Statistics and Causal Inference
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Drug Exposure Definition and Healthy User Bias Impacts on the Evaluation of Oral Anti Hyperglycemic Therapies
Drug Exposure Definition and Healthy User Bias Impacts on the Evaluation of Oral Anti Hyperglycemic Therapies by Maxim Eskin A thesis submitted in partial fulfillment of the requirements for the degree of Master of Science in Health Policy Research School of Public Health University of Alberta © Maxim Eskin, 2017 ABSTRACT Accurate estimation of medication use is an essential component of any pharmacoepidemiological research as exposure misclassification will threaten study validity and lead to spurious associations. Many pharmacoepidemiological studies use simple definitions, such as the categorical “any versus no use” to classify oral antihyperglycemic medications exposure, which has potentially serious drawbacks. This approach has led to numerous highly publicized observational studies of metformin effect on health outcomes reporting exaggerated relationships that were later contradicted by randomized controlled trials. Although selection bias, unmeasured confounding, and many other factors contribute to the discrepancies, one critical element, which is often overlooked, is the method used to define exposure. Another factor that may provide additional explanation for the discrepancy between randomized controlled trials and observational study results of the association between metformin use and various health outcomes is the healthy user effect. Aspects of a healthy lifestyle such as following a balanced diet, exercising regularly, avoiding tobacco use, etc., are not recorded in typical administrative databases and failure to account for these factors in observational studies may introduce bias and produce spurious associations. The influence of a healthy user bias has not been fully examined when evaluating the effect of oral antihyperglycemic therapies in observational studies. It is possible that some, if not all, of the benefit, observed with metformin in observational studies may be related to analytic design and exposure definitions. -
Efficiency of Average Treatment Effect Estimation When the True
econometrics Article Efficiency of Average Treatment Effect Estimation When the True Propensity Is Parametric Kyoo il Kim Department of Economics, Michigan State University, 486 W. Circle Dr., East Lansing, MI 48824, USA; [email protected]; Tel.: +1-517-353-9008 Received: 8 March 2019; Accepted: 28 May 2019; Published: 31 May 2019 Abstract: It is well known that efficient estimation of average treatment effects can be obtained by the method of inverse propensity score weighting, using the estimated propensity score, even when the true one is known. When the true propensity score is unknown but parametric, it is conjectured from the literature that we still need nonparametric propensity score estimation to achieve the efficiency. We formalize this argument and further identify the source of the efficiency loss arising from parametric estimation of the propensity score. We also provide an intuition of why this overfitting is necessary. Our finding suggests that, even when we know that the true propensity score belongs to a parametric class, we still need to estimate the propensity score by a nonparametric method in applications. Keywords: average treatment effect; efficiency bound; propensity score; sieve MLE JEL Classification: C14; C18; C21 1. Introduction Estimating treatment effects of a binary treatment or a policy has been one of the most important topics in evaluation studies. In estimating treatment effects, a subject’s selection into a treatment may contaminate the estimate, and two approaches are popularly used in the literature to remove the bias due to this sample selection. One is regression-based control function method (see, e.g., Rubin(1973) ; Hahn(1998); and Imbens(2004)) and the other is matching method (see, e.g., Rubin and Thomas(1996); Heckman et al.(1998); and Abadie and Imbens(2002, 2006)). -
10. Sample Bias, Bias of Selection and Double-Blind
SEC 4 Page 1 of 8 10. SAMPLE BIAS, BIAS OF SELECTION AND DOUBLE-BLIND 10.1 SAMPLE BIAS: In statistics, sampling bias is a bias in which a sample is collected in such a way that some members of the intended population are less likely to be included than others. It results in abiased sample, a non-random sample[1] of a population (or non-human factors) in which all individuals, or instances, were not equally likely to have been selected.[2] If this is not accounted for, results can be erroneously attributed to the phenomenon under study rather than to the method of sampling. Medical sources sometimes refer to sampling bias as ascertainment bias.[3][4] Ascertainment bias has basically the same definition,[5][6] but is still sometimes classified as a separate type of bias Types of sampling bias Selection from a specific real area. For example, a survey of high school students to measure teenage use of illegal drugs will be a biased sample because it does not include home-schooled students or dropouts. A sample is also biased if certain members are underrepresented or overrepresented relative to others in the population. For example, a "man on the street" interview which selects people who walk by a certain location is going to have an overrepresentation of healthy individuals who are more likely to be out of the home than individuals with a chronic illness. This may be an extreme form of biased sampling, because certain members of the population are totally excluded from the sample (that is, they have zero probability of being selected). -
Week 10: Causality with Measured Confounding
Week 10: Causality with Measured Confounding Brandon Stewart1 Princeton November 28 and 30, 2016 1These slides are heavily influenced by Matt Blackwell, Jens Hainmueller, Erin Hartman, Kosuke Imai and Gary King. Stewart (Princeton) Week 10: Measured Confounding November 28 and 30, 2016 1 / 176 Where We've Been and Where We're Going... Last Week I regression diagnostics This Week I Monday: F experimental Ideal F identification with measured confounding I Wednesday: F regression estimation Next Week I identification with unmeasured confounding I instrumental variables Long Run I causality with measured confounding ! unmeasured confounding ! repeated data Questions? Stewart (Princeton) Week 10: Measured Confounding November 28 and 30, 2016 2 / 176 1 The Experimental Ideal 2 Assumption of No Unmeasured Confounding 3 Fun With Censorship 4 Regression Estimators 5 Agnostic Regression 6 Regression and Causality 7 Regression Under Heterogeneous Effects 8 Fun with Visualization, Replication and the NYT 9 Appendix Subclassification Identification under Random Assignment Estimation Under Random Assignment Blocking Stewart (Princeton) Week 10: Measured Confounding November 28 and 30, 2016 3 / 176 Lancet 2001: negative correlation between coronary heart disease mortality and level of vitamin C in bloodstream (controlling for age, gender, blood pressure, diabetes, and smoking) Stewart (Princeton) Week 10: Measured Confounding November 28 and 30, 2016 4 / 176 Lancet 2002: no effect of vitamin C on mortality in controlled placebo trial (controlling for nothing) Stewart (Princeton) Week 10: Measured Confounding November 28 and 30, 2016 4 / 176 Lancet 2003: comparing among individuals with the same age, gender, blood pressure, diabetes, and smoking, those with higher vitamin C levels have lower levels of obesity, lower levels of alcohol consumption, are less likely to grow up in working class, etc. -
STATS 361: Causal Inference
STATS 361: Causal Inference Stefan Wager Stanford University Spring 2020 Contents 1 Randomized Controlled Trials 2 2 Unconfoundedness and the Propensity Score 9 3 Efficient Treatment Effect Estimation via Augmented IPW 18 4 Estimating Treatment Heterogeneity 27 5 Regression Discontinuity Designs 35 6 Finite Sample Inference in RDDs 43 7 Balancing Estimators 52 8 Methods for Panel Data 61 9 Instrumental Variables Regression 68 10 Local Average Treatment Effects 74 11 Policy Learning 83 12 Evaluating Dynamic Policies 91 13 Structural Equation Modeling 99 14 Adaptive Experiments 107 1 Lecture 1 Randomized Controlled Trials Randomized controlled trials (RCTs) form the foundation of statistical causal inference. When available, evidence drawn from RCTs is often considered gold statistical evidence; and even when RCTs cannot be run for ethical or practical reasons, the quality of observational studies is often assessed in terms of how well the observational study approximates an RCT. Today's lecture is about estimation of average treatment effects in RCTs in terms of the potential outcomes model, and discusses the role of regression adjustments for causal effect estimation. The average treatment effect is iden- tified entirely via randomization (or, by design of the experiment). Regression adjustments may be used to decrease variance, but regression modeling plays no role in defining the average treatment effect. The average treatment effect We define the causal effect of a treatment via potential outcomes. For a binary treatment w 2 f0; 1g, we define potential outcomes Yi(1) and Yi(0) corresponding to the outcome the i-th subject would have experienced had they respectively received the treatment or not. -
Efficient Estimation of Average Treatment Effects Using the Estimated Propensity Score
Econometrica, Vol. 71, No. 4 (July, 2003), 1161-1189 EFFICIENT ESTIMATION OF AVERAGE TREATMENT EFFECTS USING THE ESTIMATED PROPENSITY SCORE BY KiEisuKEHIRANO, GUIDO W. IMBENS,AND GEERT RIDDER' We are interestedin estimatingthe averageeffect of a binarytreatment on a scalar outcome.If assignmentto the treatmentis exogenousor unconfounded,that is, indepen- dent of the potentialoutcomes given covariates,biases associatedwith simple treatment- controlaverage comparisons can be removedby adjustingfor differencesin the covariates. Rosenbaumand Rubin (1983) show that adjustingsolely for differencesbetween treated and controlunits in the propensityscore removesall biases associatedwith differencesin covariates.Although adjusting for differencesin the propensityscore removesall the bias, this can come at the expenseof efficiency,as shownby Hahn (1998), Heckman,Ichimura, and Todd (1998), and Robins,Mark, and Newey (1992). We show that weightingby the inverseof a nonparametricestimate of the propensityscore, rather than the truepropensity score, leads to an efficientestimate of the averagetreatment effect. We provideintuition for this resultby showingthat this estimatorcan be interpretedas an empiricallikelihood estimatorthat efficientlyincorporates the informationabout the propensityscore. KEYWORDS: Propensity score, treatment effects, semiparametricefficiency, sieve estimator. 1. INTRODUCTION ESTIMATINGTHE AVERAGEEFFECT of a binary treatment or policy on a scalar outcome is a basic goal of manyempirical studies in economics.If assignmentto the -
Nber Working Paper Series Regression Discontinuity
NBER WORKING PAPER SERIES REGRESSION DISCONTINUITY DESIGNS IN ECONOMICS David S. Lee Thomas Lemieux Working Paper 14723 http://www.nber.org/papers/w14723 NATIONAL BUREAU OF ECONOMIC RESEARCH 1050 Massachusetts Avenue Cambridge, MA 02138 February 2009 We thank David Autor, David Card, John DiNardo, Guido Imbens, and Justin McCrary for suggestions for this article, as well as for numerous illuminating discussions on the various topics we cover in this review. We also thank two anonymous referees for their helpful suggestions and comments, and Mike Geruso, Andrew Marder, and Zhuan Pei for their careful reading of earlier drafts. Diane Alexander, Emily Buchsbaum, Elizabeth Debraggio, Enkeleda Gjeci, Ashley Hodgson, Yan Lau, Pauline Leung, and Xiaotong Niu provided excellent research assistance. The views expressed herein are those of the author(s) and do not necessarily reflect the views of the National Bureau of Economic Research. © 2009 by David S. Lee and Thomas Lemieux. All rights reserved. Short sections of text, not to exceed two paragraphs, may be quoted without explicit permission provided that full credit, including © notice, is given to the source. Regression Discontinuity Designs in Economics David S. Lee and Thomas Lemieux NBER Working Paper No. 14723 February 2009, Revised October 2009 JEL No. C1,H0,I0,J0 ABSTRACT This paper provides an introduction and "user guide" to Regression Discontinuity (RD) designs for empirical researchers. It presents the basic theory behind the research design, details when RD is likely to be valid or invalid given economic incentives, explains why it is considered a "quasi-experimental" design, and summarizes different ways (with their advantages and disadvantages) of estimating RD designs and the limitations of interpreting these estimates. -
Targeted Estimation and Inference for the Sample Average Treatment Effect
View metadata, citation and similar papers at core.ac.uk brought to you by CORE provided by Collection Of Biostatistics Research Archive University of California, Berkeley U.C. Berkeley Division of Biostatistics Working Paper Series Year 2015 Paper 334 Targeted Estimation and Inference for the Sample Average Treatment Effect Laura B. Balzer∗ Maya L. Peterseny Mark J. van der Laanz ∗Division of Biostatistics, University of California, Berkeley - the SEARCH Consortium, lb- [email protected] yDivision of Biostatistics, University of California, Berkeley - the SEARCH Consortium, may- [email protected] zDivision of Biostatistics, University of California, Berkeley - the SEARCH Consortium, [email protected] This working paper is hosted by The Berkeley Electronic Press (bepress) and may not be commer- cially reproduced without the permission of the copyright holder. http://biostats.bepress.com/ucbbiostat/paper334 Copyright c 2015 by the authors. Targeted Estimation and Inference for the Sample Average Treatment Effect Laura B. Balzer, Maya L. Petersen, and Mark J. van der Laan Abstract While the population average treatment effect has been the subject of extensive methods and applied research, less consideration has been given to the sample average treatment effect: the mean difference in the counterfactual outcomes for the study units. The sample parameter is easily interpretable and is arguably the most relevant when the study units are not representative of a greater population or when the exposure’s impact is heterogeneous. Formally, the sample effect is not identifiable from the observed data distribution. Nonetheless, targeted maxi- mum likelihood estimation (TMLE) can provide an asymptotically unbiased and efficient estimate of both the population and sample parameters. -
Average Treatment Effects in the Presence of Unknown
Average treatment effects in the presence of unknown interference Fredrik Sävje∗ Peter M. Aronowy Michael G. Hudgensz October 25, 2019 Abstract We investigate large-sample properties of treatment effect estimators under unknown interference in randomized experiments. The inferential target is a generalization of the average treatment effect estimand that marginalizes over potential spillover ef- fects. We show that estimators commonly used to estimate treatment effects under no interference are consistent for the generalized estimand for several common exper- imental designs under limited but otherwise arbitrary and unknown interference. The rates of convergence depend on the rate at which the amount of interference grows and the degree to which it aligns with dependencies in treatment assignment. Importantly for practitioners, the results imply that if one erroneously assumes that units do not interfere in a setting with limited, or even moderate, interference, standard estima- tors are nevertheless likely to be close to an average treatment effect if the sample is sufficiently large. Conventional confidence statements may, however, not be accurate. arXiv:1711.06399v4 [math.ST] 23 Oct 2019 We thank Alexander D’Amour, Matthew Blackwell, David Choi, Forrest Crawford, Peng Ding, Naoki Egami, Avi Feller, Owen Francis, Elizabeth Halloran, Lihua Lei, Cyrus Samii, Jasjeet Sekhon and Daniel Spielman for helpful suggestions and discussions. A previous version of this article was circulated under the title “A folk theorem on interference in experiments.” ∗Departments of Political Science and Statistics & Data Science, Yale University. yDepartments of Political Science, Biostatistics and Statistics & Data Science, Yale University. zDepartment of Biostatistics, University of North Carolina, Chapel Hill. 1 1 Introduction Investigators of causality routinely assume that subjects under study do not interfere with each other. -
How to Examine External Validity Within an Experiment∗
How to Examine External Validity Within an Experiment∗ Amanda E. Kowalski August 7, 2019 Abstract A fundamental concern for researchers who analyze and design experiments is that the experimental estimate might not be externally valid for all policies. Researchers often attempt to assess external validity by comparing data from an experiment to external data. In this paper, I discuss approaches from the treatment effects literature that researchers can use to begin the examination of external validity internally, within the data from a single experiment. I focus on presenting the approaches simply using figures. ∗I thank Pauline Mourot, Ljubica Ristovska, Sukanya Sravasti, Rae Staben, and Matthew Tauzer for excellent research assistance. NSF CAREER Award 1350132 provided support. I thank Magne Mogstad, Jeffrey Smith, Edward Vytlacil, and anonymous referees for helpful feedback. 1 1 Introduction The traditional reason that a researcher runs an experiment is to address selection into treatment. For example, a researcher might be worried that individuals with better outcomes regardless of treatment are more likely to select into treatment, so the simple comparison of treated to untreated individuals will reflect a selection effect as well as a treatment effect. By running an experiment, the reasoning goes, a researcher isolates a single treatment effect by eliminating selection. However, there is still room for selection within experiments. In many experiments, some lottery losers receive treatment and some lottery winners forgo treatment (see Heckman et al. (2000)). Throughout this paper, I consider experiments in which both occur, experiments with \two-sided noncompliance." In these experiments, individuals participate in a lottery. Individuals who win the lottery are in the intervention group. -
A User's Guide to Debiasing
A USER’S GUIDE TO DEBIASING Jack B. Soll Fuqua School of Business Duke University Katherine L. Milkman The Wharton School The University of Pennsylvania John W. Payne Fuqua School of Business Duke University Forthcoming in Wiley-Blackwell Handbook of Judgment and Decision Making Gideon Keren and George Wu (Editors) Improving the human capacity to decide represents one of the great global challenges for the future, along with addressing problems such as climate change, the lack of clean water, and conflict between nations. So says the Millenium Project (Glenn, Gordon, & Florescu, 2012), a joint effort initiated by several esteemed organizations including the United Nations and the Smithsonian Institution. Of course, decision making is not a new challenge—people have been making decisions since, well, the beginning of the species. Why focus greater attention on decision making now? Among other factors such as increased interdependency, the Millenium Project emphasizes the proliferation of choices available to people. Many decisions, ranging from personal finance to health care to starting a business, are more complex than they used to be. Along with more choices comes greater uncertainty and greater demand on cognitive resources. The cost of being ill-equipped to choose, as an individual, is greater now than ever. What can be done to improve the capacity to decide? We believe that judgment and decision making researchers have produced many insights that can help answer this question. Decades of research in our field have yielded an array of debiasing strategies that can improve judgments and decisions across a wide range of settings in fields such as business, medicine, and policy. -
Inference on the Average Treatment Effect on the Treated from a Bayesian Perspective
UNIVERSIDADE FEDERAL DO RIO DE JANEIRO INSTITUTO DE MATEMÁTICA DEPARTAMENTO DE MÉTODOS ESTATÍSTICOS Inference on the Average Treatment Effect on the Treated from a Bayesian Perspective Estelina Serrano de Marins Capistrano Rio de Janeiro – RJ 2019 Inference on the Average Treatment Effect on the Treated from a Bayesian Perspective Estelina Serrano de Marins Capistrano Doctoral thesis submitted to the Graduate Program in Statistics of Universidade Federal do Rio de Janeiro as part of the requirements for the degree of Doctor in Statistics. Advisors: Prof. Alexandra M. Schmidt, Ph.D. Prof. Erica E. M. Moodie, Ph.D. Rio de Janeiro, August 27th of 2019. Inference on the Average Treatment Effect on the Treated from a Bayesian Perspective Estelina Serrano de Marins Capistrano Doctoral thesis submitted to the Graduate Program in Statistics of Universidade Federal do Rio de Janeiro as part of the requirements for the degree of Doctor in Statistics. Approved by: Prof. Alexandra M. Schmidt, Ph.D. IM - UFRJ (Brazil) / McGill University (Canada) - Advisor Prof. Erica E. M. Moodie, Ph.D. McGill University (Canada) - Advisor Prof. Hedibert F. Lopes, Ph.D. INSPER - SP (Brazil) Prof. Helio dos S. Migon, Ph.D. IM - UFRJ (Brazil) Prof. Mariane B. Alves, Ph.D. IM - UFRJ (Brazil) Rio de Janeiro, August 27th of 2019. CIP - Catalogação na Publicação Capistrano, Estelina Serrano de Marins C243i Inference on the Average Treatment Effect on the Treated from a Bayesian Perspective / Estelina Serrano de Marins Capistrano. -- Rio de Janeiro, 2019. 106 f. Orientador: Alexandra Schmidt. Coorientador: Erica Moodie. Tese (doutorado) - Universidade Federal do Rio de Janeiro, Instituto de Matemática, Programa de Pós Graduação em Estatística, 2019.