On the Kaplan–Meier estimator based on ranked set samples

When quantification of all sampling units is expensive but a set of units can be ranked, without formal measurement, ranked set sampling (RSS) is a cost-efficient alternate to simple random sampling (SRS). In this paper, we study the Kaplan–Meier estimator of survival probability based on RSS under random censoring time setup, and propose nonparametric estimators of the population mean. We present a simulation study to compare the performance of the suggested estimators. It turns out that RSS design can yield a substantial improvement in efficiency over the SRS design. Additionally, we apply the proposed methods to a real data set from an environmental study.     

Cost-efficient designs based on linearly associated biomarkers

A major limiting factor in much of the epidemiological and environmental researches is the cost of measuring the biomarkers or analytes of interest. Often, the number of specimens available for analysis is greater than the number of assays that is budgeted for. These assays are then performed on a random sample of specimens. Regression calibration is then utilized to infer biomarker levels of expensive assays from other correlated biomarkers that are relatively inexpensive to obtain and analyze. In other contexts, use of pooled specimens has been shown to increase efficiency in estimation. In this article, we examine two types of pooling in lieu of a random sample. The first is random (or traditional) pooling, and we characterize the second as “optimal” pooling. The second, which we propose for regression analysis, is pooling based on specimens ranked on the less expensive biomarker. The more expensive assay is then performed on the pool of relatively similar measurements. The optimal nature of this technique is also exemplified via Monte Carlo evaluations and real biomarker data. By displaying the considerable robustness of our method via a Monte Carlo study, it is shown that the proposed pooling design is a viable option whenever expensive assays are considered.     

Adaptive nonparametric estimation in the presence of dependence

We consider nonparametric estimation problems in the presence of dependent data, notably nonparametric regression with random design and nonparametric density estimation. The proposed estimation procedure is based on a dimension reduction. The minimax optimal rate of convergence of the estimator is derived assuming a sufficiently weak dependence characterised by fast decreasing mixing coefficients. We illustrate these results by considering classical smoothness assumptions. However, the proposed estimator requires an optimal choice of a dimension parameter depending on certain characteristics of the function of interest, which are not known in practice. The main issue addressed in our work is an adaptive choice of this dimension parameter combining model selection and Lepski's method. It is inspired by the recent work of Goldenshluger and Lepski [(2011), ‘Bandwidth Selection in Kernel Density Estimation: Oracle Inequalities and Adaptive Minimax Optimality’, The Annals of Statistics, 39, 1608–1632]. We show that this data-driven estimator can attain the lower risk bound up to a constant provided a fast decay of the mixing coefficients.     

Evaluating the ROC performance of markers for future events

Receiver operating characteristic (ROC) curves play a central role in the evaluation of biomarkers and tests for disease diagnosis. Predictors for event time outcomes can also be evaluated with ROC curves, but the time lag between marker measurement and event time must be acknowledged. We discuss different definitions of time-dependent ROC curves in the context of real applications. Several approaches have been proposed for estimation. We contrast retrospective versus prospective methods in regards to assumptions and flexibility, including their capacities to incorporate censored data, competing risks and different sampling schemes. Applications to two datasets are presented.     

A Permutation Approach to Split-Plot Experiments

Split-plot design may be refer to a common experimental setting where a particular type of restricted randomization has occurred during a planned experiment. The aim of this article is to suggest a new method to perform inference on split-plot experiments by combination-based permutation tests. This novel nonparametric approach has been studied and validated using a Monte Carlo simulation study where we compared it with the parametric and nonparametric procedures proposed in the literature. Results suggest that in each experimental situation where normality is hard to justify and especially when errors have heavy-tailed distribution, the proposed nonparametric procedure can be considered as a valid solution.     

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.     

Optimal Sample Designs With Preliminary Tests of Significance

A common problem faced in social experiments is that of designing a sampling strategy before it is known whether various control groups can be pooled. The classical sample choice is between a large supposedly unpoolable sample or a smaller supposedly poolable sample. This article suggests a compromise strategy between these two extremes based on preliminary tests of significance that allow one to embed judgments about the likelihood of pooling into a classical sample design problem.     

On a class of partially sequential two-sample test procedures for multivariate continuous data

In a two-sample testing problem, sometimes one of the sample observations are difficult and/or costlier to collect compared to the other one. Also, it may be the situation that sample observations from one of the populations have been previously collected and for operational advantages we do not wish to collect any more observations from the second population that are necessary for reaching a decision. Partially sequential technique is found to be very useful in such situations. The technique gained its popularity in statistics literature due to its very nature of capitalizing the best aspects of both fixed and sequential procedures. The literature is enriched with various types of partially sequential techniques useable under different types of data set-up. Nonetheless, there is no mention of multivariate data framework in this context, although very common in practice. The present paper aims at developing a class of partially sequential nonparametric test procedures for two-sample multivariate continuous data. For this we suggest a suitable stopping rule adopting inverse sampling technique and propose a class of test statistics based on the samples drawn using the suggested sampling scheme. Various asymptotic properties of the proposed tests are explored. An extensive simulation study is also performed to study the asymptotic performance of the tests. Finally the benefit of the proposed test procedure is demonstrated with an application to a real-life data on liver disease.     

Enhancement of the adaptive signature design for learning and confirming in a single pivotal trial

Because of the complexity of cancer biology, often the target pathway is not well understood at the time that phase III trials are initiated. A 2‐stage trial design was previously proposed for identifying a subgroup of interest in a learn stage, on the basis of 1 or more baseline biomarkers, and then subsequently confirming it in a confirmation stage. In this article, we discuss some practical aspects of this type of design and describe an enhancement to this approach that can be built into the study randomization to increase the robustness of the evaluation. Furthermore, we show via simulation studies how the proportion of patients allocated to the learn stage versus the confirm stage impacts the power and provide recommendations.     

Joint modeling of longitudinal and survival data with the Cox model and two-phase sampling

A common objective of cohort studies and clinical trials is to assess time-varying longitudinal continuous biomarkers as correlates of the instantaneous hazard of a study endpoint. We consider the setting where the biomarkers are measured in a designed sub-sample (i.e., case-cohort or two-phase sampling design), as is normative for prevention trials. We address this problem via joint models, with underlying biomarker trajectories characterized by a random effects model and their relationship with instantaneous risk characterized by a Cox model. For estimation and inference we extend the conditional score method of Tsiatis and Davidian (Biometrika 88(2):447–458, 2001) to accommodate the two-phase biomarker sampling design using augmented inverse probability weighting with nonparametric kernel regression. We present theoretical properties of the proposed estimators and finite-sample properties derived through simulations, and illustrate the methods with application to the AIDS Clinical Trials Group 175 antiretroviral therapy trial. We discuss how the methods are useful for evaluating a Prentice surrogate endpoint, mediation, and for generating hypotheses about biological mechanisms of treatment efficacy.     

A review of nonparametric ranked-set sampling methodology

Ranked-set sampling is an alternative to random sampling for settings in which measurements are difficult or costly. Ranked-set sampling utilizes information gained without measurement to structure the eventual measured sample. This additional information yields improved properties for ranked-set sample procedures relative to their simple random sample counterparts. We review the available nonparametric procedures for data from ranked-set samples. Estimation of the distribution function was the first nonparametric setting to which ranked-set sampling methodology was applied. Since the first paper on the ranked-set sample empirical distribution function, the two-sample location setting, the sign test, and the signed-rank test have all been examined for ranked-set samples. In addition, estimation of the distribution function has been considered in a more general setting. We discuss the similarities and differences in the properties of the ranked-set sample procedures for the various settings     

A maximin criterion for the logistic random intercept model with covariates

A maximin criterion is used to find optimal designs for the logistic random intercept model with dichotomous independent variables. The dichotomous independent variables can be subdivided into variables for which the distribution is specified prior to data sampling, called variates, and into variables for which the distribution is not specified prior to data sampling, but is obtained from data sampling, called covariates. The proposed maximin criterion maximizes the smallest possible relative efficiency not only with respect to all possible values of the model parameters, but also with respect to the joint distribution of the covariates. We have shown that, under certain conditions, the maximin design is balanced with respect to the joint distribution of the variates. The proposed method will be used to plan a (stratified) clinical trial where variates and covariates are involved.     

More powerful permutation test based on multistage ranked set sampling

Many researches have used ranked set sampling (RSS) method instead of simple random sampling (SRS) to improve power of some nonparametric tests. In this study, the two-sample permutation test within multistage ranked set sampling (MSRSS) is proposed and investigated. The power of this test is compared with the SRS permutation test for some symmetric and asymmetric distributions through Monte Carlo simulations. It has been found that this test is more powerful than the SRS permutation test; its power increased by set size and/or number of cycles and/or number of stages. Symmetric distributions power increased better than asymmetric distributions power.     

Design of kernel M-smoothers for spatial data

Robust nonparametric smoothers have been proved effective to preserve edges in image denoising. As an extension, they should be capable to estimate multivariate surfaces containing discontinuities on the basis of a random spatial sampling. A crucial problem is the design of their coefficients, in particular those of the kernels which concern robustness. In this paper it is shown that bandwidths which regard smoothness can consistently be estimated, whereas those which concern robustness cannot be estimated with plug-in and cross-validation criteria. Heuristic and graphical methods are proposed for their selection and their efficacy is proved in simulation experiments.     

Comparing Nonparametric Estimators for Length-Biased Data

Length-biased data appear when sampling lifetimes by cross-section. Right-censoring may affect the sampled information due to time limitation in following-up, lost to follow-up cases, etc. In this article, we compare by simulations two alternative nonparametric estimators of the lifetime distribution function when the data are length-biased and right-censored. These estimates, recently introduced in the literature, are based on nonparametric maximum-likelihood and moment-based principles. It is shown that the relative benefits associated to each estimator depend on several factors, such as the shape of the underlying distribution, sample size, or censoring level.     

A two-sample empirical likelihood ratio test based on samples entropy

Powerful entropy-based tests for normality, uniformity and exponentiality have been well addressed in the statistical literature. The density-based empirical likelihood approach improves the performance of these tests for goodness-of-fit, forming them into approximate likelihood ratios. This method is extended to develop two-sample empirical likelihood approximations to optimal parametric likelihood ratios, resulting in an efficient test based on samples entropy. The proposed and examined distribution-free two-sample test is shown to be very competitive with well-known nonparametric tests. For example, the new test has high and stable power detecting a nonconstant shift in the two-sample problem, when Wilcoxon’s test may break down completely. This is partly due to the inherent structure developed within Neyman-Pearson type lemmas. The outputs of an extensive Monte Carlo analysis and real data example support our theoretical results. The Monte Carlo simulation study indicates that the proposed test compares favorably with the standard procedures, for a wide range of null and alternative distributions.     

Improving The Efficiency of The Case-Crossover Design

The case-crossover design has been used by many researchers to study the transient effect of an exposure on the risk of a rare outcome. In a case-crossover design, only cases are sampled and each case will act as his/her own control. The time of failure acts as the case and non failure times act as the controls. Case-crossover designs have frequently been used to study the effect of environmental exposures on rare diseases or mortality. Time trends and seasonal confounding may be present in environmental studies and thus need to be controlled for by the sampling design. Several sampling methods are available for this purpose. In time-stratified sampling, disjoint strata of equal size are formed and the control times within the case stratum are used for comparison. The random semi-symmetric sampling design randomly selects a control time for comparison from two possible control times. The fixed semi-symmetric sampling design is a modified version of the random semi-symmetric sampling design that removes the random selection. Simulations show that the fixed semi-symmetric sampling design improves the variance of the random semi-symmetric sampling estimator by at least 35% for the exposures we studied. We derive expressions for the asymptotic variance of risk estimators for these designs, and show, that while the designs are not theoretically equivalent, in many realistic situations, the random semi-symmetric sampling design has similar efficiency to a time-stratified sampling design of size two and the fixed semi-symmetric sampling design has similar efficiency to a time-stratified sampling design of size three.     

A class of nonparametric bivariate survival function estimators for randomly censored and truncated data

This paper proposes a class of nonparametric estimators for the bivariate survival function estimation under both random truncation and random censoring. In practice, the pair of random variables under consideration may have certain parametric relationship. The proposed class of nonparametric estimators uses such parametric information via a data transformation approach and thus provides more accurate estimates than existing methods without using such information. The large sample properties of the new class of estimators and a general guidance of how to find a good data transformation are given. The proposed method is also justified via a simulation study and an application on an economic data set.     

NONPARAMETRIC ESTIMATION OF GENEWISE VARIANCE FOR MICROARRAY DATA

Estimation of genewise variance arises from two important applications in microarray data analysis: selecting significantly differentially expressed genes and validation tests for normalization of microarray data. We approach the problem by introducing a two-way nonparametric model, which is an extension of the famous Neyman-Scott model and is applicable beyond microarray data. The problem itself poses interesting challenges because the number of nuisance parameters is proportional to the sample size and it is not obvious how the variance function can be estimated when measurements are correlated. In such a high-dimensional nonparametric problem, we proposed two novel nonparametric estimators for genewise variance function and semiparametric estimators for measurement correlation, via solving a system of nonlinear equations. Their asymptotic normality is established. The finite sample property is demonstrated by simulation studies. The estimators also improve the power of the tests for detecting statistically differentially expressed genes. The methodology is illustrated by the data from MicroArray Quality Control (MAQC) project.     

Review of Statistical Filmstrip Programs

We develop a novel nonparametric likelihood ratio test for independence between two random variables using a technique that is free of the common constraints of defining a given set of specific dependence structures. Our methodology revolves around an exact density-based empirical likelihood ratio test statistic that approximates in a distribution-free fashion the corresponding most powerful parametric likelihood ratio test. We demonstrate that the proposed test is very powerful in detecting general structures of dependence between two random variables, including nonlinear and/or random-effect dependence structures. An extensive Monte Carlo study confirms that the proposed test is superior to the classical nonparametric procedures across a variety of settings. The real-world applicability of the proposed test is illustrated using data from a study of biomarkers associated with myocardial infarction. Supplementary materials for this article are available online.