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The distribution of power-related random variables (and their use in clinical trials) 与功率有关的随机变量的分布(及其在临床试验中的应用)
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-19 DOI: 10.1007/s00362-024-01599-1
Francesco Mariani, Fulvio De Santis, Stefania Gubbiotti

In the hybrid Bayesian-frequentist approach to hypotheses tests, the power function, i.e. the probability of rejecting the null hypothesis, is a random variable and a pre-experimental evaluation of the study is commonly carried out through the so-called probability of success (PoS). PoS is usually defined as the expected value of the random power that is not necessarily a well-representative summary of the entire distribution. Here, we consider the main definitions of PoS and investigate the power related random variables that induce them. We provide general expressions for their cumulative distribution and probability density functions, as well as closed-form expressions when the test statistic is, at least asymptotically, normal. The analysis of such distributions highlights discrepancies in the main definitions of PoS, leading us to prefer the one based on the utility function of the test. We illustrate our idea through an example and an application to clinical trials, which is a framework where PoS is commonly employed.

在贝叶斯-频率主义混合假设检验方法中,幂函数(即拒绝零假设的概率)是一个随机变量,通常通过所谓的成功概率(PoS)对研究进行实验前评估。PoS 通常被定义为随机幂的期望值,它不一定是整个分布的代表性总结。在此,我们考虑了 PoS 的主要定义,并研究了引起 PoS 的与功率相关的随机变量。我们提供了它们的累积分布和概率密度函数的一般表达式,以及当检验统计量至少在渐近上是正态时的闭式表达式。对这些分布的分析凸显了 PoS 主要定义中的差异,使我们更倾向于基于检验效用函数的定义。我们通过一个例子来说明我们的想法,并将其应用到临床试验中,临床试验是常用 PoS 的框架。
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引用次数: 0
The cost of sequential adaptation and the lower bound for mean squared error 顺序适应的成本和均方误差的下限
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-17 DOI: 10.1007/s00362-024-01565-x
Sergey Tarima, Nancy Flournoy

Informative interim adaptations lead to random sample sizes. The random sample size becomes a component of the sufficient statistic and estimation based solely on observed samples or on the likelihood function does not use all available statistical evidence. The total Fisher Information (FI) is decomposed into the design FI and a conditional-on-design FI. The FI unspent by a design’s informative interim adaptation decomposes further into a weighted linear combination of FIs conditional-on-stopping decisions. Then, these components are used to determine the new lower mean squared error (MSE) in post-adaptation estimation because the Cramer–Rao lower bound (1945, 1946) and its sequential version suggested by Wolfowitz (Ann Math Stat 18(2):215–230, 1947) for non-informative stopping are not applicable to post-informative-adaptation estimation. In addition, we also show that the new proposed lower boundary on the MSE is reached by the maximum likelihood estimators in designs with informative adaptations when data are coming from one-parameter exponential family. Theoretical results are illustrated with simple normal samples collected according to a two-stage design with a possibility of early stopping.

有启发性的临时调整会产生随机样本规模。随机样本规模成为充分统计量的一个组成部分,而仅仅基于观测样本或似然函数的估计并没有使用所有可用的统计证据。总费雪信息 (FI) 分解为设计 FI 和条件设计 FI。设计信息中期调整未消耗的费雪信息进一步分解为以停止决策为条件的费雪信息的加权线性组合。然后,这些成分被用来确定适应后估计中新的均方误差下限(MSE),因为 Wolfowitz(Ann Math Stat 18(2):215-230, 1947)提出的用于非信息停止的 Cramer-Rao 下限(1945, 1946)及其顺序版本不适用于信息适应后估计。此外,我们还证明,当数据来自单参数指数族时,在有信息适应的设计中,最大似然估计值可以达到新提出的 MSE 下限。理论结果以根据两阶段设计收集的简单正态样本为例作了说明,该设计有可能提前停止。
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引用次数: 0
Nested strong orthogonal arrays 嵌套强正交阵列
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-16 DOI: 10.1007/s00362-024-01609-2
Chunwei Zheng, Wenlong Li, Jian-Feng Yang

Nested space-filling designs are popular for conducting multiple computer experiments with different levels of accuracy. Strong orthogonal arrays (SOAs) are a special type of space-filling designs which possess attractive low-dimensional stratifications. Combining these two kinds of designs, we propose a new type of design called a nested strong orthogonal array. Such a design is a special nested space-filling design that consists of two layers, i.e., the large SOA and the small SOA, where they enjoy different strengths, and the small one is nested in the large one. The proposed construction method is easy to use, capable of accommodating a larger number of columns, and the resulting designs possess better stratifications than the existing nested space-filling designs in two dimensions. The construction method is based on regular second order saturated designs and nonregular designs. Some comparisons with the existing nested space-filling designs are given to show the usefulness of the proposed designs.

嵌套空间填充设计是进行不同精度的多重计算机实验的常用方法。强正交阵列(SOA)是一种特殊的空间填充设计,它拥有极具吸引力的低维分层。结合这两种设计,我们提出了一种名为嵌套强正交阵列的新型设计。这种设计是一种特殊的嵌套空间填充设计,由两层组成,即大 SOA 和小 SOA,它们具有不同的强度,小 SOA 嵌套在大 SOA 中。与现有的二维嵌套式空间填充设计相比,所提出的构建方法易于使用,能够容纳更多的柱子,而且所产生的设计具有更好的分层效果。该构造方法基于规则二阶饱和设计和非规则设计。与现有的嵌套空间填充设计进行了一些比较,以显示拟议设计的实用性。
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引用次数: 0
Tests for time-varying coefficient spatial autoregressive panel data model with fixed effects 具有固定效应的时变系数空间自回归面板数据模型检验
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-14 DOI: 10.1007/s00362-024-01607-4
Lingling Tian, Yunan Su, Chuanhua Wei

As an extension of the spatial autoregressive panel data model and the time-varying coefficient panel data model, the time-varying coefficient spatial autoregressive panel data model is useful in analysis of spatial panel data. While research has addressed the estimation problem of this model, less attention has been given to hypotheses tests. This paper studies two tests for this semiparametric spatial panel data model. One considers the existence of the spatial lag term, and the other determines whether some time-varying coefficients are constants. We employ the profile generalized likelihood ratio test procedure to construct the corresponding test statistic, and the residual-based bootstrap procedure is used to derive the p-value of the tests. Some simulations are conducted to evaluate the performance of the proposed test method, the results show that the proposed methods have good finite sample properties. Finally, we apply the proposed test methods to the provincial carbon emission data of China. Our findings suggest that the partially linear time-varying coefficients spatial autoregressive panel data model provides a better fit for the carbon emission data.

作为空间自回归面板数据模型和时变系数面板数据模型的扩展,时变系数空间自回归面板数据模型在空间面板数据分析中非常有用。虽然已有研究解决了该模型的估计问题,但较少关注假设检验。本文研究了该半参数空间面板数据模型的两种检验方法。一个是考虑空间滞后项的存在,另一个是确定某些时变系数是否为常数。我们采用剖面广义似然比检验程序来构建相应的检验统计量,并使用基于残差的引导程序来得出检验的 p 值。我们进行了一些模拟来评估所提出的检验方法的性能,结果表明所提出的方法具有良好的有限样本特性。最后,我们将所提出的检验方法应用于中国省级碳排放数据。我们的研究结果表明,部分线性时变系数空间自回归面板数据模型能更好地拟合碳排放数据。
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引用次数: 0
On the consistency of supervised learning with missing values 论缺失值监督学习的一致性
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-12 DOI: 10.1007/s00362-024-01550-4
Julie Josse, Jacob M. Chen, Nicolas Prost, Gaël Varoquaux, Erwan Scornet

In many application settings, data have missing entries, which makes subsequent analyses challenging. An abundant literature addresses missing values in an inferential framework, aiming at estimating parameters and their variance from incomplete tables. Here, we consider supervised-learning settings: predicting a target when missing values appear in both training and test data. We first rewrite classic missing values results for this setting. We then show the consistency of two approaches, test-time multiple imputation and single imputation in prediction. A striking result is that the widely-used method of imputing with a constant prior to learning is consistent when missing values are not informative. This contrasts with inferential settings where mean imputation is frowned upon as it distorts the distribution of the data. The consistency of such a popular simple approach is important in practice. Finally, to contrast procedures based on imputation prior to learning with procedures that optimize the missing-value handling for prediction, we consider decision trees. Indeed, decision trees are among the few methods that can tackle empirical risk minimization with missing values, due to their ability to handle the half-discrete nature of incomplete variables. After comparing empirically different missing values strategies in trees, we recommend using the “missing incorporated in attribute” method as it can handle both non-informative and informative missing values.

在许多应用环境中,数据会有缺失项,这给后续分析带来了挑战。有大量文献在推理框架中处理缺失值问题,目的是从不完整的表格中估计参数及其方差。在这里,我们考虑的是监督学习环境:当训练数据和测试数据中都出现缺失值时预测目标。我们首先重写了这种情况下的经典缺失值结果。然后,我们展示了两种方法的一致性,即预测中的测试时间多重估算和单一估算。一个惊人的结果是,当缺失值不具有信息性时,广泛使用的在学习前使用常数估算的方法是一致的。这与推断环境形成了鲜明对比,在推断环境中,平均估算会扭曲数据的分布,因此受到人们的鄙视。这种流行的简单方法的一致性在实践中非常重要。最后,为了将基于学习前估算的程序与优化缺失值处理以进行预测的程序进行对比,我们考虑了决策树。事实上,决策树是少数几种能够处理缺失值的经验风险最小化的方法之一,这是因为决策树能够处理不完全变量的半离散性质。在对决策树中不同的缺失值策略进行经验比较后,我们推荐使用 "属性缺失并入 "方法,因为它既能处理非信息性缺失值,也能处理信息性缺失值。
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引用次数: 0
Maximum likelihood estimation for left-truncated log-logistic distributions with a given truncation point 对给定截断点的左截断对数-逻辑分布进行最大似然估计
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-10 DOI: 10.1007/s00362-024-01603-8
Markus Kreer, Ayşe Kızılersü, Jake Guscott, Lukas Christopher Schmitz, Anthony W. Thomas

For a sample (X_1, X_2,ldots X_N) of independent identically distributed copies of a log-logistically distributed random variable X the maximum likelihood estimation is analysed in detail if a left-truncation point (x_L>0) is introduced. Due to scaling properties it is sufficient to investigate the case (x_L=1). Here the corresponding maximum likelihood equations for a normalised sample (i.e. a sample divided by (x_L)) do not always possess a solution. A simple criterion guarantees the existence of a solution: Let (mathbb {E}(cdot )) denote the expectation induced by the normalised sample and denote by (beta _0=mathbb {E}(ln {X})^{-1}), the inverse value of expectation of the logarithm of the sampled random variable X (which is greater than (x_L=1)). If this value (beta _0) is bigger than a certain positive number (beta _C) then a solution of the maximum likelihood equation exists. Here the number (beta _C) is the unique solution of a moment equation,(mathbb {E}(X^{-beta _C})=frac{1}{2}). In the case of existence a profile likelihood function can be constructed and the optimisation problem is reduced to one dimension leading to a robust numerical algorithm. When the maximum likelihood equations do not admit a solution for certain data samples, it is shown that the Pareto distribution is the (L^1)-limit of the degenerated left-truncated log-logistic distribution, where (L^1(mathbb {R}^+)) is the usual Banach space of functions whose absolute value is Lebesgue-integrable. A large sample analysis showing consistency and asymptotic normality complements our analysis. Finally, two applications to real world data are presented.

对于对数逻辑分布随机变量 X 的独立同分布副本的样本 (X_1,X_2,ldotsX_N),如果引入一个左截断点 (x_L>0),就可以详细分析最大似然估计。由于缩放特性,研究 (x_L=1)的情况就足够了。在这里,归一化样本(即样本除以 (x_L))的相应最大似然方程并不总是有解。一个简单的标准可以保证解的存在:让 (mathbb {E}(cdot )) 表示归一化样本引起的期望值,用 (beta _0=mathbb {E}(ln {X})^{-1})表示采样随机变量 X 的对数(大于 (x_L=1))的反期望值。如果这个值(beta _0)大于某个正数(beta _C),那么就存在最大似然方程的解。这里的数(beta _C)是矩方程的唯一解,(mathbb {E}(X^{-beta _C})=frac{1}{2})。在存在的情况下,可以构建一个轮廓似然函数,并将优化问题简化为一个维度,从而产生一种稳健的数值算法。当最大似然方程对某些数据样本不允许求解时,可以证明帕累托分布是退化的左截断对数-逻辑分布的 (L^1)-limit ,其中 (L^1(mathbb {R}^+)) 是绝对值可被勒贝格积分的函数的通常巴拿赫空间。大样本分析显示了一致性和渐近正态性,补充了我们的分析。最后,我们介绍了现实世界数据的两个应用。
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引用次数: 0
Confidence bounds for compound Poisson process 复合泊松过程的置信区间
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-09-05 DOI: 10.1007/s00362-024-01604-7
Marek Skarupski, Qinhao Wu

The compound Poisson process (CPP) is a common mathematical model for describing many phenomena in medicine, reliability theory and risk theory. However, in the case of low-frequency phenomena, we are often unable to collect a sufficiently large database to conduct analysis. In this article, we focused on methods for determining confidence intervals for the rate of the CPP when the sample size is small. Based on the properties of process parameter estimators, we proposed a new method for constructing such intervals and compared it with other known approaches. In numerical simulations, we used synthetic data from several continuous and discrete distributions. The case of CPP, in which rewards came from exponential distribution, was discussed separately. The recommendation of how to use each method to have a more precise confidence interval is given. All simulations were performed in R version 4.2.1.

复合泊松过程(CPP)是描述医学、可靠性理论和风险理论中许多现象的常用数学模型。然而,对于低频现象,我们往往无法收集足够大的数据库来进行分析。在本文中,我们重点讨论了在样本量较小时确定 CPP 率置信区间的方法。基于过程参数估计器的特性,我们提出了一种构建此类区间的新方法,并将其与其他已知方法进行了比较。在数值模拟中,我们使用了几种连续和离散分布的合成数据。我们单独讨论了 CPP 的情况,其中奖励来自指数分布。我们给出了如何使用每种方法获得更精确置信区间的建议。所有模拟均在 R 4.2.1 版本中进行。
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引用次数: 0
Confidence intervals for overall response rate difference in the sequential parallel comparison design 顺序平行比较设计中总体答复率差异的置信区间
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-08-31 DOI: 10.1007/s00362-024-01606-5
Guogen Shan, Xinlin Lu, Yahui Zhang, Samuel S. Wu

High placebo responses could significantly reduce the treatment effect in a parallel randomized trial. To combat that challenge, several approaches were developed, including the sequential parallel comparison design (SPCD) that was shown to increase the statistical power as compared to the traditional randomized trial. A linear combination of the response rate differences from two phases per the SPCD is commonly used to measure the overall treatment effect size. The traditional approach to calculate the confidence interval for the overall rate difference is based on the delta method using the variance–covariance matrix of all outcomes. As outcomes from a multinomial distribution are correlated, we suggest utilizing a constrained variance–covariance matrix in the delta method. In the observation of anti-conservative coverages from asymptotic intervals, we further propose using importance sampling to develop accurate intervals. Simulation studies show that accurate intervals have better coverage probabilities than others and the interval width of accurate intervals is similar to the interval width of others. Two real trials to treat major depressive disorder are used to illustrate the application of the proposed intervals.

在平行随机试验中,高安慰剂反应可能会大大降低治疗效果。为了应对这一挑战,人们开发了多种方法,其中包括序列平行比较设计(SPCD),与传统的随机试验相比,该设计被证明可以提高统计功率。通常使用 SPCD 两个阶段响应率差异的线性组合来衡量总体治疗效果大小。计算总比率差异置信区间的传统方法是基于使用所有结果的方差-协方差矩阵的德尔塔法。由于多叉分布的结果具有相关性,我们建议在 delta 法中使用受约束的方差-协方差矩阵。在观察渐近区间的反保守覆盖率时,我们进一步建议使用重要性抽样来建立精确区间。模拟研究表明,精确区间比其他区间具有更好的覆盖概率,而且精确区间的区间宽度与其他区间的区间宽度相似。两个治疗重度抑郁障碍的真实试验用于说明所建议区间的应用。
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引用次数: 0
Bayesian and frequentist inference derived from the maximum entropy principle with applications to propagating uncertainty about statistical methods 从最大熵原理推导出的贝叶斯推理和频数推理,在传播统计方法的不确定性方面的应用
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-08-27 DOI: 10.1007/s00362-024-01597-3
David R. Bickel

Using statistical methods to analyze data requires considering the data set to be randomly generated from a probability distribution that is unknown but idealized according to a mathematical model consisting of constraints, assumptions about the distribution. Since the choice of such a model is up to the scientist, there is an understandable bias toward choosing models that make scientific conclusions appear more certain than they really are. There is a similar bias in the scientist’s choice of whether to use Bayesian or frequentist methods. This article provides tools to mitigate both of those biases on the basis of a principle of information theory. It is found that the same principle unifies Bayesianism with the fiducial version of frequentism. The principle arguably overcomes not only the main objections against fiducial inference but also the main Bayesian objection against the use of confidence intervals.

使用统计方法分析数据需要考虑到数据集是从一个未知的概率分布中随机生成的,但根据一个数学模型进行了理想化,该数学模型由有关分布的约束条件和假设组成。由于这种模型的选择是由科学家决定的,因此存在一种可以理解的偏差,即选择那些能使科学结论看起来比实际情况更确定的模型。科学家在选择使用贝叶斯方法还是频数方法时也存在类似的偏差。本文根据信息论原理,提供了减轻这两种偏差的工具。研究发现,同一原则将贝叶斯主义与频数主义的信条版本统一起来。可以说,该原理不仅克服了对信实推理的主要反对意见,也克服了贝叶斯主义对使用置信区间的主要反对意见。
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引用次数: 0
Reduced bias estimation of the log odds ratio 对数概率的减偏估计
IF 1.3 3区 数学 Q2 STATISTICS & PROBABILITY Pub Date : 2024-08-26 DOI: 10.1007/s00362-024-01593-7
Asma Saleh

Analysis of binary matched pairs data is problematic due to infinite maximum likelihood estimates of the log odds ratio and potentially biased estimates, especially for small samples. We propose a penalised version of the log-likelihood function based on adjusted responses which always results in a finite estimator of the log odds ratio. The probability limit of the adjusted log-likelihood estimator is derived and it is shown that in certain settings the maximum likelihood, conditional and modified profile log-likelihood estimators drop out as special cases of the former estimator. We implement indirect inference to the adjusted log-likelihood estimator. It is shown, through a complete enumeration study, that the indirect inference estimator is competitive in terms of bias and variance in comparison to the maximum likelihood, conditional, modified profile log-likelihood and Firth’s penalised log-likelihood estimators.

二元配对数据的分析存在问题,因为对数概率的最大似然估计值是无限的,而且估计值可能存在偏差,特别是对于小样本而言。我们提出了一种基于调整后响应的对数似然函数的惩罚版本,它总能得到对数几率比的有限估计值。我们推导出了调整后对数似然估计值的概率极限,并证明在某些情况下,最大似然估计值、条件似然估计值和修正的剖面对数似然估计值会作为前者的特例而消失。我们对调整后的对数似然估计器进行了间接推理。通过完整的枚举研究表明,就偏差和方差而言,间接推理估计器与最大似然估计器、条件估计器、修正轮廓对数似然估计器和 Firth 惩罚对数似然估计器相比具有竞争力。
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引用次数: 0
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