Statistical Inference of Adaptive Designs with Binary Responses

Adaptive designs of clinical trials are ethical alternatives when the traditional randomization becomes ethically infeasible in desperate medical situations. However, such a design creates a dependency among trial data and its statistical analysis becomes more complex than the analysis for traditional randomized clinical trials. In this article, we examine adaptive designs with dichotomous responses from two treatments and extend some commonly used statistical methods for independent data. Under a regularity condition, the estimated odds ratio and its logarithm are shown to follow asymptotically normal distributions. Moreover, the ordinary goodness-of-fit test statistic for two-by-two contingency tables with dependent data is shown to be asymptotically chi-square distributed. We also discuss the consistency of maximum likelihood estimators of the unknown parameters for a wide class of adaptive designs.     

Hypothesis Testing Problems in an Unbalanced Longitudinal Ophthalmology Study

ABSTRACT

The analysis of clustered data in a longitudinal ophthalmology study is complicated by correlations between repeatedly measured visual outcomes of paired eyes in a participant and missing observations due to the loss of follow-up. In the present article we consider hypothesis testing problems in an ophthalmology study, where eligible eyes are randomized to two treatments (when two eyes of a participant are eligible, the paired eyes are assigned to different treatments), and vision function outcomes are repeatedly measured over time. A large sample-based nonparametric test statistic and a nonparametric Bootstrap test analog are proposed for testing an interaction effect of two factors and testing an effect of a eye-specific factor within a level of the other person-specific factor on visual function outcomes. Both test statistics allow for missing observations, correlations between repeatedly measured outcomes on individual eyes, and correlations between repeatedly measured outcomes on both eyes of each participant. A simulation study shows that these proposed test statistics maintain nominal significance levels approximately and comparable powers to each other, as well as higher powers than the naive test statistic ignoring correlations between repeated bilateral measurements of both eyes in the same person. For illustration, we apply the proposed test statistics to the changes of visual field defect score in the Advanced Glaucoma Intervention Study.     

L1 for the simple linear regression model

By modifying the direct method to solve the overdetermined linear system we are able to present an algorithm for L₁ estimation which appears to be superior computationally to any other known algorithm for the simple linear regression problem.     

Kolmogorov-type tests of goodness-of-fit against stochastic ordering

This article addresses the problem of testing the null hypothesis H₀ that a random sample of size n is from a distribution with the completely specified continuous cumulative distribution function F_n(x). Kolmogorov-type tests for H₀ are based on the statistics C⁺ _n = Sup[F_n(x)?F₀(x)] and C^? _n=Sup[F₀(x)?F_n(x)], where F_n(x) is an empirical distribution function. Let F(x) be the true cumulative distribution function, and consider the ordered alternative H₁: F(x)≥F₀(x) for all x and with strict inequality for some x. Although it is natural to reject H₀ and accept H₁ if C ⁺ _n is large, this article shows that a test that is superior in some ways rejects F₀ and accepts H₁ if C^mdash _n is small. Properties of the two tests are compared based on theoretical results and simulated results.     

Smoothing techniques via the bezier curve

Although the Bezier curve is very popular in the area of computational graphics it has rarely been used by statisticians. In this paper we develop methods and techniques for use of the Bezier curve in estimation of density and regression function. Also, asymptotic mean integrated square error for both estimators are derived. Comparisons with kernel estimator are conducted using simulation.     

Sieve Estimation of Time-Varying Panel Data Models With Latent Structures

We propose a heterogeneous time-varying panel data model with a latent group structure that allows the coefficients to vary over both individuals and time. We assume that the coefficients change smoothly over time and form different unobserved groups. When treated as smooth functions of time, the individual functional coefficients are heterogeneous across groups but homogeneous within a group. We propose a penalized-sieve-estimation-based classifier-Lasso (C-Lasso) procedure to identify the individuals’ membership and to estimate the group-specific functional coefficients in a single step. The classification exhibits the desirable property of uniform consistency. The C-Lasso estimators and their post-Lasso versions achieve the oracle property so that the group-specific functional coefficients can be estimated as well as if the individuals’ membership were known. Several extensions are discussed. Simulations demonstrate excellent finite sample performance of the approach in both classification and estimation. We apply our method to study the heterogeneous trending behavior of GDP per capita across 91 countries for the period 1960–2012 and find four latent groups.     

Nonparametric estimation of quantile functions for randomly right censored data

In this paper we compare four nonparametric quantile function estimators for randomly right censored data: the Kaplan–Meier estimator, the linearly interpolated Kaplan–Meier estimator, the kernel-type survival function estimator, and the Bézier curve smoothing estimator. Also, we compare several kinds of confidence intervals of quantiles for four nonparametric quantile function estimators.     

Directional mixture models and optimal estimation of the mixing density

The authors develop consistent nonparametric estimation techniques for the directional mixing density. Classical spherical harmonics are used to adapt Euclidean techniques to this directional environment. Minimax rates of convergence are obtained for rotation ally invariant densities verifying various smoothness conditions. It is found that the differences in smoothness between the Laplace, the Gaussian and the von Mises‐Fisher distributions lead to contrasting inferential conclusions.