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Full-Text Articles in Statistical Methodology

Variable Importance Analysis With The Multipim R Package, Stephan J. Ritter, Nicholas P. Jewell, Alan E. Hubbard Jul 2011

Variable Importance Analysis With The Multipim R Package, Stephan J. Ritter, Nicholas P. Jewell, Alan E. Hubbard

U.C. Berkeley Division of Biostatistics Working Paper Series

We describe the R package multiPIM, including statistical background, functionality and user options. The package is for variable importance analysis, and is meant primarily for analyzing data from exploratory epidemiological studies, though it could certainly be applied in other areas as well. The approach taken to variable importance comes from the causal inference field, and is different from approaches taken in other R packages. By default, multiPIM uses a double robust targeted maximum likelihood estimator (TMLE) of a parameter akin to the attributable risk. Several regression methods/machine learning algorithms are available for estimating the nuisance parameters of the models, including …


Targeted Maximum Likelihood Estimation: A Gentle Introduction, Susan Gruber, Mark J. Van Der Laan Aug 2009

Targeted Maximum Likelihood Estimation: A Gentle Introduction, Susan Gruber, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

This paper provides a concise introduction to targeted maximum likelihood estimation (TMLE) of causal effect parameters. The interested analyst should gain sufficient understanding of TMLE from this introductory tutorial to be able to apply the method in practice. A program written in R is provided. This program implements a basic version of TMLE that can be used to estimate the effect of a binary point treatment on a continuous or binary outcome.


Loss-Based Estimation With Evolutionary Algorithms And Cross-Validation, David Shilane, Richard H. Liang, Sandrine Dudoit Nov 2007

Loss-Based Estimation With Evolutionary Algorithms And Cross-Validation, David Shilane, Richard H. Liang, Sandrine Dudoit

U.C. Berkeley Division of Biostatistics Working Paper Series

Many statistical inference methods rely upon selection procedures to estimate a parameter of the joint distribution of explanatory and outcome data, such as the regression function. Within the general framework for loss-based estimation of Dudoit and van der Laan, this project proposes an evolutionary algorithm (EA) as a procedure for risk optimization. We also analyze the size of the parameter space for polynomial regression under an interaction constraints along with constraints on either the polynomial or variable degree.


Multiple Testing Procedures And Applications To Genomics, Merrill D. Birkner, Katherine S. Pollard, Mark J. Van Der Laan, Sandrine Dudoit Jan 2005

Multiple Testing Procedures And Applications To Genomics, Merrill D. Birkner, Katherine S. Pollard, Mark J. Van Der Laan, Sandrine Dudoit

U.C. Berkeley Division of Biostatistics Working Paper Series

This chapter proposes widely applicable resampling-based single-step and stepwise multiple testing procedures (MTP) for controlling a broad class of Type I error rates, in testing problems involving general data generating distributions (with arbitrary dependence structures among variables), null hypotheses, and test statistics (Dudoit and van der Laan, 2005; Dudoit et al., 2004a,b; van der Laan et al., 2004a,b; Pollard and van der Laan, 2004; Pollard et al., 2005). Procedures are provided to control Type I error rates defined as tail probabilities for arbitrary functions of the numbers of Type I errors, V_n, and rejected hypotheses, R_n. These error rates include: …


Data Adaptive Estimation Of The Treatment Specific Mean, Yue Wang, Oliver Bembom, Mark J. Van Der Laan Oct 2004

Data Adaptive Estimation Of The Treatment Specific Mean, Yue Wang, Oliver Bembom, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

An important problem in epidemiology and medical research is the estimation of the causal effect of a treatment action at a single point in time on the mean of an outcome, possibly within strata of the target population defined by a subset of the baseline covariates. Current approaches to this problem are based on marginal structural models, i.e., parametric models for the marginal distribution of counterfactural outcomes as a function of treatment and effect modifiers. The various estimators developed in this context furthermore each depend on a high-dimensional nuisance parameter whose estimation currently also relies on parametric models. Since misspecification …


History-Adjusted Marginal Structural Models And Statically-Optimal Dynamic Treatment Regimes, Mark J. Van Der Laan, Maya L. Petersen Sep 2004

History-Adjusted Marginal Structural Models And Statically-Optimal Dynamic Treatment Regimes, Mark J. Van Der Laan, Maya L. Petersen

U.C. Berkeley Division of Biostatistics Working Paper Series

Marginal structural models (MSM) provide a powerful tool for estimating the causal effect of a treatment. These models, introduced by Robins, model the marginal distributions of treatment-specific counterfactual outcomes, possibly conditional on a subset of the baseline covariates. Marginal structural models are particularly useful in the context of longitudinal data structures, in which each subject's treatment and covariate history are measured over time, and an outcome is recorded at a final time point. However, the utility of these models for some applications has been limited by their inability to incorporate modification of the causal effect of treatment by time-varying covariates. …


Loss-Based Cross-Validated Deletion/Substitution/Addition Algorithms In Estimation, Sandra E. Sinisi, Mark J. Van Der Laan Mar 2004

Loss-Based Cross-Validated Deletion/Substitution/Addition Algorithms In Estimation, Sandra E. Sinisi, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

In van der Laan and Dudoit (2003) we propose and theoretically study a unified loss function based statistical methodology, which provides a road map for estimation and performance assessment. Given a parameter of interest which can be described as the minimizer of the population mean of a loss function, the road map involves as important ingredients cross-validation for estimator selection and minimizing over subsets of basis functions the empirical risk of the subset-specific estimator of the parameter of interest, where the basis functions correspond to a parameterization of a specified subspace of the complete parameter space. In this article we …


Unified Cross-Validation Methodology For Selection Among Estimators And A General Cross-Validated Adaptive Epsilon-Net Estimator: Finite Sample Oracle Inequalities And Examples, Mark J. Van Der Laan, Sandrine Dudoit Nov 2003

Unified Cross-Validation Methodology For Selection Among Estimators And A General Cross-Validated Adaptive Epsilon-Net Estimator: Finite Sample Oracle Inequalities And Examples, Mark J. Van Der Laan, Sandrine Dudoit

U.C. Berkeley Division of Biostatistics Working Paper Series

In Part I of this article we propose a general cross-validation criterian for selecting among a collection of estimators of a particular parameter of interest based on n i.i.d. observations. It is assumed that the parameter of interest minimizes the expectation (w.r.t. to the distribution of the observed data structure) of a particular loss function of a candidate parameter value and the observed data structure, possibly indexed by a nuisance parameter. The proposed cross-validation criterian is defined as the empirical mean over the validation sample of the loss function at the parameter estimate based on the training sample, averaged over …


Locally Efficient Estimation Of Nonparametric Causal Effects On Mean Outcomes In Longitudinal Studies, Romain Neugebauer, Mark J. Van Der Laan Jul 2003

Locally Efficient Estimation Of Nonparametric Causal Effects On Mean Outcomes In Longitudinal Studies, Romain Neugebauer, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

Marginal Structural Models (MSM) have been introduced by Robins (1998a) as a powerful tool for causal inference as they directly model causal curves of interest, i.e. mean treatment-specific outcomes possibly adjusted for baseline covariates. Two estimators of the corresponding MSM parameters of interest have been proposed, see van der Laan and Robins (2002): the Inverse Probability of Treatment Weighted (IPTW) and the Double Robust (DR) estimators. A parametric MSM approach to causal inference has been favored since the introduction of MSM. It relies on correct specification of a parametric MSM to consistently estimate the parameter of interest using the IPTW …


Resampling-Based Multiple Testing: Asymptotic Control Of Type I Error And Applications To Gene Expression Data, Katherine S. Pollard, Mark J. Van Der Laan Jun 2003

Resampling-Based Multiple Testing: Asymptotic Control Of Type I Error And Applications To Gene Expression Data, Katherine S. Pollard, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

We define a general statistical framework for multiple hypothesis testing and show that the correct null distribution for the test statistics is obtained by projecting the true distribution of the test statistics onto the space of mean zero distributions. For common choices of test statistics (based on an asymptotically linear parameter estimator), this distribution is asymptotically multivariate normal with mean zero and the covariance of the vector influence curve for the parameter estimator. This test statistic null distribution can be estimated by applying the non-parametric or parametric bootstrap to correctly centered test statistics. We prove that this bootstrap estimated null …


Double Robust Estimation In Longitudinal Marginal Structural Models, Zhuo Yu, Mark J. Van Der Laan Jun 2003

Double Robust Estimation In Longitudinal Marginal Structural Models, Zhuo Yu, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

Consider estimation of causal parameters in a marginal structural model for the discrete intensity of the treatment specific counting process (e.g. hazard of a treatment specific survival time) based on longitudinal observational data on treatment, covariates and survival. We assume the sequential randomization assumption (SRA) on the treatment assignment mechanism and the so called experimental treatment assignment assumption which is needed to identify the causal parameters from the observed data distribution. Under SRA, the likelihood of the observed data structure factorizes in the auxiliary treatment mechanism and the partial likelihood consisting of the product over time of conditional distributions of …


Bivariate Current Status Data, Mark J. Van Der Laan, Nicholas P. Jewell Sep 2002

Bivariate Current Status Data, Mark J. Van Der Laan, Nicholas P. Jewell

U.C. Berkeley Division of Biostatistics Working Paper Series

In many applications, it is often of interest to estimate a bivariate distribution of two survival random variables. Complete observation of such random variables is often incomplete. If one only observes whether or not each of the individual survival times exceeds a common observed monitoring time C, then the data structure is referred to as bivariate current status data (Wang and Ding, 2000). For such data, we show that the identifiable part of the joint distribution is represented by three univariate cumulative distribution functions, namely the two marginal cumulative distribution functions, and the bivariate cumulative distribution function evaluated on the …


A New Partitioning Around Medoids Algorithm, Mark J. Van Der Laan, Katherine S. Pollard, Jennifer Bryan Feb 2002

A New Partitioning Around Medoids Algorithm, Mark J. Van Der Laan, Katherine S. Pollard, Jennifer Bryan

U.C. Berkeley Division of Biostatistics Working Paper Series

Kaufman & Rousseeuw (1990) proposed a clustering algorithm Partitioning Around Medoids (PAM) which maps a distance matrix into a specified number of clusters. A particularly nice property is that PAM allows clustering with respect to any specified distance metric. In addition, the medoids are robust representations of the cluster centers, which is particularly important in the common context that many elements do not belong well to any cluster. Based on our experience in clustering gene expression data, we have noticed that PAM does have problems recognizing relatively small clusters in situations where good partitions around medoids clearly exist. In this …


Maximum Likelihood Estimation Of Ordered Multinomial Parameters, Nicholas P. Jewell, John D. Kalbfleisch Oct 2001

Maximum Likelihood Estimation Of Ordered Multinomial Parameters, Nicholas P. Jewell, John D. Kalbfleisch

U.C. Berkeley Division of Biostatistics Working Paper Series

The pool-adjacent violator-algorithm (Ayer, et al., 1955) has long been known to give the maximum likelihood estimator of a series of ordered binomial parameters, based on an independent observation from each distribution (see Barlow et al., 1972). This result has immediate application to estimation of a survival distribution based on current survival status at a set of monitoring times. This paper considers an extended problem of maximum likelihood estimation of a series of ‘ordered’ multinomial parameters. By making use of variants of the pool adjacent violator algorithm, we obtain a simple algorithm to compute the maximum likelihood estimator and demonstrate …