Biased sampling designs can be highly efficient when studying rare (binary) or low variability (continuous) endpoints. We consider longitudinal data settings in which the probability of being sampled depends on a repeatedly measured response through an outcome-related, auxiliary variable. Such auxiliary variable- or outcome-dependent sampling improves observed response and possibly exposure variability over random sampling, {even though} the auxiliary variable is not of scientific interest. {For analysis,} we propose a generalized linear model based approach using a sequence of two offsetted regressions. The first estimates the relationship of the auxiliary variable to response and covariate data using an offsetted logistic regression model. The offset hinges on the (assumed) known ratio of sampling probabilities for different values of the auxiliary variable. Results from the auxiliary model are used to estimate observation-specific probabilities of being sampled conditional on the response and covariates, and these probabilities are then used to account for bias in the second, target population model. We provide asymptotic standard errors accounting for uncertainty in the estimation of the auxiliary model, and perform simulation studies demonstrating substantial bias reduction, correct coverage probability, and improved design efficiency over simple random sampling designs. We illustrate the approaches with two examples.
Longitudinal and survival sub-models are two building blocks for joint modelling of longitudinal and time to event data. Extensive research indicates separate analysis of these two processes could result in biased outputs due to their associations. Conditional independence between measurements of biomarkers and event time process given latent classes or random effects is a common approach for characterising the association between the two sub-models while taking the heterogeneity among the population into account. However, this assumption is tricky to validate because of the unobservable laten…
Dropout represents a typical issue to be addressed when dealing with longitudinal studies. If the mechanism leading to missing information is non-ignorable, inference based on the observed data only may be severely biased. A frequent strategy to obtain reliable parameter estimates is based on the use of individual-specific random coefficients that help capture sources of unobserved heterogeneity and, at the same time, define a reasonable structure of dependence between the longitudinal and the missing data process. We refer to elements in this class as random coefficient based dropout models (…
In longitudinal panels and other regression models with unobserved effects, fixed effects estimation is often paired with cluster-robust variance estimation (CRVE) in order to account for heteroskedasticity and un-modeled dependence among the errors. CRVE is asymptotically consistent as the number of independent clusters increases, but can be biased downward for sample sizes often found in applied work, leading to hypothesis tests with overly liberal rejection rates. One solution is to use bias-reduced linearization (BRL), which corrects the CRVE so that it is unbiased under a working model, a…
Joint models for longitudinal and survival data have gained a lot of attention in recent years, with the development of myriad extensions to the basic model, including those which allow for multivariate longitudinal data, competing risks and recurrent events. Several software packages are now also available for their implementation. Although mathematically straightforward, the inclusion of multiple longitudinal outcomes in the joint model remains computationally difficult due to the large number of random effects required, which hampers the practical application of this extension. We present a…
Panel studies typically suffer from attrition, which reduces sample size and can result in biased inferences. It is impossible to know whether or not the attrition causes bias from the observed panel data alone. Refreshment samples - new, randomly sampled respondents given the questionnaire at the same time as a subsequent wave of the panel - offer information that can be used to diagnose and adjust for bias due to attrition. We review and bolster the case for the use of refreshment samples in panel studies. We include examples of both a fully Bayesian approach for analyzing the concatenated p…
Why recommended visit intervals should be extracted when conducting longitudinal analyses using electronic health record data: examining visit mechanism and sensitivity to assessment not at random
A joint model for (un)bounded longitudinal markers, competing risks, and recurrent events using patient registry data
Erasmus University Medical Center（伊拉斯姆斯大学医学中心）；Duke University（杜克大学）；Cincinnati Children’s Hospital Medical Center（辛辛那提儿童医院医疗中心）；University of Cincinnati（辛辛那提大学）
Adressing Separation: A Firth-corrected Joint Model for Longitudinal and Time-to-event Data with an Application on Dropout from Vocational Training
Georg-August University Göttingen（哥廷根乔治-奥古斯特大学）；Chair of Spatial Data Science and Statistical Learning（空间数据科学与统计学习教席）；Campus Institute Data Science (CIDAS)（校园数据科学研究所（CIDAS））
Gradient boosting from the field of statistical learning is widely known as a powerful framework for estimation and selection of predictor effects in various regression models by adapting concepts from classification theory. Current boosting approaches also offer methods accounting for random effects and thus enable prediction of mixed models for longitudinal and clustered data. However, these approaches include several flaws resulting in unbalanced effect selection with falsely induced shrinkage and a low convergence rate on the one hand and biased estimates of the random effects on the other…
Generalized estimating equations (GEE) are widely used to analyze longitudinal data; however, they are not appropriate for heteroscedastic data, because they only estimate regressor effects on the mean response{\textemdash}and therefore do not account for data heterogeneity. Here, we combine the GEE with the asymmetric least squares (expectile) regression to derive a new class of estimators, which we call generalized expectile estimating equations (GEEE). The GEEE model estimates regressor effects on the expectiles of the response distribution, which provides a detailed view of regressor effec…
Although increasingly used as a data resource for assembling cohorts, electronic health records (EHRs) pose many analytic challenges. In particular, a patient's health status influences when and what data are recorded, generating sampling bias in the collected data. In this paper, we consider recurrent event analysis using EHR data. Conventional regression methods for event risk analysis usually require the values of covariates to be observed throughout the follow-up period. In EHR databases, time-dependent covariates are intermittently measured during clinical visits, and the timing of these …
