Unbiasedness and efficiency of non-parametric and UMVUE estimators of the probabilistic index and related statistics.

Abstract:

:In reliability theory, diagnostic accuracy, and clinical trials, the quantity P ( X > Y ) + 1 / 2 P ( X = Y ) , also known as the Probabilistic Index (PI), is a common treatment effect measure when comparing two groups of observations. The quantity P ( X > Y ) - P ( Y > X ) , a linear transformation of PI known as the net benefit, has also been advocated as an intuitively appealing treatment effect measure. Parametric estimation of PI has received a lot of attention in the past 40 years, with the formulation of the Uniformly Minimum-Variance Unbiased Estimator (UMVUE) for many distributions. However, the non-parametric Mann-Whitney estimator of the PI is also known to be UMVUE in some situations. To understand this seeming contradiction, in this paper a systematic comparison is performed between the non-parametric estimator for the PI and parametric UMVUE estimators in various settings. We show that the Mann-Whitney estimator is always an unbiased estimator of the PI with univariate, completely observed data, while the parametric UMVUE is not when the distribution is misspecified. Additionally, the Mann-Whitney estimator is the UMVUE when observations belong to an unrestricted family. When observations come from a more restrictive family of distributions, the loss in efficiency for the non-parametric estimator is limited in realistic clinical scenarios. In conclusion, the Mann-Whitney estimator is simple to use and is a reliable estimator for the PI and net benefit in realistic clinical scenarios.

journal_name

Stat Methods Med Res

authors

Verbeeck J,Deltuvaite-Thomas V,Berckmoes B,Burzykowski T,Aerts M,Thas O,Buyse M,Molenberghs G

doi

10.1177/0962280220966629

subject

Has Abstract

pub_date

2020-12-01 00:00:00

pages

962280220966629

eissn

0962-2802

issn

1477-0334

pub_type

杂志文章
  • Analysis of phase II methodologies for single-arm clinical trials with multiple endpoints in rare cancers: An example in Ewing's sarcoma.

    abstract::Trials run in either rare diseases, such as rare cancers, or rare sub-populations of common diseases are challenging in terms of identifying, recruiting and treating sufficient patients in a sensible period. Treatments for rare diseases are often designed for other disease areas and then later proposed as possible tre...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216662070

    authors: Dutton P,Love SB,Billingham L,Hassan AB

    更新日期:2018-05-01 00:00:00

  • Advanced colorectal neoplasia risk stratification by penalized logistic regression.

    abstract::Colorectal cancer is the second leading cause of death from cancer in the United States. To facilitate the efficiency of colorectal cancer screening, there is a need to stratify risk for colorectal cancer among the 90% of US residents who are considered "average risk." In this article, we investigate such risk stratif...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280213497432

    authors: Lin Y,Yu M,Wang S,Chappell R,Imperiale TF

    更新日期:2016-08-01 00:00:00

  • Regression towards the mean, historically considered.

    abstract::The simple yet subtle concept of regression towards the mean is reviewed historically. Verbal, geometric, and mathematical expressions of the concept date to the discoverer of the concept, Francis Galton. That discovery and subsequent understanding (and misunderstanding) of the concept are surveyed. ...

    journal_title:Statistical methods in medical research

    pub_type: 传,历史文章,杂志文章,评审

    doi:10.1177/096228029700600202

    authors: Stigler SM

    更新日期:1997-06-01 00:00:00

  • Measuring continuous baseline covariate imbalances in clinical trial data.

    abstract::This paper presents and compares several methods of measuring continuous baseline covariate imbalance in clinical trial data. Simulations illustrate that though the t-test is an inappropriate method of assessing continuous baseline covariate imbalance, the test statistic itself is a robust measure in capturing imbalan...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211416038

    authors: Ciolino JD,Martin RH,Zhao W,Hill MD,Jauch EC,Palesch YY

    更新日期:2015-04-01 00:00:00

  • Measurement error correction using validation data: a review of methods and their applicability in case-control studies.

    abstract::Measurement error is a serious problem in the analysis of epidemiological data. In the past 20 years, a large number of methods for the correction of measurement error have been developed. While at the beginning mostly methods for cohort studies were considered, recently more attention has been paid to case-control st...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/096228020000900504

    authors: Thürigen D,Spiegelman D,Blettner M,Heuer C,Brenner H

    更新日期:2000-10-01 00:00:00

  • A comparative review of methods for comparing means using partially paired data.

    abstract::In medical experiments with the objective of testing the equality of two means, data are often partially paired by design or because of missing data. The partially paired data represent a combination of paired and unpaired observations. In this article, we review and compare nine methods for analyzing partially paired...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/0962280215577111

    authors: Guo B,Yuan Y

    更新日期:2017-06-01 00:00:00

  • A mixed-effects, spatially varying coefficients model with application to multi-resolution functional magnetic resonance imaging data.

    abstract::Spatial resolution plays an important role in functional magnetic resonance imaging studies as the signal-to-noise ratio increases linearly with voxel volume. In scientific studies, where functional magnetic resonance imaging is widely used, the standard spatial resolution typically used is relatively low which ensure...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217752378

    authors: Liu Z,Bartsch AJ,Berrocal VJ,Johnson TD

    更新日期:2019-04-01 00:00:00

  • Model selection in multivariate semiparametric regression.

    abstract::Variable selection in semiparametric mixed models for longitudinal data remains a challenge, especially in the presence of multiple correlated outcomes. In this paper, we propose a model selection procedure that simultaneously selects fixed and random effects using a maximum penalized likelihood method with the adapti...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217690769

    authors: Li Z,Liu H,Tu W

    更新日期:2018-10-01 00:00:00

  • A new approach to hierarchical data analysis: Targeted maximum likelihood estimation for the causal effect of a cluster-level exposure.

    abstract::We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials are applied to learn about real-world implementation, sustainability, and popula...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280218774936

    authors: Balzer LB,Zheng W,van der Laan MJ,Petersen ML

    更新日期:2019-06-01 00:00:00

  • Comparing cluster-level dynamic treatment regimens using sequential, multiple assignment, randomized trials: Regression estimation and sample size considerations.

    abstract::Cluster-level dynamic treatment regimens can be used to guide sequential treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level dynamic treatment regimen, the treatment is potentially adapted and re-adapted over time based on changes in the cl...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217708654

    authors: NeCamp T,Kilbourne A,Almirall D

    更新日期:2017-08-01 00:00:00

  • A frequentist approach to estimating the force of infection for a respiratory disease using repeated measurement data from a birth cohort.

    abstract::This article aims to develop a probability-based model involving the use of direct likelihood formulation and generalised linear modelling (GLM) approaches useful in estimating important disease parameters from longitudinal or repeated measurement data. The current application is based on infection with respiratory sy...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210385749

    authors: Mwambi H,Ramroop S,White Lj,Okiro E,Nokes Dj,Shkedy Z,Molenberghs G

    更新日期:2011-10-01 00:00:00

  • Optimal scheduling of post-therapeutic follow-up of patients treated for cancer for early detection of relapses.

    abstract::Post-therapeutic surveillance is one important component of cancer care. However, there still is no evidence-based strategies to schedule patients' follow-up examinations. Our approach is based on the modeling of the probability of the onset of relapse at an early asymptotic or preclinical stage and its transition to ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214524178

    authors: Somda SM,Leconte E,Boher JM,Asselain B,Kramar A,Filleron T

    更新日期:2016-12-01 00:00:00

  • Maximum likelihood estimation based on Newton-Raphson iteration for the bivariate random effects model in test accuracy meta-analysis.

    abstract::A bivariate generalised linear mixed model is often used for meta-analysis of test accuracy studies. The model is complex and requires five parameters to be estimated. As there is no closed form for the likelihood function for the model, maximum likelihood estimates for the parameters have to be obtained numerically. ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280219853602

    authors: Willis BH,Baragilly M,Coomar D

    更新日期:2020-04-01 00:00:00

  • Analysis of clustered competing risks data using subdistribution hazard models with multivariate frailties.

    abstract::Competing risks data often exist within a center in multi-center randomized clinical trials where the treatment effects or baseline risks may vary among centers. In this paper, we propose a subdistribution hazard regression model with multivariate frailty to investigate heterogeneity in treatment effects among centers...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214526193

    authors: Ha ID,Christian NJ,Jeong JH,Park J,Lee Y

    更新日期:2016-12-01 00:00:00

  • A measure of association for ordered categorical data in population-based studies.

    abstract::Ordinal classification scales are commonly used to define a patient's disease status in screening and diagnostic tests such as mammography. Challenges arise in agreement studies when evaluating the association between many raters' classifications of patients' disease or health status when an ordered categorical scale ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216643347

    authors: Nelson KP,Edwards D

    更新日期:2018-03-01 00:00:00

  • Assessing the reliability of ordered categorical scales using kappa-type statistics.

    abstract::Methods for the analysis of reliability of ordered categorical scales are discussed, focussing on the limitation of the single summary-weighted kappa coefficients. A symmetric matrix of kappa-type coefficients is suggested as an alternative. The method is proposed as being suitable for ordinal scale where there is no ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1191/0962280205sm413oa

    authors: Roberts C,McNamee R

    更新日期:2005-10-01 00:00:00

  • Nonparametric estimation of risk tracking indices for longitudinal studies.

    abstract::Tracking a subject's risk factors or health status over time is an important objective in long-term epidemiological studies with repeated measurements. An important issue of time-trend tracking is to define appropriate statistical indices to quantitatively measure the tracking abilities of the targeted risk factors or...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280219839427

    authors: Wu CO,Tian X,Tian L,Reis JP,Zhao L,Allen NB,Bae S,Liu K

    更新日期:2020-02-01 00:00:00

  • Continuous(ly) missing outcome data in network meta-analysis: A one-stage pattern-mixture model approach.

    abstract::Appropriate handling of aggregate missing outcome data is necessary to minimise bias in the conclusions of systematic reviews. The two-stage pattern-mixture model has been already proposed to address aggregate missing continuous outcome data. While this approach is more proper compared with the exclusion of missing co...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280220983544

    authors: Spineli LM,Kalyvas C,Papadimitropoulou K

    更新日期:2021-01-06 00:00:00

  • Estimation of sensitivity depending on sojourn time and time spent in preclinical state.

    abstract::The probability model for periodic screening was extended to provide statistical inference for sensitivity depending on sojourn time, in which the sensitivity was modeled as a function of time spent in the preclinical state and the sojourn time. The likelihood function with the proposed sensitivity model was then eval...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212465499

    authors: Kim S,Wu D

    更新日期:2016-04-01 00:00:00

  • A new diagnostic accuracy measure and cut-point selection criterion.

    abstract::Most diagnostic accuracy measures and criteria for selecting optimal cut-points are only applicable to diseases with binary or three stages. Currently, there exist two diagnostic measures for diseases with general k stages: the hypervolume under the manifold and the generalized Youden index. While hypervolume under th...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280215611631

    authors: Dong T,Attwood K,Hutson A,Liu S,Tian L

    更新日期:2017-12-01 00:00:00

  • Bayesian modeling and prediction of accrual in multi-regional clinical trials.

    abstract::In multi-regional trials, the underlying overall and region-specific accrual rates often do not hold constant over time and different regions could have different start-up times, which combined with initial jump in accrual within each region often leads to a discontinuous overall accrual rate, and these issues associa...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214557581

    authors: Deng Y,Zhang X,Long Q

    更新日期:2017-04-01 00:00:00

  • Random-effects models for multivariate repeated measures.

    abstract::Mixed models are widely used for the analysis of one repeatedly measured outcome. If more than one outcome is present, a mixed model can be used for each one. These separate models can be tied together into a multivariate mixed model by specifying a joint distribution for their random effects. This strategy has been u...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206075305

    authors: Fieuws S,Verbeke G,Molenberghs G

    更新日期:2007-10-01 00:00:00

  • Projections of cancer mortality risks using spatio-temporal P-spline models.

    abstract::Cancer mortality risk estimates are essential for planning resource allocation and designing and evaluating cancer prevention and management strategies. However, mortality figures generally become available after a few years, making necessary to develop reliable procedures to provide current and near future mortality ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212446366

    authors: Ugarte MD,Goicoa T,Etxeberria J,Militino AF

    更新日期:2012-10-01 00:00:00

  • Estimating the personal cure rate of cancer patients using population-based grouped cancer survival data.

    abstract::Cancer patients are subject to multiple competing risks of death and may die from causes other than the cancer diagnosed. The probability of not dying from the cancer diagnosed, which is one of the patients' main concerns, is sometimes called the 'personal cure' rate. Two approaches of modelling competing-risk surviva...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280209347046

    authors: Binbing Yu,Tiwari RC,Feuer EJ

    更新日期:2011-06-01 00:00:00

  • Mixture modelling for cluster analysis.

    abstract::Cluster analysis via a finite mixture model approach is considered. With this approach to clustering, the data can be partitioned into a specified number of clusters g by first fitting a mixture model with g components. An outright clustering of the data is then obtained by assigning an observation to the component to...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1191/0962280204sm372ra

    authors: McLachlan GJ,Chang SU

    更新日期:2004-10-01 00:00:00

  • A monotone data augmentation algorithm for longitudinal data analysis via multivariate skew-t, skew-normal or t distributions.

    abstract::The mixed effects model for repeated measures has been widely used for the analysis of longitudinal clinical data collected at a number of fixed time points. We propose a robust extension of the mixed effects model for repeated measures for skewed and heavy-tailed data on basis of the multivariate skew-t distribution,...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280219865579

    authors: Tang Y

    更新日期:2020-06-01 00:00:00

  • Parametric models for incomplete continuous and categorical longitudinal data.

    abstract::This paper reviews models for incomplete continuous and categorical longitudinal data. In terms of Rubin's classification of missing value processes we are specifically concerned with the problem of nonrandom missingness. A distinction is drawn between the classes of selection and pattern-mixture models and, using sev...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/096228029900800105

    authors: Kenward MG,Molenberghs G

    更新日期:1999-03-01 00:00:00

  • Combining estimates of the odds ratio: the state of the art.

    abstract::Medical research commonly relies on the combination of 2 x 2 tables of counted data for making inferences about treatment effects or about the causes of disease. This article reviews point estimation and interval estimation for a common odds ratio. Traditional methods for providing these estimates face special challen...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/096228029400300204

    authors: Emerson JD

    更新日期:1994-01-01 00:00:00

  • Measurement error, time lag, unmeasured confounding: Considerations for longitudinal estimation of the effect of a mediator in randomised clinical trials.

    abstract::Clinical trials are expensive and time-consuming and so should also be used to study how treatments work, allowing for the evaluation of theoretical treatment models and refinement and improvement of treatments. These treatment processes can be studied using mediation analysis. Randomised treatment makes some of the a...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216666111

    authors: Goldsmith KA,Chalder T,White PD,Sharpe M,Pickles A

    更新日期:2018-06-01 00:00:00

  • Statistical methods in computational anatomy.

    abstract::This paper reviews recent developments by the Washington/Brown groups for the study of anatomical shape in the emerging new discipline of computational anatomy. Parametric representations of anatomical variation for computational anatomy are reviewed, restricted to the assumption of small deformations. The generation ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/096228029700600305

    authors: Miller M,Banerjee A,Christensen G,Joshi S,Khaneja N,Grenander U,Matejic L

    更新日期:1997-09-01 00:00:00