Physical Sciences and Mathematics Commons^™

Statistical Methods For Assessing Drug Interactions And Identifying Effect Modifiers Using Observational Data., Qian Xu

Electronic Theses and Dissertations

This dissertation consists of three projects related to causal inference based on observational data. In the first project, we propose a double robust to identify the effect modifiers and estimate optimal treatment. Observational studies differ from experimental studies in that assignment of subjects to treatments is not randomized but rather occurs due to natural mechanisms, which are usually hidden from the researchers. Many statistical methods to identify the treatment effect and select the optimal personalized treatment for experimental studies may not be suitable for observational studies any more. In this project, we propose a exible outcome model to select the …

Measurements Of Generalizability And Adjustment For Bias In Clinical Trials, Yuanyuan Lu

USF Tampa Graduate Theses and Dissertations

While randomized controlled trials (RCTs) are widely used as a gold standard in clinical research and public health, they are criticized because of a potential lack of generalizability, as the trial patients may be unrepresentative of the target patient population. Few research addresses how to assess and evaluate the generalizability of RCTs. As we know, patients are rarely selected on a random basis from a well-defined patient population of interest into a clinical trial. Generalizing findings from the RCT samples to the patient population has begun to receive increasing attention. We simulate a patient population with treatment effect size of …

Inter-Retailer Channel Competition: Empirical Analyses Of Store Entry Effects On Online Purchases, Qian Tang, Mei Lin, Youngsoo Kim

Research Collection School Of Computing and Information Systems

This study empirically examines the effect of offline store entry on a competing online retailer in the footwear industry and investigates how this effect depends on the relative product assortment and price between the offline store and the online retailer. Using transaction data from a large online footwear retailer and offline store entry data from 19 major shoe retail chains and 3 department store chains, we quantify the entry effect of offline stores. Categorizing offline stores by assortment and price, we find that the entry of regular-price narrow-assortment stores generates a complementary effect that increases online purchases, while the entry …

Modified-Half-Normal Distribution And Different Methods To Estimate Average Treatment Effect., Jingchao Sun

Electronic Theses and Dissertations

This dissertation consists of three projects related to Modified-Half-Normal distribution and causal inference. In my first project, a new distribution called Modified-Half-Normal distribution was introduced. I explored a few of its distributional properties, the procedures for generating random samples based on Bayesian approaches, and the parameter estimation based on the method of moments. The second project deals with the problem of selection bias of average treatment effect (ATE) if we use the observational data. I combined the propensity score based inverse probability of treatment weighting (IPTW) method and the directed acyclic graph (DAG) to solve this problem. The third project …

Aspects Of Causal Inference., John A. Craycroft

Electronic Theses and Dissertations

Observational studies differ from experimental studies in that assignment of subjects to treatments is not randomized but rather occurs due to natural mechanisms, which are usually hidden from researchers. Yet objectives of the two studies are frequently the same: identify the causal – rather than merely associational – relationship between some treatment or exposure and an outcome. The statistical issues that arise in properly analyzing observational data for this goal are numerous and fascinating, and these issues are encompassed in the domain of causal inference. The research presented in this dissertation explores several distinct aspects of causal inference. This dissertation …

Investigating The Performance Of Propensity Score Approaches For Differential Item Functioning Analysis, Yan Liu, Chanmin Kim, Amrey D. Wu, Paul Gustafson, Edward Kroc, Bruno D. Zumbo

Journal of Modern Applied Statistical Methods

To evaluate the performance of propensity score approaches for differential item functioning analysis, this simulation study was conducted to assess bias, mean square error, Type I error, and power under different levels of effect size and a variety of model misspecification conditions, including different types and missing patterns of covariates.

Estimating Marginal Hazard Ratios By Simultaneously Using A Set Of Propensity Score Models: A Multiply Robust Approach, Di Shu, Peisong Han, Rui Wang, Sengwee Toh

Harvard University Biostatistics Working Paper Series

The inverse probability weighted Cox model is frequently used to estimate marginal hazard ratios. Its validity requires a crucial condition that the propensity score model is correctly specified. To provide protection against misspecification of the propensity score model, we propose a weighted estimation method rooted in empirical likelihood theory. The proposed estimator is multiply robust in that it is guaranteed to be consistent when a set of postulated propensity score models contains a correctly specified model. Our simulation studies demonstrate satisfactory finite sample performance of the proposed method in terms of consistency and efficiency. We apply the proposed method to …

Causal Effect Random Forest Of Interaction Trees For Learning Individualized Treatment Regimes In Observational Studies: With Applications To Education Study Data, Luo Li

CGU Theses & Dissertations

Learning individualized treatment regimes (ITR) using observational data holds great interest in various fields, as treatment recommendations based on individual characteristics may improve individual treatment benefits with a reduced cost. It has long been observed that different individuals may respond to a certain treatment with significant heterogeneity. ITR can be defined as a mapping between individual characteristics to a treatment assignment. The optimal ITR is the treatment assignment that maximizes expected individual treatment effects. Rooted from personalized medicine, many studies and applications of ITR are in medical fields and clinical practice. Heterogeneous responses are also well documented in educational interventions. …

Statistical Methods For Estimating And Testing Treatment Effect For Multiple Treatment Groups In Observational Studies., Xiaofang Yan

Electronic Theses and Dissertations

Note: Abstract would not save due to an issue with some of the characters.

Controlling For Confounding Via Propensity Score Methods Can Result In Biased Estimation Of The Conditional Auc: A Simulation Study, Hadiza I. Galadima, Donna K. Mcclish

Community & Environmental Health Faculty Publications

In the medical literature, there has been an increased interest in evaluating association between exposure and outcomes using nonrandomized observational studies. However, because assignments to exposure are not random in observational studies, comparisons of outcomes between exposed and nonexposed subjects must account for the effect of confounders. Propensity score methods have been widely used to control for confounding, when estimating exposure effect. Previous studies have shown that conditioning on the propensity score results in biased estimation of conditional odds ratio and hazard ratio. However, research is lacking on the performance of propensity score methods for covariate adjustment when estimating the …

Using Sensitivity Analyses For Unobserved Confounding To Address Covariate Measurement Error In Propensity Score Methods, Kara E. Rudolph, Elizabeth A. Stuart

Johns Hopkins University, Dept. of Biostatistics Working Papers

Propensity score methods are a popular tool to control for confounding in observational data, but their bias-reduction properties are threatened by covariate measurement error. There are few easy-to-implement methods to correct for such bias. We describe and demonstrate how existing sensitivity analyses for unobserved confounding---propensity score calibration, Vanderweele and Arah's bias formulas, and Rosenbaum's sensitivity analysis---can be adapted to address this problem. In a simulation study, we examined the extent to which these sensitivity analyses can correct for several measurement error structures: classical, systematic differential, and heteroscedastic covariate measurement error. We then apply these approaches to address covariate measurement error …

A Comparison Between Propensity Score Matching, Weighting, And Stratification In Multiple Treatment Groups: A Simulation Study, Priyalatha Govindasamy

Electronic Theses and Dissertations

The application of propensity score techniques (matching, stratification, and weighting) with multiple treatment levels are similar to those used in binary groups. However, given that the application of propensity scores in multiple treatment groups is new, factors affecting the performance of matching, stratification, and weighting in multiple treatment groups are less explored. Therefore, this study was conducted to determine the performance of different propensity score techniques with multiple treatment groups under various circumstances. Specifically, the study focused on examining how the three propensity score corrective techniques perform in estimating treatment effects under (1) overt and (2) hidden types of selection …

Contrails: Causal Inference Using Propensity Scores, Dean S. Barron

Journal of Modern Applied Statistical Methods

Contrails are clouds caused by airplane exhausts, which geologists contend decrease daily temperature ranges on Earth. Following the 2001 World Trade Center attack, cancelled domestic flights triggered the first absence of contrails in decades. Resultant exceptional data capacitated causal inference analysis by propensity score matching. Estimated contrail effect was 6.8981°F.

Showrooming Vs. Competing: How Does Brand Selection Matter?, Qian Tang, Mei Lin

Research Collection School Of Computing and Information Systems

In this study, we empirically examine the effect of local shoe store openings on the sales of a competing, major online shoe retailer. Both showrooming and competing effects can play a role: Under the showrooming effect, the local store opening can lead to more online sales for the online retailer, whereas the competing effect created by the local store opening can substitute away the demand for the online retailer. We examine when one effect dominates the other by classifying local stores into single- and mixed-brand stores. We find that the showrooming effect is dominant for a single-brand store opening, and …

Moving Towards Best Practice When Using Inverse Probability Of Treatment Weighting (Iptw) Using The Propensity Score To Estimate Causal Treatment Effects In Observational Studies, Peter Austin, Elizabeth Stuart

Peter Austin

The propensity score is defined as a subject’s probability of treatment selection, conditional on observed baseline covariates.Weighting subjects by the inverse probability of treatment received creates a synthetic sample in which treatment assignment is independent of measured baseline covariates. Inverse probability of treatment weighting (IPTW) using the propensity score allows one to obtain unbiased estimates of average treatment effects. However, these estimates are only valid if there are no residual systematic differences in observed baseline characteristics between treated and control subjects in the sample weighted by the estimated inverse probability of treatment. We report on a systematic literature review, in …

Optimal Full Matching For Survival Outcomes: A Method That Merits More Widespread Use, Peter Austin, Elizabeth Stuart

Peter Austin

Matching on the propensity score is a commonly used analytic method for estimating the effects of treatments on outcomes. Commonly used propensity score matching methods include nearest neighbor matching and nearest neighbor caliper matching. Rosenbaum (1991) proposed an optimal full matching approach, in which matched strata are formed consisting of either one treated subject and at least one control subject or one control subject and at least one treated subject. Full matching has been used rarely in the applied literature. Furthermore, its performance for use with survival outcomes has not been rigorously evaluated. We propose a method to use full …

Constrained Bayesian Estimation Of Inverse Probability Weights For Nonmonotone Missing Data, Baoluo Sun, Eric J. Tchetgen Tchetgen

Harvard University Biostatistics Working Paper Series

No abstract provided.

A Comparison Of 12 Algorithms For Matching On The Propensity Score, Peter C. Austin

Peter Austin

Propensity-score matching is increasingly being used to reduce the confounding that can occur in observational studies examining the effects of treatments or interventions on outcomes. We used Monte Carlo simulations to examine the following algorithms for forming matched pairs of treated and untreated subjects: optimal matching, greedy nearest neighbor matching without replacement, and greedy nearest neighbor matching without replacement within specified caliper widths. For each of the latter two algorithms, we examined four different sub-algorithms defined by the order in which treated subjects were selected for matching to an untreated subject: lowest to highest propensity score, highest to lowest propensity …

The Use Of Propensity Score Methods With Survival Or Time-To-Event Outcomes: Reporting Measures Of Effect Similar To Those Used In Randomized Experiments, Peter C. Austin

Peter Austin

Propensity score methods are increasingly being used to estimate causal treatment effects in observational studies. In medical and epidemiological studies, outcomes are frequently time-to-event in nature. Propensity-score methods are often applied incorrectly when estimating the effect of treatment on time-to-event outcomes. This article describes how two different propensity score methods (matching and inverse probability of treatment weighting) can be used to estimate the measures of effect that are frequently reported in randomized controlled trials: (i) marginal survival curves, which describe survival in the population if all subjects were treated or if all subjects were untreated; and (ii) marginal hazard ratios. …

The Use Of Bootstrapping When Using Propensity-Score Matching Without Replacement: A Simulation Study, Peter Austin, Dylan Small

Peter Austin

Propensity-score matching is frequently used to estimate the effect of treatments, exposures, and interventions when using observational data. An important issue when using propensity-score matching is how to estimate the standard error of the estimated treatment effect. Accurate variance estimation permits construction of confidence intervals that have the advertised coverage rates and tests of statistical significance that have the correct type I error rates. There is disagreement in the literature as to how standard errors should be estimated. The bootstrap is a commonly used resampling method that permits estimation of the sampling variability of estimated parameters. Bootstrap methods are rarely …

The Performance Of Different Propensity Score Methods For Estimating Absolute Effects Of Treatments On Survival Outcomes: A Simulation Study, Peter C. Austin

Peter Austin

Observational studies are increasingly being used to estimate the effect of treatments, interventions and exposures on outcomes that can occur over time. Historically, the hazard ratio, which is a relative measure of effect, has been reported. However, medical decision making is best informed when both relative and absolute measures of effect are reported. When outcomes are time-to-event in nature, the effect of treatment can also be quantified as the change in mean or median survival time due to treatment and the absolute reduction in the probability of the occurrence of an event within a specified duration of follow-up. We describe …

Double Propensity-Score Adjustment: A Solution To Design Bias Or Bias Due To Incomplete Matching, Peter Austin

Peter Austin

Propensity-score matching is frequently used to reduce the effects of confounding when using observational data to estimate the effects of treatments. Matching allows one to estimate the average effect of treatment in the treated. Rosenbaum and Rubin coined the term "bias due to incomplete matching" to describe the bias that can occur when some treated subjects are excluded from the matched sample because no appropriate control subject was available. The presence of incomplete matching raises important questions around the generalizability of estimated treatment effects to the entire population of treated subjects. We describe an analytic solution to address the bias …

Analysis Of Subgroup Data Of Clinical Trials, Kao-Tai Tsai, Karl E. Peace

Biostatistics Faculty Publications

Large randomized controlled clinical trials are the gold standard to evaluate and compare the effects of treatments. It is common practice for investigators to explore and even attempt to compare treatments, beyond the first round of primary analyses, for various subsets of the study populations based on scientific or clinical interests to take advantage of the potentially rich information contained in the clinical database. Although subjects are randomized to treatment groups in clinical trials, this does not imply the same degree of randomization among sub-populations of the original trials. Therefore, comparisons of treatments in sub-populations may not produce fair and …

Balancing Score Adjusted Targeted Minimum Loss-Based Estimation, Samuel D. Lendle, Bruce Fireman, Mark J. Van Der Laan

U.C. Berkeley Division of Biostatistics Working Paper Series

Adjusting for a balancing score is sufficient for bias reduction when estimating causal effects including the average treatment effect and effect among the treated. Estimators that adjust for the propensity score in a nonparametric way, such as matching on an estimate of the propensity score, can be consistent when the estimated propensity score is not consistent for the true propensity score but converges to some other balancing score. We call this property the balancing score property, and discuss a class of estimators that have this property. We introduce a targeted minimum loss-based estimator (TMLE) for a treatment specific mean with …

A Comparative Study Of Exact Versus Propensity Matching Techniques Using Monte Carlo Simulation, Mukaria J. J. Itang'ata

Dissertations

Often researchers face situations where comparative studies between two or more programs are necessary to make causal inferences for informed policy decision-making. Experimental designs employing randomization provide the strongest evidence for causal inferences. However, many pragmatic and ethical challenges may preclude the use of randomized designs. In such situations, subject matching provides an alternative design approach for conducting causal inference studies. This study examined various design conditions hypothesized to affect matching procedures’ bias recovery ability.

See attachment for full abstract.

A Targeted Confounder Selection Strategy For Propensity Score Estimation, Susan Gruber

Susan Gruber

These slides provide an introduction to data-adaptive propensity score estimation, and the collaborative targeted maximum likelihood estimator (C-TMLE) of van der Laan and Gruber. The notation has been greatly simplified, which makes the work accessible to a more general audience, but loses a little in the translation.

Using Ensemble-Based Methods For Directly Estimating Causal Effects: An Investigation Of Tree-Based G-Computation, Peter C. Austin

Peter Austin

Researchers are increasingly using observational or nonrandomized data to estimate causal treatment effects. Essential to the production of high-quality evidence is the ability to reduce or minimize the confounding that frequently occurs in observational studies. When using the potential outcome framework to define causal treatment effects, one requires the potential outcome under each possible treatment. However, only the outcome under the actual treatment received is observed, whereas the potential outcomes under the other treatments are considered missing data. Some authors have proposed that parametric regression models be used to estimate potential outcomes. In this study, we examined the use of …

Comparing Paired Vs. Non-Paired Statistical Methods Of Analyses When Making Inferences About Absolute Risk Reductions In Propensity-Score Matched Samples., Peter C. Austin

Peter Austin

Propensity-score matching allows one to reduce the effects of treatment-selection bias or confounding when estimating the effects of treatments when using observational data. Some authors have suggested that methods of inference appropriate for independent samples can be used for assessing the statistical significance of treatment effects when using propensity-score matching. Indeed, many authors in the applied medical literature use methods for independent samples when making inferences about treatment effects using propensity-score matched samples. Dichotomous outcomes are common in healthcare research. In this study, we used Monte Carlo simulations to examine the effect on inferences about risk differences (or absolute risk …

Optimal Caliper Widths For Propensity-Score Matching When Estimating Differences In Means And Differences In Proportions In Observational Studies., Peter C. Austin

Peter Austin

In a study comparing the effects of two treatments, the propensity score is the probability of assignment to one treatment conditional on a subject's measured baseline covariates. Propensity-score matching is increasingly being used to estimate the effects of exposures using observational data. In the most common implementation of propensity-score matching, pairs of treated and untreated subjects are formed whose propensity scores differ by at most a pre-specified amount (the caliper width). There has been a little research into the optimal caliper width. We conducted an extensive series of Monte Carlo simulations to determine the optimal caliper width for estimating differences …

A Tutorial And Case Study In Propensity Score Analysis: An Application To Estimating The Effect Of In-Hospital Smoking Cessation Counseling On Mortality, Peter C. Austin

Peter Austin

Propensity score methods allow investigators to estimate causal treatment effects using observational or nonrandomized data. In this article we provide a practical illustration of the appropriate steps in conducting propensity score analyses. For illustrative purposes, we use a sample of current smokers who were discharged alive after being hospitalized with a diagnosis of acute myocardial infarction. The exposure of interest was receipt of smoking cessation counseling prior to hospital discharge and the outcome was mortality with 3 years of hospital discharge. We illustrate the following concepts: first, how to specify the propensity score model; second, how to match treated and …