Joint modeling of longitudinal and survival data with the Cox model and two-phase sampling

A common objective of cohort studies and clinical trials is to assess time-varying longitudinal continuous biomarkers as correlates of the instantaneous hazard of a study endpoint. We consider the setting where the biomarkers are measured in a designed sub-sample (i.e., case-cohort or two-phase sampling design), as is normative for prevention trials. We address this problem via joint models, with underlying biomarker trajectories characterized by a random effects model and their relationship with instantaneous risk characterized by a Cox model. For estimation and inference we extend the conditional score method of Tsiatis and Davidian (Biometrika 88(2):447–458, 2001) to accommodate the two-phase biomarker sampling design using augmented inverse probability weighting with nonparametric kernel regression. We present theoretical properties of the proposed estimators and finite-sample properties derived through simulations, and illustrate the methods with application to the AIDS Clinical Trials Group 175 antiretroviral therapy trial. We discuss how the methods are useful for evaluating a Prentice surrogate endpoint, mediation, and for generating hypotheses about biological mechanisms of treatment efficacy.     

Additive mixed effect model for recurrent gap time data

Gap times between recurrent events are often of primary interest in medical and observational studies. The additive hazards model, focusing on risk differences rather than risk ratios, has been widely used in practice. However, the marginal additive hazards model does not take the dependence among gap times into account. In this paper, we propose an additive mixed effect model to analyze gap time data, and the proposed model includes a subject-specific random effect to account for the dependence among the gap times. Estimating equation approaches are developed for parameter estimation, and the asymptotic properties of the resulting estimators are established. In addition, some graphical and numerical procedures are presented for model checking. The finite sample behavior of the proposed methods is evaluated through simulation studies, and an application to a data set from a clinic study on chronic granulomatous disease is provided.     

Variable selection in discrete survival models including heterogeneity

Several variable selection procedures are available for continuous time-to-event data. However, if time is measured in a discrete way and therefore many ties occur models for continuous time are inadequate. We propose penalized likelihood methods that perform efficient variable selection in discrete survival modeling with explicit modeling of the heterogeneity in the population. The method is based on a combination of ridge and lasso type penalties that are tailored to the case of discrete survival. The performance is studied in simulation studies and an application to the birth of the first child.     

Term structure forecasting in affine framework with time-varying volatility

This study extends the affine Nelson–Siegel model by introducing the time-varying volatility component in the observation equation of yield curve, modeled as a standard EGARCH process. The model is illustrated in state-space framework and empirically compared to the standard affine and dynamic Nelson–Siegel model in terms of in-sample fit and out-of-sample forecast accuracy. The affine based extended model that accounts for time-varying volatility outpaces the other models for fitting the yield curve and produces relatively more accurate 6- and 12-month ahead forecasts, while the standard affine model comes with more precise forecasts for the very short forecast horizons. The study concludes that the standard and affine Nelson–Siegel models have higher forecasting capability than their counterpart EGARCH based models for the short forecast horizons, i.e., 1 month. The EGARCH based extended models have excellent performance for the medium and longer forecast horizons.     

$$D_s$$-optimality in copula models

Optimum experimental design theory has recently been extended for parameter estimation in copula models. The use of these models allows one to gain in flexibility by considering the model parameter set split into marginal and dependence parameters. However, this separation also leads to the natural issue of estimating only a subset of all model parameters. In this work, we treat this problem with the application of the \(D_s\)-optimality to copula models. First, we provide an extension of the corresponding equivalence theory. Then, we analyze a wide range of flexible copula models to highlight the usefulness of \(D_s\)-optimality in many possible scenarios. Finally, we discuss how the usage of the introduced design criterion also relates to the more general issue of copula selection and optimal design for model discrimination.     

Improved multivariate prediction regions for Markov process models

This paper concerns the specification of multivariate prediction regions which may be useful in time series applications whenever we aim at considering not just one single forecast but a group of consecutive forecasts. We review a general result on improved multivariate prediction and we use it in order to calculate conditional prediction intervals for Markov process models so that the associated coverage probability turns out to be close to the target value. This improved solution is asymptotically superior to the estimative one, which is simpler but it may lead to unreliable predictive conclusions. An application to general autoregressive models is presented, focusing in particular on AR and ARCH models.     

On the $$L_p$$ norms of kernel regression estimators for incomplete data with applications to classification

We consider kernel methods to construct nonparametric estimators of a regression function based on incomplete data. To tackle the presence of incomplete covariates, we employ Horvitz–Thompson-type inverse weighting techniques, where the weights are the selection probabilities. The unknown selection probabilities are themselves estimated using (1) kernel regression, when the functional form of these probabilities are completely unknown, and (2) the least-squares method, when the selection probabilities belong to a known class of candidate functions. To assess the overall performance of the proposed estimators, we establish exponential upper bounds on the \(L_p\) norms, \(1\le p<\infty \), of our estimators; these bounds immediately yield various strong convergence results. We also apply our results to deal with the important problem of statistical classification with partially observed covariates.     

A note on budget constraints and outside options in common agency

Bernheim and Whinston (Q J Econ 101:1–31, 1986) show that, in a common agency problem without budget constraints, the set of Nash equilibria with truthful strategies (TNE), the set of coalition-proof Nash equilibria (CPNE), and the principal-optimal core of the underlying coalitional game are non-empty and all equivalent in payoff space. We show that, with budget constraints, none of Bernheim and Whinston’s (Q J Econ 101:1–31, 1986) results hold: (i) a CPNE may not exist, (ii) a TNE may not exist even when a CPNE exists, (iii) a TNE may not be a CPNE, and (iv) both TNE and CPNE payoffs are core allocations but are not necessarily principal-optimal. However, when principals have outside options but no budget constraints, (i), and (iii) continue to hold but not for (ii) and (iv). In particular, a TNE always exists but the core may be empty.     

(Sub) Optimality and (non) optimal satisficing in risky decision experiments

We implement a risky choice experiment based on one-dimensional choice variables and risk neutrality induced via binary lottery incentives. Each participant confronts many parameter constellations with varying optimal payoffs. We assess (sub)optimality, as well as (non)optimal satisficing by eliciting aspirations in addition to choices. Treatments differ in the probability that a binary random event, which are payoff—but not optimal choice—relevant is experimentally induced and whether participants choose portfolios directly or via satisficing, i.e., by forming aspirations and checking for satisficing before making their choice. By incentivizing aspiration formation, we can test satisficing, and in cases of satisficing, determine whether it is optimal.     

When and how to satisfice: an experimental investigation

This paper is about satisficing behaviour. Rather tautologically, this is when decision-makers are satisfied with achieving some objective, rather than in obtaining the best outcome. The term was coined by Simon (Q J Econ 69:99–118, 1955), and has stimulated many discussions and theories. Prominent amongst these theories are models of incomplete preferences, models of behaviour under ambiguity, theories of rational inattention, and search theories. Most of these, however, seem to lack an answer to at least one of two key questions: when should the decision-maker (DM) satisfice; and how should the DM satisfice. In a sense, search models answer the latter question (in that the theory tells the DM when to stop searching), but not the former; moreover, usually the question as to whether any search at all is justified is left to a footnote. A recent paper by Manski (Theory Decis. doi: 10.1007/s11238-017-9592-1, 2017) fills the gaps in the literature and answers the questions: when and how to satisfice? He achieves this by setting the decision problem in an ambiguous situation (so that probabilities do not exist, and many preference functionals can therefore not be applied) and by using the Minimax Regret criterion as the preference functional. The results are simple and intuitive. This paper reports on an experimental test of his theory. The results show that some of his propositions (those relating to the ‘how’) appear to be empirically valid while others (those relating to the ‘when’) are less so.