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We provide a new flexible framework for inference with the instrumental variable model. Rather than using linear specifications, functions characterizing the effects of instruments and other explanatory variables are estimated using machine learning via Bayesian Additive Regression Trees (BART). Error terms and their distribution are inferred using Dirichlet Process mixtures. Simulated and real examples show that when the true functions are linear, little is lost. But when nonlinearities are present, dramatic improvements are obtained with virtually no manual tuning., Comment: 33 pages, 7 figures

Many time-to-event studies are complicated by the presence of competing risks. Such data are often analyzed using Cox models for the cause specific hazard function or Fine-Gray models for the subdistribution hazard. In practice regression relationships in competing risks data with either strategy are often complex and may include nonlinear functions of covariates, interactions, high-dimensional parameter spaces and nonproportional cause specific or subdistribution hazards. Model misspecification can lead to poor predictive performance. To address these issues, we propose a novel approach to flexible prediction modeling of competing risks data using Bayesian Additive Regression Trees (BART). We study the simulation performance in two-sample scenarios as well as a complex regression setting, and benchmark its performance against standard regression techniques as well as random survival forests. We illustrate the use of the proposed method on a recently published study of patients undergoing hematopoietic stem cell transplantation., Comment: 32 pages

Nonparametric regression models such as Bayesian Additive Regression Trees (BART) can be useful in fitting flexible functions of a set of covariates to a response, while accounting for nonlinearities and interactions. However, they are often cumbersome to interpret. Breaking down the function into additive components, if appropriate, could simplify the interpretation and improve the utility of the model. On the other hand, establishing nonadditivity can be useful in determining the need for individualized predictions and treatment selection. Testing additivity of single covariates in nonparametric regression models has been extensively studied. However, additivity assessment of nonparametric functions of disjoint sets of variables has not received as much attention. We propose a method for detection of nonadditivity of two disjoint sets of variables by fitting the sum of two BART models, each using its own set of variables. We then compare the pseudo marginal likelihood (PsML) of this sum-of- BARTs model vs. a single-BART model with all the variables together, in a ratio known as Pseudo Bayes Factor (PsBF). A special case of our method checks additivity between one variable of interest and another set of variables, where the additive model allows for direct interpretation of the variable of interest while adjusting for the remaining variables in a flexible, nonparametric manner. We extended the above approaches to allow a binary response using a logit link. We also propose a systematic way to design simulations that are used in additivity assessment. In simulation studies, PsBF showed better performance compared to out-of-sample prediction error in choosing the correct model, while avoiding computationally expensive cross-validation and providing an interpretable criterion for model selection. We applied our approach to two different examples with a continuous and binary outcomes., Comment: 33 pages, 8 figures

Individualized treatment rules (ITR) can improve health outcomes by recognizing that patients may respond differently to treatment and assigning therapy with the most desirable predicted outcome for each individual. Flexible and efficient prediction models are desired as a basis for such ITRs to handle potentially complex interactions between patient factors and treatment. Modern Bayesian semiparametric and nonparametric regression models provide an attractive avenue in this regard as these allow natural posterior uncertainty quantification of patient specific treatment decisions as well as the population wide value of the prediction-based ITR. In addition, via the use of such models, inference is also available for the value of the Optimal ITR. We propose such an approach and implement it using Bayesian Additive Regression Trees (BART) as this model has been shown to perform well in fitting nonparametric regression functions to continuous and binary responses, even with many covariates. It is also computationally efficient for use in practice. With BART we investigate a treatment strategy which utilizes individualized predictions of patient outcomes from BART models. Posterior distributions of patient outcomes under each treatment are used to assign the treatment that maximizes the expected posterior utility. We also describe how to approximate such a treatment policy with a clinically interpretable ITR, and quantify its expected outcome. The proposed method performs very well in extensive simulation studies in comparison with several existing methods. We illustrate the usage of the proposed method to identify an individualized choice of conditioning regimen for patients undergoing hematopoietic cell transplantation and quantify the value of this method of choice in relation to the Optimal ITR as well as non-individualized treatment strategies., Comment: 24 pages, 6 figures