No Arabic abstract
The Youden index is a popular summary statistic for receiver operating characteristic curve. It gives the optimal cutoff point of a biomarker to distinguish the diseased and healthy individuals. In this paper, we propose to model the distributions of a biomarker for individuals in the healthy and diseased groups via a semiparametric density ratio model. Based on this model, we use the maximum empirical likelihood method to estimate the Youden index and the optimal cutoff point. We further establish the asymptotic normality of the proposed estimators and construct valid confidence intervals for the Youden index and the corresponding optimal cutoff point. The proposed method automatically covers both cases when there is no lower limit of detection (LLOD) and when there is a fixed and finite LLOD for the biomarker. Extensive simulation studies and a real data example are used to illustrate the effectiveness of the proposed method and its advantages over the existing methods.
The density ratio model (DRM) provides a flexible and useful platform for combining information from multiple sources. In this paper, we consider statistical inference under two-sample DRMs with additional parameters defined through and/or additional auxiliary information expressed as estimating equations. We examine the asymptotic properties of the maximum empirical likelihood estimators (MELEs) of the unknown parameters in the DRMs and/or defined through estimating equations, and establish the chi-square limiting distributions for the empirical likelihood ratio (ELR) statistics. We show that the asymptotic variance of the MELEs of the unknown parameters does not decrease if one estimating equation is dropped. Similar properties are obtained for inferences on the cumulative distribution function and quantiles of each of the populations involved. We also propose an ELR test for the validity and usefulness of the auxiliary information. Simulation studies show that correctly specified estimating equations for the auxiliary information result in more efficient estimators and shorter confidence intervals. Two real-data examples are used for illustrations.
Skepticism about the assumption of no unmeasured confounding, also known as exchangeability, is often warranted in making causal inferences from observational data; because exchangeability hinges on an investigators ability to accurately measure covariates that capture all potential sources of confounding. In practice, the most one can hope for is that covariate measurements are at best proxies of the true underlying confounding mechanism operating in a given observational study. In this paper, we consider the framework of proximal causal inference introduced by Tchetgen Tchetgen et al. (2020), which while explicitly acknowledging covariate measurements as imperfect proxies of confounding mechanisms, offers an opportunity to learn about causal effects in settings where exchangeability on the basis of measured covariates fails. We make a number of contributions to proximal inference including (i) an alternative set of conditions for nonparametric proximal identification of the average treatment effect; (ii) general semiparametric theory for proximal estimation of the average treatment effect including efficiency bounds for key semiparametric models of interest; (iii) a characterization of proximal doubly robust and locally efficient estimators of the average treatment effect. Moreover, we provide analogous identification and efficiency results for the average treatment effect on the treated. Our approach is illustrated via simulation studies and a data application on evaluating the effectiveness of right heart catheterization in the intensive care unit of critically ill patients.
The Gini index is a popular inequality measure with many applications in social and economic studies. This paper studies semiparametric inference on the Gini indices of two semicontinuous populations. We characterize the distribution of each semicontinuous population by a mixture of a discrete point mass at zero and a continuous skewed positive component. A semiparametric density ratio model is then employed to link the positive components of the two distributions. We propose the maximum empirical likelihood estimators of the two Gini indices and their difference, and further investigate the asymptotic properties of the proposed estimators. The asymptotic results enable us to construct confidence intervals and perform hypothesis tests for the two Gini indices and their difference. We show that the proposed estimators are more efficient than the existing fully nonparametric estimators. The proposed estimators and the asymptotic results are also applicable to cases without excessive zero values. Simulation studies show the superiority of our proposed method over existing methods. Two real-data applications are presented using the proposed methods.
There is a wide range of applications where the local extrema of a function are the key quantity of interest. However, there is surprisingly little work on methods to infer local extrema with uncertainty quantification in the presence of noise. By viewing the function as an infinite-dimensional nuisance parameter, a semiparametric formulation of this problem poses daunting challenges, both methodologically and theoretically, as (i) the number of local extrema may be unknown, and (ii) the induced shape constraints associated with local extrema are highly irregular. In this article, we address these challenges by suggesting an encompassing strategy that eliminates the need to specify the number of local extrema, which leads to a remarkably simple, fast semiparametric Bayesian approach for inference on local extrema. We provide closed-form characterization of the posterior distribution and study its large sample behaviors under this encompassing regime. We show a multi-modal Bernstein-von Mises phenomenon in which the posterior measure converges to a mixture of Gaussians with the number of components matching the underlying truth, leading to posterior exploration that accounts for multi-modality. We illustrate the method through simulations and a real data application to event-related potential analysis.
In this paper, we propose new semiparametric procedures for making inference on linear functionals and their functions of two semicontinuous populations. The distribution of each population is usually characterized by a mixture of a discrete point mass at zero and a continuous skewed positive component, and hence such distribution is semicontinuous in the nature. To utilize the information from both populations, we model the positive components of the two mixture distributions via a semiparametric density ratio model. Under this model setup, we construct the maximum empirical likelihood estimators of the linear functionals and their functions, and establish the asymptotic normality of the proposed estimators. We show the proposed estimators of the linear functionals are more efficient than the fully nonparametric ones. The developed asymptotic results enable us to construct confidence regions and perform hypothesis tests for the linear functionals and their functions. We further apply these results to several important summary quantities such as the moments, the mean ratio, the coefficient of variation, and the generalized entropy class of inequality measures. Simulation studies demonstrate the advantages of our proposed semiparametric method over some existing methods. Two real data examples are provided for illustration.