Physical Sciences and Mathematics Commons^™

Oracle And Multiple Robustness Properties Of Survey Calibration Estimator In Missing Response Problem, Kwun Chuen Gary Chan

UW Biostatistics Working Paper Series

In the presence of missing response, reweighting the complete case subsample by the inverse of nonmissing probability is both intuitive and easy to implement. However, inverse probability weighting is not efficient in general and is not robust against misspecification of the missing probability model. Calibration was developed by survey statisticians for improving efficiency of inverse probability weighting estimators when population totals of auxiliary variables are known and when inclusion probability is known by design. In missing data problem we can calibrate auxiliary variables in the complete case subsample to the full sample. However, the inclusion probability is unknown in general …

Modification And Improvement Of Empirical Likelihood For Missing Response Problem, Kwun Chuen Gary Chan

UW Biostatistics Working Paper Series

An empirical likelihood (EL) estimator was proposed by Qin and Zhang (2007) for a missing response problem under a missing at random assumption. They showed by simulation studies that the finite sample performance of EL estimator is better than some existing estimators. However, the empirical likelihood estimator does not have a uniformly smaller asymptotic variance than other estimators in general. We consider several modifications to the empirical likelihood estimator and show that the proposed estimator dominates the empirical likelihood estimator and several other existing estimators in terms of asymptotic efficiencies. The proposed estimator also attains the minimum asymptotic variance among …

Modification And Improvement Of Empirical Liklihood For Missing Response Problem, Gary Chan

UW Biostatistics Working Paper Series

An empirical likelihood (EL) estimator was proposed by Qin and Zhang (2007) for a missing response problem under a missing at random assumption. They showed by simulation studies that the finite sample performance of EL estimator is better than some existing estimators. However, the empirical likelihood estimator does not have a uniformly smaller asymptotic variance than other estimators in general. We consider several modifications to the empirical likelihood estimator and show that the proposed estimator dominates the empirical likelihood estimator and several other existing estimators in terms of asymptotic efficiencies. The proposed estimator also attains the minimum asymptotic variance among …

Efficient Measurement Error Correction With Spatially Misaligned Data, Adam A. Szpiro, Lianne Sheppard, Thomas Lumley

UW Biostatistics Working Paper Series

Association studies in environmental statistics often involve exposure and outcome data that are misaligned in space. A common strategy is to employ a spatial model such as universal kriging to predict exposures at locations with outcome data and then estimate a regression parameter of interest using the predicted exposures. This results in measurement error because the predicted exposures do not correspond exactly to the true values. We characterize the measurement error by decomposing it into Berkson-like and classical-like components. One correction approach is the parametric bootstrap, which is effective but computationally intensive since it requires solving a nonlinear optimization problem …

On Two-Stage Hypothesis Testing Procedures Via Asymptotically Independent Statistics, James Dai, Charles Kooperberg, Michael L. Leblanc, Ross Prentice

UW Biostatistics Working Paper Series

Kooperberg and LeBlanc (2008) proposed a two-stage testing procedure to screen for significant interactions in genome-wide association (GWA) studies by a soft threshold on marginal associations (MA), though its theoretical properties and generalization have not been elaborated. In this article, we discuss conditions that are required to achieve strong control of the Family-Wise Error Rate (FWER) by such procedures for low or high-dimensional hypothesis testing. We provide proof of asymptotic independence of marginal association statistics and interaction statistics in linear regression, logistic regression, and Cox proportional hazard models in a randomized clinical trial (RCT) with a rare event. In case-control …

On Two-Stage Hypothesis Testing Procedures Via Asymptotically Independent Statistics, James Y. Dai, Charles Kooperberg, Michael Leblanc, Ross L. Prentice

UW Biostatistics Working Paper Series

Kooperberg08 proposed a two-stage testing procedure to screen for significant interactions in genome-wide association (GWA) studies by a soft threshold on marginal associations (MA), though its theoretical properties and generalization have not been elaborated. In this article, we discuss conditions that are required to achieve strong control of the Family-Wise Error Rate (FWER) by such procedures for low or high-dimensional hypothesis testing. We provide proof of asymptotic independence of marginal association statistics and interaction statistics in linear regression, logistic regression, and Cox proportional hazard models in a randomized clinical trial (RCT) with a rare event. In case-control studies nested within …

Multi-State Life Tables, Equilibrium Prevalence, And Baseline Selection Bias, Paula Diehr, David Yanez

UW Biostatistics Working Paper Series

Consider a 3-state system with one absorbing state, such as Healthy, Sick, and Dead. If the system satisfies the 1-step Markov conditions, the prevalence of the Healthy state will converge to a value that is independent of the initial distribution. This equilibrium prevalence and its variance are known under the assumption of time homogeneity, and provided reasonable estimates in the time non-homogeneous systems studied. Here, we derived the equilibrium prevalence for a system with more than three states. Under time homogeneity, the equilibrium prevalence distribution was shown to be an eigenvector of a partition of the matrix of transition probabilities. …

Model-Robust Regression And A Bayesian `Sandwich' Estimator, Adam A. Szpiro, Kenneth M. Rice, Thomas Lumley

UW Biostatistics Working Paper Series

The published version of this paper in Annals of Applied Statistics (Vol. 4, No. 4 (2010), 2099–2113) is available from the journal web site at http://dx.doi.org/10.1214/10-AOAS362.

We present a new Bayesian approach to model-robust linear regression that leads to uncertainty estimates with the same robustness properties as the Huber-White sandwich estimator. The sandwich estimator is known to provide asymptotically correct frequentist inference, even when standard modeling assumptions such as linearity and homoscedasticity in the data-generating mechanism are violated. Our derivation provides a compelling Bayesian justification for using this simple and popular tool, and it also clarifies what is being estimated …

Asymptotic Properties Of The Sequential Empirical Roc And Ppv Curves, Joseph S. Koopmeiners, Ziding Feng

UW Biostatistics Working Paper Series

The receiver operating characteristic (ROC) curve, the positive predictive value (PPV) curve and the negative predictive value (NPV) curve are three common measures of performance for a diagnostic biomarker. The independent increments covariance structure assumption is common in the group sequential study design literature. Showing that summary measures of the ROC, PPV and NPV curves have an independent increments covariance structure will provide the theoretical foundation for designing group sequential diagnostic biomarker studies. The ROC, PPV and NPV curves are often estimated empirically to avoid assumptions about the distributional form of the biomarkers. In this paper we derive asymptotic theory …

Nonparametric And Semiparametric Analysis Of Current Status Data Subject To Outcome Misclassification, Victor G. Sal Y Rosas, James P. Hughes

UW Biostatistics Working Paper Series

In this article, we present nonparametric and semiparametric methods to analyze current status data subject to outcome misclassification. Our methods use nonparametric maximum likelihood estimation (NPMLE) to estimate the distribution function of the failure time when sensitivity and specificity may vary among subgroups. A nonparametric test is proposed for the two sample hypothesis testing. In regression analysis, we apply the Cox proportional hazard model and likelihood ratio based confidence intervals for the regression coefficients are proposed. Our methods are motivated and demonstrated by data collected from an infectious disease study in Seattle, WA.

Panel Count Data Regression With Informative Observation Times, Petra Buzkova

UW Biostatistics Working Paper Series

When patients are monitored for potentially recurrent events such as infections or tumor metastases, it is common for clinicians to ask patients to come back sooner for follow-up based on the results of the most recent exam. This means that subjects’ observation times will be irregular and related to subject-specific factors. Previously proposed methods for handling such panel count data assume that the dependence between the events process and the observation time process is time-invariant. This article considers situations where the observation times are predicted by time-varying factors, such as the outcome observed at the last visit or cumulative exposure. …

Bio-Creep In Non-Inferiority Clinical Trials, Siobhan P. Everson-Stewart, Scott S. Emerson

UW Biostatistics Working Paper Series

After a non-inferiority clinical trial, a new therapy may be accepted as effective, even if its treatment effect is slightly smaller than the current standard. It is therefore possible that, after a series of trials where the new therapy is slightly worse than the preceding drugs, an ineffective or harmful therapy might be incorrectly declared efficacious; this is known as “bio-creep.” Several factors may influence the rate at which bio-creep occurs, including the distribution of the effects of the new agents being tested and how that changes over time, the choice of active comparator, the method used to model the …

Estimates Of Information Growth In Longitudinal Clinical Trials, Abigail Shoben, Kyle Rudser, Scott S. Emerson

UW Biostatistics Working Paper Series

In group sequential clinical trials, it is necessary to estimate the amount of information present at interim analysis times relative to the amount of information that would be present at the final analysis. If only one measurement is made per individual, this is often the ratio of sample sizes available at the interim and final analyses. However, as discussed by Wu and Lan (1992), when the statistic of interest is a change over time, as with longitudinal data, such an approach overstates the information. In this paper, we discuss other problems that can result in overestimating the information, such as …

Robustness Of Approaches To Roc Curve Modeling Under Misspecification Of The Underlying Probability Model, Sean Devlin, Elizabeth Thomas, Scott S. Emerson

UW Biostatistics Working Paper Series

The receiver operating characteristic (ROC) curve is a tool of particular use in disease status classification with a continuous medical test (marker). A variety of statistical regression models have been proposed for the comparison of ROC curves for different markers across covariate groups. A full parametric modeling of the marker distribution has been generally found to be overly reliant on the strong parametric assumptions. Pepe (2003) has instead developed parametric models for the ROC curve that induce a semi-parametric model for the marker distributions. The estimating equations proposed for use in these ROC-GLM models may differ from commonly used estimating …

Exploring The Benefits Of Adaptive Sequential Designs In Time-To-Event Endpoint Settings, Sarah C. Emerson, Kyle Rudser, Scott S. Emerson

UW Biostatistics Working Paper Series

Sequential analysis is frequently employed to address ethical and financial issues in clinical trials. Sequential analysis may be performed using standard group sequential designs, or, more recently, with adaptive designs that use estimates of treatment effect to modify the maximal statistical information to be collected. In the general setting in which statistical information and clinical trial costs are functions of the number of subjects used, it has yet to be established whether there is any major efficiency advantage to adaptive designs over traditional group sequential designs. In survival analysis, however, statistical information (and hence efficiency) is most closely related to …