A simulation study on the confidence interval procedures of some mean cumulative function estimators

Recurrence data are collected to study the recurrent events in biological, physical, and other systems. Quantities of interest include the mean cumulative number of events and the mean cumulative cost of the events. The mean cumulative function (MCF) can be estimated using non-parametric (NP) methods or by fitting parametric models, and many procedures have been suggested to construct the confidence intervals (CIs) for the MCF. This paper summarizes the results of a large simulation study that was designed to compare five CI procedures for both NP and parametric estimation. When performing parametric estimation, we assume the power law non-homogeneous Poisson process (NHPP) model. Our results include the evaluation of these procedures when they are used for window-observation recurrence data where recurrence histories of some systems are available only in observation windows with gaps in between.     

Asymptotic results for model robust regression

Since the mid 1980's many statisticians have studied methods for combining parametric and nonparametric models to improve the quality of fits in a regression problem. Notably Einsporn (1987) proposed the Model Robust Regression 1 estimate (MRRl) in which the parametric function, f, and the nonparametric functiong were combined in a straightforward fashion via the use of a mixing parameter, λ This technique was studied extensively atsmall samples and was shown to be quite effective at modeling various unusual functions. In this paper we have asymptotic results for the MRRl estimate in the case where λ is theoretically optimal, is asymptotically optimal and data driven, and is chosen with the PRESS statistic (Allen, 1971) We demonstrate that the MRRl estimate with λchosen by the PRESS statistic is slightly inferior asymptotically to the other two estimates, but, nevertheless possesses positive asymptotic qualities.     

A finite sample comparison of nonparametric estimates of the effective dose in quantal bioassay

To estimate the effective dose level ED_α in the common binary response model, several parametric and nonparametric estimators have been proposed in the literature. In the present article, we focus on nonparametric methods and present a detailed numerical comparison of four different approaches to estimate the ED_α nonparametrically. The methods are briefly reviewed and their finite sample properties are studied by means of a detailed simulation study. Moreover, a data example is presented to illustrate the different concepts.     

Semiparametric multiple kernel estimators and model diagnostics for count regression functions

Abstract

This study concerns semiparametric approaches to estimate discrete multivariate count regression functions. The semiparametric approaches investigated consist of combining discrete multivariate nonparametric kernel and parametric estimations such that (i) a prior knowledge of the conditional distribution of model response may be incorporated and (ii) the bias of the traditional nonparametric kernel regression estimator of Nadaraya-Watson may be reduced. We are precisely interested in combination of the two estimations approaches with some asymptotic properties of the resulting estimators. Asymptotic normality results were showed for nonparametric correction terms of parametric start function of the estimators. The performance of discrete semiparametric multivariate kernel estimators studied is illustrated using simulations and real count data. In addition, diagnostic checks are performed to test the adequacy of the parametric start model to the true discrete regression model. Finally, using discrete semiparametric multivariate kernel estimators provides a bias reduction when the parametric multivariate regression model used as start regression function belongs to a neighborhood of the true regression model.     

Estimation and inference for generalized semi-varying coefficient models

This paper is concerned with the estimation and inference in generalized semi-varying coefficient models. An orthogonal projection local quasi-likelihood estimation is investigated, which can easily be used to estimate the model parametric and nonparametric parts. Then an empirical likelihood logarithmic approach to construct the confidence regions/intervals of the nonparametric parts is developed. Under some mild conditions, the asymptotic properties of the resulting estimators are studied explicitly, respectively. Some simulation studies are carried out to examine the finite sample performance of the proposed methods. Finally, the methodologies are illustrated by a real data set.     

Consistent bandwidth selection for kernel binary regression

The use of nonparametric regression techniques for binary regression is a promising alternative to parametric methods. As in other nonparametric smoothing problems, the choice of smoothing parameter is critical to the performance of the estimator and the appearance of the resulting estimate. In this paper, we discuss the use of selection criteria based on estimates of squared prediction risk and show consistency and asymptotic normality of the selected bandwidths. The usefulness of the methods is explored on a data set and in a small simulation study.     

Nonparametric versus Parametric Estimation of the Cure Fraction Using Interval Censored Data

This article discusses estimation of the cure rate by means of the bounded cumulative hazard (BCH) model using interval censored data. The parametric and nonparametric estimation methods within the framework of the EM algorithm were employed for cure rate estimation and their results compared. The Turnbull estimator was used in the nonparametric estimation while in parametric method both the exponential and Weibull distributions were considered. We show via simulation that the nonparametric method is a viable alternative to the parametric one when the censoring rate is rapidly increasing.     

Prediction bands for the EDF of a future sample

We develop both nonparametric and parametric methods for obtaining prediction bands for the empirical distribution function (EDF) of a future sample. These methods yield simultaneous prediction intervals for all order statistics of the future sample, and they also correspond to tests for the two-sample problem. The nonparametric prediction bands correspond to the two-sample Kolmogorov-Smirnov test and related nonparametric tests, but the parametric prediction bands correspond to entirely new parametric two-sample tests. The parametric prediction bands tend to outperform the nonparametric bands when the parametric assumptions hold, but they may have true coverage probabilities well below their nominal levels when the parametric assumptions fail. A new computational algorithm is used to obtain critical values in the nonparametric case.     

Outlier Resistant Estimation in Difference-Based Semiparametric Partially Linear Models

Partially linear models are extensions of linear models that include a nonparametric function of some covariate allowing an adequate and more flexible handling of explanatory variables than in linear models. The difference-based estimation in partially linear models is an approach designed to estimate parametric component by using the ordinary least squares estimator after removing the nonparametric component from the model by differencing. However, it is known that least squares estimates do not provide useful information for the majority of data when the error distribution is not normal, particularly when the errors are heavy-tailed and when outliers are present in the dataset. This paper aims to find an outlier-resistant fit that represents the information in the majority of the data by robustly estimating the parametric and the nonparametric components of the partially linear model. Simulations and a real data example are used to illustrate the feasibility of the proposed methodology and to compare it with the classical difference-based estimator when outliers exist.     

Estimation of regression parameters in a semiparametric transformation model

A semiparametric approach to model skewed/heteroscedastic regression data is discussed. We work with a semiparametric transform-both-sides regression model, which contains a parametric regression function and a nonparametric transformation. This model is adequate when the relationship between the median response and the explanatory variable has been specified by a theoretical result or a previous empirical study. The transform-both-sides model with a parametric transformation has been studied extensively and applied successfully to a number data sets. Allowing a nonparametric transformation function increases the flexibility of the model. In this article, we estimate the nonparametric transformation function by the conditional kernel density approach developed by Wang and Ruppert (1995), and then use a pseudo-maximum likelihood estimator to estimate the regression parameters. This estimate of the regression parameters has not been studied previously. In this article, the asymptotic distribution of this pseudo-MLE is derived. We also show that when σ, the standard deviation of the error, goes to zero (small σ asymptotics), this estimator is adaptive. Adaptive means that the regression parameters are estimated as precisely as when the transformation is known exactly. A similar result holds in the parametric approaches of Carroll and Ruppert (1984) and Ruppert and Aldershof (1989). Simulated and real examples are provided to illustrate the performance of the proposed estimator for finite sample size.     

Nonparametric empirical Bayes method for comparison of treatment effects with application to stress urinary incontinence data

Hierarchical models are widely used in medical research to structure complicated models and produce statistical inferences. In a hierarchical model, observations are sampled conditional on some parameters and these parameters are sampled from a common prior distribution. Bayes and empirical Bayes (EB) methods have been effectively applied in analyzing these models. Despite many successes, parametric Bayes and EB methods may be sensitive to misspecification of prior distributions. In this paper, without specific restriction on the form of the prior distribution, we propose a nonparametric EB method to estimate the treatment effect of each group and develop a testing procedure to compare between-group differences. Simulation studies demonstrate that the proposed EB method was more efficient than some standard procedures. An illustrative example is provided with data from a clinical trial evaluating a new treatment for patients with stress urinary incontinence.     

Testing the Homogeneity of Two Survival Functions Against a Mixture Alternative Based on Censored Data

When the survival distribution in a treatment group is a mixture of two distributions of the same family, traditional parametric methods that ignore the existence of mixture components or the nonparametric methods may not be very powerful. We develop a modified likelihood ratio test (MLRT) for testing homogeneity in a two sample problem with censored data and compare the actual type I error and power of the MLRT with that nonparametric log-rank test and parametric test through Monte-Carlo simulations. The proposed test is also applied to analyze data from a clinical trial on early breast cancer.     

Statistical inference for a single-index varying-coefficient model

We investigate the estimators of parameters of interest for a single-index varying-coefficient model. To estimate the unknown parameter efficiently, we first estimate the nonparametric component using local linear smoothing, then construct an estimator of parametric component by using estimating equations. Our estimator for the parametric component is asymptotically efficient, and the estimator of nonparametric component has asymptotic normality and optimal uniform convergence rate. Our results provide ways to construct confidence regions for the involved unknown parameters. The finite-sample behavior of the new estimators is evaluated through simulation studies, and applications to two real data are illustrated.     

A new orthogonality-based estimation for varying-coefficient partially linear models

Varying coefficient partially linear models are usually used for longitudinal data analysis, and an interest is mainly to improve efficiency of regression coefficients. By the orthogonality estimation technology and the quadratic inference function method, we propose a new orthogonality-based estimation method to estimate parameter and nonparametric components in varying coefficient partially linear models with longitudinal data. The proposed procedure can separately estimate the parametric and nonparametric components, and the resulting estimators do not affect each other. Under some mild conditions, we establish some asymptotic properties of the resulting estimators. Furthermore, the finite sample performance of the proposed procedure is assessed by some simulation experiments.     

Fourth-order kernel method for simple linear degradation model

Degradation analysis is a useful technique when life tests result in few or even no failures. The degradation measurements are recorded over time and the estimation of time-to-failure distribution plays a vital role in degradation analysis. The parametric method to estimate the time-to-failure distribution assumed a specific parametric model with known shape for the random effects parameter. To avoid any assumption about the model shape, a nonparametric method can be used. In this paper, we suggest to use the nonparametric fourth-order kernel method to estimate the time-to-failure distribution and its percentiles for the simple linear degradation model. The performances of the proposed method are investigated and compared with the classical kernel; maximum likelihood and ordinary least squares methods via simulation technique. The numerical results show the good performance of the fourth-order kernel method and demonstrate its superiority over the parametric method when there is no information about the shape of the random effect parameter distribution.     

Semiparametric Analysis of Truncated Data

Randomly truncated data are frequently encountered in many studies where truncation arises as a result of the sampling design. In the literature, nonparametric and semiparametric methods have been proposed to estimate parameters in one-sample models. This paper considers a semiparametric model and develops an efficient method for the estimation of unknown parameters. The model assumes that K populations have a common probability distribution but the populations are observed subject to different truncation mechanisms. Semiparametric likelihood estimation is studied and the corresponding inferences are derived for both parametric and nonparametric components in the model. The method can also be applied to two-sample problems to test the difference of lifetime distributions. Simulation results and a real data analysis are presented to illustrate the methods.     

LASSO-type estimators for semiparametric nonlinear mixed-effects models estimation

Parametric nonlinear mixed effects models (NLMEs) are now widely used in biometrical studies, especially in pharmacokinetics research and HIV dynamics models, due to, among other aspects, the computational advances achieved during the last years. However, this kind of models may not be flexible enough for complex longitudinal data analysis. Semiparametric NLMEs (SNMMs) have been proposed as an extension of NLMEs. These models are a good compromise and retain nice features of both parametric and nonparametric models resulting in more flexible models than standard parametric NLMEs. However, SNMMs are complex models for which estimation still remains a challenge. Previous estimation procedures are based on a combination of log-likelihood approximation methods for parametric estimation and smoothing splines techniques for nonparametric estimation. In this work, we propose new estimation strategies in SNMMs. On the one hand, we use the Stochastic Approximation version of EM algorithm (SAEM) to obtain exact ML and REML estimates of the fixed effects and variance components. On the other hand, we propose a LASSO-type method to estimate the unknown nonlinear function. We derive oracle inequalities for this nonparametric estimator. We combine the two approaches in a general estimation procedure that we illustrate with simulations and through the analysis of a real data set of price evolution in on-line auctions.     

Parametric and nonparametric methods for confidence intervals and sample size planning for win probability in parallel-group randomized trials with Likert item and Likert scale data

Data on the Likert scale are ubiquitous in medical research, including randomized trials. Statistical analysis of such data may be conducted using the means of raw scores or the rank information of the scores. In the context of parallel-group randomized trials, we quantify treatment effects by the probability that a subject in the treatment group has a better score than (or a win over) a subject in the control group. Asymptotic parametric and nonparametric confidence intervals for this win probability and associated sample size formulas are derived for studies with only follow-up scores, and those with both baseline and follow-up measurements. We assessed the performance of both the parametric and nonparametric approaches using simulation studies based on real studies with Likert item and Likert scale data. The simulation results demonstrate that even without baseline adjustment, the parametric methods did not perform well, in terms of bias, interval coverage percentage, balance of tail error, and assurance of achieving a pre-specified precision. In contrast, the nonparametric approach performed very well for both the unadjusted and adjusted win probability. We illustrate the methods with two examples: one using Likert item data and the other using Like scale data. We conclude that non-parametric methods are preferable for two-group randomization trials with Likert data. Illustrative SAS code for the nonparametric approach using existing procedures is provided.     

Statistical analysis of right-censored failure-time data with partially specified hazard rates

This paper discusses the analysis of right-censored failure-time data in which the failure rate may have different forms in different time intervals. Such data occur naturally, for example, in demography studies and leukemia research, and a number of methods for the analysis have been proposed in the literature. However, most methods are purely parametric or nonparametric. Matthews and Farewell (1982), for example, discussed this problem and proposed a method for testing a constant failure rate against a failure rate involving a change point. To estimate an absolute limit on the attainable human life span, Zelterman (1992) discussed a hazard function that has different parametric forms over different time intervals. We consider a different situation in which the hazard function may follow a parametric form before a change point and is completely unknown after the change point. To test the existence of the change point, a modified maximal-censored-likelihood-ratio test is proposed and its asymptotic properties are studied. A bootstrap method is described for finding critical values of the proposed test. Simulation results indicate that the test performs well.     

Semiparametric estimation of the cumulative incidence function under competing risks and left-truncated sampling

In this article, we propose semiparametric methods to estimate the cumulative incidence function of two dependent competing risks for left-truncated and right-censored data. The proposed method is based on work by Huang and Wang (1995). We extend previous model by allowing for a general parametric truncation distribution and a third competing risk before recruitment. Based on work by Vardi (1989), several iterative algorithms are proposed to obtain the semiparametric estimates of cumulative incidence functions. The asymptotic properties of the semiparametric estimators are derived. Simulation results show that a semiparametric approach assuming the parametric truncation distribution is correctly specified produces estimates with smaller mean squared error than those obtained in a fully nonparametric model.