arXiv subjects

Dylan S. Small

Publications and source records attributed to Dylan S. Small.

At least 37 records · Page 2Linked to original sources

arXiv2023

Combining Broad and Narrow Case Definitions in Matched Case-Control Studies: Firearms in the Home and Suicide Risk

Does having firearms in the home increase suicide risk? To test this hypothesis, a matched case-control study can be performed, in which suicide case subjects are compared to living controls who are similar in observed covariates in terms of their retrospective exposure to firearms at home. In this application, cases can be defined using a broad case definition (suicide) or a narrow case definition (suicide occurred at home). The broad case definition offers a larger number of cases but the narrow case definition may offer a larger effect size. Moreover, restricting to the narrow case definition may introduce selection bias (i.e., bias due to selecting samples based on characteristics affected by the treatment) because exposure to firearms in the home may affect the location of suicide and thus the type of a case a subject is. We propose a new sensitivity analysis framework for combining broad and narrow case definitions in matched case-control studies, that considers the unmeasured confounding bias and selection bias simultaneously. We develop a valid randomization-based testing procedure using only the narrow case matched sets when the effect of the unmeasured confounder on receiving treatment and the effect of the treatment on case definition among the always-cases are controlled by sensitivity parameters. We then use the Bonferroni method to combine the testing procedures using the broad and narrow case definitions. With the proposed methods, we find robust evidence that having firearms at home increases suicide risk.

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arXiv2023

Model-robust and efficient covariate adjustment for cluster-randomized experiments

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of model-based covariate adjustment is unclear when the working models are misspecified, leading to ambiguity of estimands and risk of bias. In this article, we first adapt two conventional model-based methods, generalized estimating equations and linear mixed models, with weighted g-computation to achieve robust inference for cluster-average and individual-average treatment effects. To further overcome the limitations of model-based covariate adjustment methods, we propose an efficient estimator for each estimand that allows for flexible covariate adjustment and additionally addresses cluster size variation dependent on treatment assignment and other cluster characteristics. Such cluster size variations often occur post-randomization and, if ignored, can lead to bias of model-based estimators. For our proposed efficient covariate-adjusted estimator, we prove that when the nuisance functions are consistently estimated by machine learning algorithms, the estimator is consistent, asymptotically normal, and efficient. When the nuisance functions are estimated via parametric working models, the estimator is triply-robust. Simulation studies and analyses of three real-world cluster-randomized experiments demonstrate that the proposed methods are superior to existing alternatives.

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arXiv2023

RobustIV and controlfunctionIV: Causal Inference for Linear and Nonlinear Models with Invalid Instrumental Variables

We present R software packages RobustIV and controlfunctionIV for causal inference with possibly invalid instrumental variables. RobustIV focuses on the linear outcome model. It implements the two-stage hard thresholding method to select valid instrumental variables from a set of candidate instrumental variables and make inferences for the causal effect in both low- and high-dimensional settings. Furthermore, RobustIV implements the high-dimensional endogeneity test and the searching and sampling method, a uniformly valid inference method robust to errors in instrumental variable selection. controlfunctionIV considers the nonlinear outcome model and makes inferences about the causal effect based on the control function method. Our packages are demonstrated using two publicly available economic data sets together with applications to the Framingham Heart Study.

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arXiv2023

Protocol for an Observational Study on the Effects of Giving Births from Unintended Pregnancies on Later Life Physical and Mental Health

There has been increasing interest in studying the effect of giving births to unintended pregnancies on later life physical and mental health. In this article, we provide the protocol for our planned observational study on the long-term mental and physical health consequences for mothers who bear children resulting from unintended pregnancies. We aim to use the data from the Wisconsin Longitudinal Study (WLS) and examine the effect of births from unintended pregnancies on a broad range of outcomes, including mental depression, psychological well-being, physical health, alcohol usage, and economic well-being. To strengthen our causal findings, we plan to address our research questions on two subgroups, Catholics and non-Catholics, and discover the "replicable" outcomes for which the effect of unintended pregnancy is negative (or, positive) in both subgroups. Following the idea of non-random cross-screening, the data will be split according to whether the woman is Catholic or not, and then one part of the data will be used to select the hypotheses and design the corresponding tests for the second part of the data. In past use of cross-screening (automatic cross-screening) there was only one team of investigators that dealt with both parts of the data so that the investigators would need to decide on an analysis plan before looking at the data. In this protocol, we describe plans to carry out a novel flexible cross-screening in which there will be two teams of investigators with access only to one part of data and each team will use their part of the data to decide how to plan the analysis for the second team's data. In addition to the above replicability analysis, we also discuss the plan to test the global null hypothesis that is intended to identify the outcomes which are affected by unintended pregnancy for at least one of the two subgroups of Catholics and non-Catholics.

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arXiv2022

Randomization-based Test for Censored Outcomes: A New Look at the Logrank Test

Two-sample tests with censored outcomes are a classical topic in statistics with wide use even in cutting edge applications. There are at least two modes of inference used to justify two-sample tests. One is usual superpopulation inference assuming that units are independent and identically distributed (i.i.d.) samples from some superpopulation; the other is finite population inference that relies on the random assignments of units into different groups. When randomization is actually implemented, the latter has the advantage of avoiding distributional assumptions on the outcomes. In this paper, we focus on finite population inference for censored outcomes, which has been less explored in the literature. Moreover, we allow the censoring time to depend on treatment assignment, under which exact permutation inference is unachievable. We find that, surprisingly, the usual logrank test can also be justified by randomization. Specifically, under a Bernoulli randomized experiment with non-informative i.i.d. censoring, the logrank test is asymptotically valid for testing Fisher's null hypothesis of no treatment effect on any unit. The asymptotic validity of the logrank test does not require any distributional assumption on the potential event times. We further extend the theory to the stratified logrank test, which is useful for randomized block designs and when censoring mechanisms vary across strata. In sum, the developed theory for the logrank test from finite population inference supplements its classical theory from usual superpopulation inference, and helps provide a broader justification for the logrank test.

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arXiv2022

Structural mean models for instrumented difference-in-differences

In the standard difference-in-differences research design, the parallel trends assumption may be violated when the relationship between the exposure trend and the outcome trend is confounded by unmeasured confounders. Progress can be made if there is an exogenous variable that (i) does not directly influence the change in outcome means (i.e. the outcome trend) except through influencing the change in exposure means (i.e. the exposure trend), and (ii) is not related to the unmeasured exposure - outcome confounders on the trend scale. Such exogenous variable is called an instrument for difference-in-differences. For continuous outcomes that lend themselves to linear modelling, so-called instrumented difference-in-differences methods have been proposed. In this paper, we will suggest novel multiplicative structural mean models for instrumented difference-in-differences, which allow one to identify and estimate the average treatment effect on count and rare binary outcomes, in the whole population or among the treated, when a valid instrument for difference-in-differences is available. We discuss the identifiability of these models, then develop efficient semi-parametric estimation approaches that allow the use of flexible, data-adaptive or machine learning methods to estimate the nuisance parameters. We apply our proposal on health care data to investigate the risk of moderate to severe weight gain under sulfonylurea treatment compared to metformin treatment, among new users of antihyperglycemic drugs.

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arXiv2022

A Negative Correlation Strategy for Bracketing in Difference-in-Differences

The method of difference-in-differences (DID) is widely used to study the causal effect of policy interventions in observational studies. DID employs a before and after comparison of the treated and control units to remove bias due to time-invariant unmeasured confounders under the parallel trends assumption. Estimates from DID, however, will be biased if the outcomes for the treated and control units evolve differently in the absence of treatment, namely if the parallel trends assumption is violated. We propose a general identification strategy that leverages two groups of control units whose outcomes relative to the treated units exhibit a negative correlation, and achieves partial identification of the average treatment effect for the treated. The identified set is of a union bounds form that involves the minimum and maximum operators, which makes the canonical bootstrap generally inconsistent and naive methods overly conservative. By utilizing the directional inconsistency of the bootstrap distribution, we develop a novel bootstrap method to construct uniformly valid confidence intervals for the identified set and parameter of interest when the identified set is of a union bounds form, and we establish the method's theoretical properties. We develop a simple falsification test and sensitivity analysis. We apply the proposed strategy for bracketing to study whether minimum wage laws affect employment levels.

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arXiv2022

Randomization Inference for Cluster-Randomized Test-Negative Designs with Application to Dengue Studies: Unbiased estimation, Partial compliance, and Stepped-wedge design

In 2019, the World Health Organization identified dengue as one of the top ten global health threats. For the control of dengue, the Applying Wolbachia to Eliminate Dengue (AWED) study group conducted a cluster-randomized trial in Yogyakarta, Indonesia, and used a novel design, called the cluster-randomized test-negative design (CR-TND). This design can yield valid statistical inference with data collected by a passive surveillance system and thus has the advantage of cost-efficiency compared to traditional cluster-randomized trials. We investigate the statistical assumptions and properties of CR-TND under a randomization inference framework, which is known to be robust and efficient for small-sample problems. We find that, when the differential healthcare-seeking behavior comparing intervention and control varies across clusters (in contrast to the setting of Dufault and Jewell, 2020 where the differential healthcare-seeking behavior is constant across clusters), current analysis methods for CR-TND can be biased and have inflated type I error. We propose the log-contrast estimator that can eliminate such bias and improve precision by adjusting for covariates. Furthermore, we extend our methods to handle partial intervention compliance and a stepped-wedge design, both of which appear frequently in cluster-randomized trials. Finally, we demonstrate our results by simulation studies and re-analysis of the AWED study.

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arXiv2021

Selective inference for effect modification via the lasso

Effect modification occurs when the effect of the treatment on an outcome varies according to the level of other covariates and often has important implications in decision making. When there are tens or hundreds of covariates, it becomes necessary to use the observed data to select a simpler model for effect modification and then make valid statistical inference. We propose a two stage procedure to solve this problem. First, we use Robinson's transformation to decouple the nuisance parameters from the treatment effect of interest and use machine learning algorithms to estimate the nuisance parameters. Next, after plugging in the estimates of the nuisance parameters, we use the Lasso to choose a low-complexity model for effect modification. Compared to a full model consisting of all the covariates, the selected model is much more interpretable. Compared to the univariate subgroup analyses, the selected model greatly reduces the number of false discoveries. We show that the conditional selective inference for the selected model is asymptotically valid given the rate assumptions in classical semiparametric regression. Extensive simulation studies are conducted to verify the asymptotic results and an epidemiological application is used to demonstrate the method.

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arXiv2021

Instrumented Difference-in-Differences

Unmeasured confounding is a key threat to reliable causal inference based on observational studies. Motivated from two powerful natural experiment devices, the instrumental variables and difference-in-differences, we propose a new method called instrumented difference-in-differences that explicitly leverages exogenous randomness in an exposure trend to estimate the average and conditional average treatment effect in the presence of unmeasured confounding. We develop the identification assumptions using the potential outcomes framework. We propose a Wald estimator and a class of multiply robust and efficient semiparametric estimators, with provable consistency and asymptotic normality. In addition, we extend the instrumented difference-in-differences to a two-sample design to facilitate investigations of delayed treatment effect and provide a measure of weak identification. We demonstrate our results in simulated and real datasets.

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arXiv2021

Re-Evaluating Strengthened-IV Designs: Asymptotic Efficiency, Bias Formula, and the Validity and Power of Sensitivity Analyses

Instrumental variables (IVs) are extensively used to estimate treatment effects when the treatment and outcome are confounded by unmeasured confounders; however, weak IVs are often encountered in empirical studies and may cause problems. Many studies have considered building a stronger IV from the original, possibly weak, IV in the design stage of a matched study at the cost of not using some of the samples in the analysis. It is widely accepted that strengthening an IV tends to render nonparametric tests more powerful and will increase the power of sensitivity analyses in large samples. In this article, we re-evaluate this conventional wisdom to bring new insights into this topic. We consider matched observational studies from three perspectives. First, we evaluate the trade-off between IV strength and sample size on nonparametric tests assuming the IV is valid and exhibit conditions under which strengthening an IV increases power and conversely conditions under which it decreases power. Second, we derive a necessary condition for a valid sensitivity analysis model with continuous doses. We show that the $Γ$ sensitivity analysis model, which has been previously used to come to the conclusion that strengthening an IV increases the power of sensitivity analyses in large samples, does not apply to the continuous IV setting and thus this previously reached conclusion may be invalid. Third, we quantify the bias of the Wald estimator with a possibly invalid IV under an oracle and leverage it to develop a valid sensitivity analysis framework; under this framework, we show that strengthening an IV may amplify or mitigate the bias of the estimator, and may or may not increase the power of sensitivity analyses. We also discuss how to better adjust for the observed covariates when building an IV in matched studies.

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arXiv2021

Social Distancing and COVID-19: Randomization Inference for a Structured Dose-Response Relationship

Social distancing is widely acknowledged as an effective public health policy combating the novel coronavirus. But extreme social distancing has costs and it is not clear how much social distancing is needed to achieve public health effects. In this article, we develop a design-based framework to make inference about the dose-response relationship between social distancing and COVID-19 related death toll and case numbers. We first discuss how to embed observational data with a time-independent, continuous treatment dose into an approximate randomized experiment, and develop a randomization-based procedure that tests if a structured dose-response relationship fits the data. We then generalize the design and testing procedure to accommodate a time-dependent, treatment dose trajectory, and generalize a dose-response relationship to a longitudinal setting. Finally, we apply the proposed design and testing procedures to investigate the effect of social distancing during the phased reopening in the United States on public health outcomes using data compiled from sources including Unacast, the United States Census Bureau, and the County Health Rankings and Roadmaps Program. We rejected a primary analysis null hypothesis that stated the social distancing from April 27, 2020, to June 28, 2020, had no effect on the COVID-19-related death toll from June 29, 2020, to August 2, 2020 (p-value < 0.001), and found that it took more reduction in mobility to prevent exponential growth in case numbers for non-rural counties compared to rural counties.

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arXiv2021

Relationship between changing malaria burden and low birth weight in sub-Saharan Africa: a difference-in-differences study via a pair-of-pairs approach

Although interventional studies demonstrate that preventing malaria during pregnancy can reduce the low birth weight (i.e., child's birth weight $<$ 2,500 grams) rate, it remains unknown whether natural changes in parasite transmission and malaria burden can improve birth outcomes. We conduct an observational study of the effect of changing malaria burden on low birth weight using data from 18,112 births in 19 countries in sub-Saharan African countries during the years 2000--2015. A malaria prevalence decline from a high rate (Plasmodium falciparum parasite rate in children aged 2-up-to-10 (i.e., $Pf\text{PR}_{2-10}$) $>$ 0.4) to a low rate ($Pf\text{PR}_{2-10}$ $<$ 0.2) is estimated to reduce the rate of low birth weight by 1.48 percentage points (95% confidence interval: 3.70 percentage points reduction, 0.74 percentage points increase), which is a 17% reduction in the low birth weight rate compared to the average (8.6%) in our study population with observed birth weight records (1.48/8.6 $\approx$ 17%). When focusing on first pregnancies, a decline in malaria prevalence from high to low is estimated to have a greater impact on the low birth weight rate than for all births: 3.73 percentage points (95% confidence interval: 9.11 percentage points reduction, 1.64 percentage points increase). Although the confidence intervals cannot rule out the possibility of no effect at the 95% confidence level, the concurrence between our primary analysis, secondary analyses, and sensitivity analyses, and the magnitude of the effect size, contribute to the weight of the evidence suggesting that declining malaria burden has an important effect on birth weight at the population level. The novel statistical methodology developed in this article, a pair-of-pairs approach to a difference-in-differences study, could be useful for many settings in which the units observed are different at different times.

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arXiv2021

Nonparametric maximum likelihood estimation under a likelihood ratio order

Comparison of two univariate distributions based on independent samples from them is a fundamental problem in statistics, with applications in a wide variety of scientific disciplines. In many situations, we might hypothesize that the two distributions are stochastically ordered, meaning intuitively that samples from one distribution tend to be larger than those from the other. One type of stochastic order that arises in economics, biomedicine, and elsewhere is the likelihood ratio order, also known as the density ratio order, in which the ratio of the density functions of the two distributions is monotone non-decreasing. In this article, we derive and study the nonparametric maximum likelihood estimator of the individual distributions and the ratio of their densities under the likelihood ratio order. Our work applies to discrete distributions, continuous distributions, and mixed continuous-discrete distributions. We demonstrate convergence in distribution of the estimator in certain cases, and we illustrate our results using numerical experiments and an analysis of a biomarker for predicting bacterial infection in children with systemic inflammatory response syndrome.

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arXiv2021

Sharpening the Rosenbaum Sensitivity Bounds to Address Concerns About Interactions Between Observed and Unobserved Covariates

In observational studies, it is typically unrealistic to assume that treatments are randomly assigned, even conditional on adjusting for all observed covariates. Therefore, a sensitivity analysis is often needed to examine how hidden biases due to unobserved covariates would affect inferences on treatment effects. In matched observational studies where each treated unit is matched to one or multiple untreated controls for observed covariates, the Rosenbaum bounds sensitivity analysis is one of the most popular sensitivity analysis models. In this paper, we show that in the presence of interactions between observed and unobserved covariates, directly applying the Rosenbaum bounds will almost inevitably exaggerate the report of sensitivity of causal conclusions to hidden bias. We give sharper odds ratio bounds to fix this deficiency. We illustrate our new method through studying the effect of anger/hostility tendency on the risk of having heart problems.

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arXiv2020

Increasing Power for Observational Studies of Aberrant Response: An Adaptive Approach

In many observational studies, the interest is in the effect of treatment on bad, aberrant outcomes rather than the average outcome. For such settings, the traditional approach is to define a dichotomous outcome indicating aberration from a continuous score and use the Mantel-Haenszel test with matched data. For example, studies of determinants of poor child growth use the World Health Organization's definition of child stunting being height-for-age z-score $\leq -2$. The traditional approach may lose power because it discards potentially useful information about the severity of aberration. We develop an adaptive approach that makes use of this information and asymptotically dominates the traditional approach. We develop our approach in two parts. First, we develop an aberrant rank approach in matched observational studies and prove a novel design sensitivity formula enabling its asymptotic comparison with the Mantel-Haenszel test under various settings. Second, we develop a new, general adaptive approach, the two-stage programming method, and use it to adaptively combine the aberrant rank test and the Mantel-Haenszel test. We apply our approach to a study of the effect of teenage pregnancy on stunting.

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arXiv2020

Constructing a More Closely Matched Control Group in a Difference-in-Differences Analysis: Its Effect on History Interacting with Group Bias

Difference-in-differences analysis with a control group that differs considerably from a treated group is vulnerable to bias from historical events that have different effects on the groups. Constructing a more closely matched control group by matching a subset of the overall control group to the treated group may result in less bias. We study this phenomenon in simulation studies. We study the effect of mountaintop removal mining (MRM) on mortality using a difference-in-differences analysis that makes use of the increase in MRM following the 1990 Clean Air Act Amendments. For a difference-in-differences analysis of the effect of MRM on mortality, we constructed a more closely matched control group and found a 95\% confidence interval that contains substantial adverse effects along with no effect and small beneficial effects.

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arXiv2020

ivmodel: An R Package for Inference and Sensitivity Analysis of Instrumental Variables Models with One Endogenous Variable

We present a comprehensive R software ivmodel for analyzing instrumental variables with one endogenous variable. The package implements a general class of estimators called k- class estimators and two confidence intervals that are fully robust to weak instruments. The package also provides power formulas for various test statistics in instrumental variables. Finally, the package contains methods for sensitivity analysis to examine the sensitivity of the inference to instrumental variables assumptions. We demonstrate the software on the data set from Card (1995), looking at the causal effect of levels of education on log earnings where the instrument is proximity to a four-year college.

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