahani Coolen-Maturi , Frank P. A. Coolen

Weighted empirical risk minimization is a common approach to prediction under distribution drift. This article studies its out-of-sample prediction error under nonstationarity. We provide a general decomposition of the excess risk into a…

Machine Learning · Statistics 2026-05-19 Tobias Brock , Thomas Nagler

Covariate balance is crucial for unconfounded descriptive or causal comparisons. However, lack of balance is common in observational studies. This article considers weighting strategies for balancing covariates. We define a general class of…

Methodology · Statistics 2016-09-30 Fan Li , Kari Lock Morgan , Alan M. Zaslavsky

Covariate balance is crucial for unconfounded descriptive or causal comparisons. However, lack of balance is common in observational studies. This article considers weighting strategies for balancing covariates. We define a general class of…

Methodology · Statistics 2016-11-17 Fan Li , Kari Lock Morgan , Alan M. Zaslavsky

Advancement in sequencing technology enables the study of association between complex disorders and rare variants with low minor allele frequencies. One of the major challenges in rare variant testing is lack of statistical power of…

Quantitative Methods · Quantitative Biology 2016-07-27 Rui Sun , Haoyi Weng , Inchi Hu , Junfeng Guo , William K. K. Wu , Benny Chung-Ying Zee , Maggie Haitian Wang

We present a new way of testing ordered hypotheses against all alternatives which overpowers the classical approach both in simplicity and statistical power. Our new method tests the constrained likelihood ratio statistic against the…

Methodology · Statistics 2018-06-26 Diaa Al Mohamad , Jelle J. Goeman , Erik W. van Zwet , Eric A. Cator