The receiver operating characteristic, or the ROC curve, is widely utilized to evaluate the diagnostic performance of a test, in other words, the accuracy of a test to discriminate normal cases from diseased cases. In the biomedical studies, we often meet with missing data, which the regular inference procedures cannot be applied to directly. In this thesis, the random hot deck imputation is used to obtain a 'complete' sample. Then empirical likelihood (EL) confidence intervals are constructed for ROC curves. The empirical log-likelihood ratio statistic is derived whose asymptotic distribution isproved to be a weighted chi-square distribution. The results of simulation study show that the EL confidence intervals perform well in terms of the coverage probability and the average length for various sample sizes and response rates.

In biomedical research and lifetime data analysis, the comparison of two hazard functions usually plays an important role in practice. In this thesis, we consider the standard independent two-sample framework under right censoring. We construct efficient and useful confidence intervals for the ratio and difference of two hazard functions using smoothed empirical likelihood methods. The empirical log-likelihood ratio is derived and its asymptotic distribution is a chi-squared distribution. Furthermore, the proposed method can be applied to medical diagnosis research. Simulation studies show that the proposed EL confidence intervals have better performance in terms of coverage accuracy and average length than the traditional normal approximation method. Finally, our methods are illustrated with real clinical trial data. It is concluded that the empirical likelihood methods provide better inferential outcomes.

In biomedical research and lifetime data analysis, the comparison of two hazard functions usually plays an important role in practice. In this thesis, we consider the standard independent two-sample framework under right censoring. We construct efficient and useful confidence intervals for the ratio and difference of two hazard functions using smoothed empirical likelihood methods. The empirical log-likelihood ratio is derived and its asymptotic distribution is a chi-squared distribution. Furthermore, the proposed method can be applied to medical diagnosis research. Simulation studies show that the proposed EL confidence intervals have better performance in terms of coverage accuracy and average length than the traditional normal approximation method. Finally, our methods are illustrated with real clinical trial data. It is concluded that the empirical likelihood methods provide better inferential outcomes.

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Master of Science / Department of Statistics / Paul I. Nelson / Quantiles are useful in describing distributions of component lifetimes. Data, consisting of the lifetimes of sample units, used to estimate quantiles are often censored. Right censoring, the setting investigated here, occurs, for example, when some test units may still be functioning when the experiment is terminated. This study investigated and compared the performance of parametric and nonparametric estimators of quantiles from right censored data generated from Weibull and Lognormal distributions, models which are commonly used in analyzing lifetime data. Parametric quantile estimators based on these assumed models were compared via simulation to each other and to quantile estimators obtained from the nonparametric Kaplan- Meier Estimator of the survival function. Various combinations of quantiles, censoring proportion, sample size, and distributions were considered. Our simulation show that the larger the sample size and the lower the censoring rate the better the performance of the estimates of the 5th percentile of Weibull data. The lognormal data are very sensitive to the censoring rate and we observed that for higher censoring rates the incorrect parametric estimates perform the best. If you do not know the underlying distribution of the data, it is risky to use parametric estimates of quantiles close to one. A limitation in using the nonparametric estimator of large quantiles is their instability when the censoring rate is high and the largest observations are censored. Key Words: Quantiles, Right Censoring, Kaplan-Meier estimator

In this thesis, we apply smoothed empirical likelihood method to investigate confidence intervals for the receiver operating characteristic (ROC) curve with right censoring. As a particular application of comparison of distributions from two populations, the ROC curve is constructed by the combination of cumulative distribution function and quantile function. Under mild conditions, the smoothed empirical likelihood ratio converges to chi-square distribution, which is the well-known Wilks's theorem. Furthermore, the performances of the empirical likelihood method are also illustrated by simulation studies in terms of coverage probability and average length of confidence intervals. Finally, a primary biliary cirrhosis data is used to illustrate the proposed empirical likelihood procedure.

In the dissertation, we study the statistical evaluation of treatment comparisons by evaluating the relative comparison of survival experiences between two treatment groups. We construct confidence interval and simultaneous confidence bands for the ratio and odds ratio of two survival functions through both parametric and nonparametric approaches.We first construct empirical likelihood confidence interval and simultaneous confidence bands for the odds ratio of two survival functions to address small sample efficacy and sufficiency. The empirical log-likelihood ratio is developed, and the corresponding asymptotic distribution is derived. Simulation studies show that the proposed empirical likelihood band has outperformed the normal approximation band in small sample size cases in the sense that it yields closer coverage probabilities to chosen nominal levels.Furthermore, in order to incorporate prognostic factors for the adjustment of survival functions in the comparison, we construct simultaneous confidence bands for the ratio and odds ratio of survival functions based on both the Cox model and the additive risk model. We develop simultaneous confidence bands by approximating the limiting distribution of cumulative hazard functions by zero-mean Gaussian processes whose distributions can be generated through Monte Carlo simulations. Simulation studies are conducted to evaluate the performance for proposed models. Real applications on published clinical trial data sets are also studied for further illustration purposes.In the end, the population attributable fraction function is studied to measure the impact of risk factors on disease incidence in the population. We develop semiparametric estimation of attributable fraction functions for cohort studies with potentially censored event time under the additive risk model.

In the dissertation, we study the statistical evaluation of treatment comparisons by evaluating the relative comparison of survival experiences between two treatment groups. We construct confidence interval and simultaneous confidence bands for the ratio and odds ratio of two survival functions through both parametric and nonparametric approaches.We first construct empirical likelihood confidence interval and simultaneous confidence bands for the odds ratio of two survival functions to address small sample efficacy and sufficiency. The empirical log-likelihood ratio is developed, and the corresponding asymptotic distribution is derived. Simulation studies show that the proposed empirical likelihood band has outperformed the normal approximation band in small sample size cases in the sense that it yields closer coverage probabilities to chosen nominal levels.Furthermore, in order to incorporate prognostic factors for the adjustment of survival functions in the comparison, we construct simultaneous confidence bands for the ratio and odds ratio of survival functions based on both the Cox model and the additive risk model. We develop simultaneous confidence bands by approximating the limiting distribution of cumulative hazard functions by zero-mean Gaussian processes whose distributions can be generated through Monte Carlo simulations. Simulation studies are conducted to evaluate the performance for proposed models. Real applications on published clinical trial data sets are also studied for further illustration purposes.In the end, the population attributable fraction function is studied to measure the impact of risk factors on disease incidence in the population. We develop semiparametric estimation of attributable fraction functions for cohort studies with potentially censored event time under the additive risk model.

In survival analysis, proportional hazards model is the most commonly used and the Cox model is the most popular. These models are developed to facilitate statistical analysis frequently encountered in medical research or reliability studies. In analyzing real data sets, checking the validity of the model assumptions is a key component. However, the presence of complicated types of censoring such as double censoring and partly interval-censoring in survival data makes model assessment difficult, and the existing tests for goodness-of-fit do not have direct extension to these complicated types of censored data. In this work, we use empirical likelihood (Owen, 1988) approach to construct goodness-of-fit test and provide estimates for the Cox model with various types of censored data. Specifically, the problems under consideration are the two-sample Cox model and stratified Cox model with right censored data, doubly censored data and partly interval-censored data. Related computational issues are discussed, and some simulation results are presented. The procedures developed in the work are applied to several real data sets with some discussion.