The partly parametric and partly nonparametric additive risk model.

Aalen's linear hazard rate regression model is a useful and increasingly popular alternative to Cox' multiplicative hazard rate model. It postulates that an individual has hazard rate function [Formula: see text] in terms of his covariate values [Formula: see text]. These are typically levels of various hazard factors, and may also be time-dependent. The hazard factor functions [Formula: see text] are the parameters of the model and are estimated from data. This is traditionally accomplished in a fully nonparametric way. This paper develops methodology for estimating the hazard factor functions when some of them are modelled parametrically while the others are left unspecified. Large-sample results are reached inside this partly parametric, partly nonparametric framework, which also enables us to assess the goodness of fit of the model's parametric components. In addition, these results are used to pinpoint how much precision is gained, using the parametric-nonparametric model, over the standard nonparametric method. A real-data application is included, along with a brief simulation study.

What price semiparametric Cox regression?

Cox's proportional hazards regression model is the standard method for modelling censored life-time data with covariates. In its standard form, this method relies on a semiparametric proportional hazards structure, leaving the baseline unspecified. Naturally, specifying a parametric model also for the baseline hazard, leading to fully parametric Cox models, will be more efficient when the parametric model is correct, or close to correct. The aim of this paper is two-fold. (a) We compare parametric and semiparametric models in terms of their asymptotic relative efficiencies when estimating different quantities. We find that for some quantities the gain of restricting the model space is substantial, while it is negligible for others. (b) To deal with such selection in practice we develop certain focused and averaged focused information criteria (FIC and AFIC). These aim at selecting the most appropriate proportional hazards models for given purposes. Our methodology applies also to the simpler case without covariates, when comparing Kaplan-Meier and Nelson-Aalen estimators to parametric counterparts. Applications to real data are also provided, along with analyses of theoretical behavioural aspects of our methods.

Fridge: Focused fine-tuning of ridge regression for personalized predictions.

Statistical prediction methods typically require some form of fine-tuning of tuning parameter(s), with K-fold cross-validation as the canonical procedure. For ridge regression, there exist numerous procedures, but common for all, including cross-validation, is that one single parameter is chosen for all future predictions. We propose instead to calculate a unique tuning parameter for each individual for which we wish to predict an outcome. This generates an individualized prediction by focusing on the vector of covariates of a specific individual. The focused ridge-fridge-procedure is introduced with a 2-part contribution: First we define an oracle tuning parameter minimizing the mean squared prediction error of a specific covariate vector, and then we propose to estimate this tuning parameter by using plug-in estimates of the regression coefficients and error variance parameter. The procedure is extended to logistic ridge regression by using parametric bootstrap. For high-dimensional data, we propose to use ridge regression with cross-validation as the plug-in estimate, and simulations show that fridge gives smaller average prediction error than ridge with cross-validation for both simulated and real data. We illustrate the new concept for both linear and logistic regression models in 2 applications of personalized medicine: predicting individual risk and treatment response based on gene expression data. The method is implemented in the R package fridge.

Hybrid combinations of parametric and empirical likelihoods.

This paper develops a hybrid likelihood (HL) method based on a compromise between parametric and nonparametric likelihoods. Consider the setting of a parametric model for the distribution of an observation Y with parameter θ. Suppose there is also an estimating function m(·, µ) identifying another parameter µ via Em(Y, µ) = 0, at the outset defined independently of the parametric model. To borrow strength from the parametric model while obtaining a degree of robustness from the empirical likelihood method, we formulate inference about θ in terms of the hybrid likelihood function Hn (θ) = Ln (θ)1-a Rn (µ(θ)) a . Here a ∈ [0,1) represents the extent of the compromise, Ln is the ordinary parametric likelihood for θ, Rn is the empirical likelihood function, and µ is considered through the lens of the parametric model. We establish asymptotic normality of the corresponding HL estimator and a version of the Wilks theorem. We also examine extensions of these results under misspecification of the parametric model, and propose methods for selecting the balance parameter a.

A new framework for semi-Markovian parametric multi-state models with interval censoring.

There are few computational and methodological tools available for the analysis of general multi-state models with interval censoring. Here, we propose a general framework for parametric inference with interval censored multi-state data. Our framework can accommodate any parametric model for the transition times, and covariates may be included in various ways. We present a general method for constructing the likelihood, which we have implemented in a ready-to-use R package, smms, available on GitHub. The R package also computes the required high-dimensional integrals in an efficient manner. Further, we explore connections between our modelling framework and existing approaches: our models fall under the class of semi-Markovian multi-state models, but with a different, and sparser parameterisation than what is often seen. We illustrate our framework through a dataset monitoring heart transplant patients. Finally, we investigate the effect of some forms of misspecification of the model assumptions through simulations.

Goodness-of-fit processes for logistic regression: simulation results.

In this paper we use simulations to compare the performance of new goodness-of-fit tests based on weighted statistical processes to three currently available tests: the Hosmer-Lemeshow decile-of-risk test; the Pearson chi-square, and the unweighted sum-of-squares tests. The simulations demonstrate that all tests have the correct size. The power for all tests to detect lack-of-fit due to an omitted quadratic term with a sample of size 100 is close to or exceeds 50 per cent to detect moderate departures from linearity and is over 90 per cent for these same alternatives for sample size 500. All tests have low power with sample size 100 to detect lack-of-fit due to an omitted interaction between a dichotomous and continuous covariate, while the power exceeds 80 per cent to detect extreme interaction with a sample size of 500. The power is low to detect any alternative link function with sample size 100 and for most alternative links for sample size 500. Only in the case of sample size 500 and an extremely asymmetric link function is the power over 80 per cent. The results from these simulations show that no single test, new or current, performs best in detecting lack-of-fit due to an omitted covariate or incorrect link function. However, one of the new weighted tests has power comparable to other tests in all settings simulated and had the highest power in the difficult case of an omitted interaction term. We illustrate the tests within the context of a model for factors associated with abstinence from drug use in a randomized trial of residential treatment programmes. We conclude the paper with a summary and specific recommendations for practice.