Abstract Bias in causal comparisons has a correspondence with distributional imbalance of covariates between treatment groups. Weighting strategies such as inverse propensity score weighting attempt to mitigate bias by either modeling the treatment assignment mechanism or balancing specified covariate moments. This article introduces a new weighting method, called energy balancing, which instead aims to balance weighted covariate distributions. By directly targeting distributional imbalance, the proposed weighting strategy can be flexibly utilized in a wide variety of causal analyses without the need for careful model or moment specification. Our energy balancing weights (EBW) approach has several advantages over existing weighting techniques. First, it offers a model-free and robust approach for obtaining covariate balance that does not require tuning parameters, obviating the need for modeling decisions of secondary nature to the scientific question at hand. Second, since this approach is based on a genuine measure of distributional balance, it provides a means for assessing the balance induced by a given set of weights for a given dataset. We demonstrate the effectiveness of this EBW approach in a suite of simulation experiments, and in studies on the safety of right heart catheterization and on three additional studies using electronic health record data.
more »
« less
Covariate Balancing Methods for Randomized Controlled Trials Are Not Adversarially Robust
The first step toward investigating the effectiveness of a treatment via a randomized trial is to split the population into control and treatment groups then compare the average response of the treatment group receiving the treatment to the control group receiving the placebo. To ensure that the difference between the two groups is caused only by the treatment, it is crucial that the control and the treatment groups have similar statistics. Indeed, the validity and reliability of a trial are determined by the similarity of two groups’ statistics. Covariate balancing methods increase the similarity between the distributions of the two groups’ covariates. However, often in practice, there are not enough samples to accurately estimate the groups’ covariate distributions. In this article, we empirically show that covariate balancing with the standardized means difference (SMD) covariate balancing measure, as well as Pocock and Simon’s sequential treatment assignment method, are susceptible to worst case treatment assignments. Worst case treatment assignments are those admitted by the covariate balance measure, but result in highest possible ATE estimation errors. We developed an adversarial attack to find adversarial treatment assignment for any given trial. Then, we provide an index to measure how close the given trial is to the worst case. To this end, we provide an optimization-based algorithm, namely adversarial treatment assignment in treatment effect trials (ATASTREET), to find the adversarial treatment assignments.
more »
« less
- PAR ID:
- 10466282
- Date Published:
- Journal Name:
- IEEE Transactions on Neural Networks and Learning Systems
- ISSN:
- 2162-237X
- Page Range / eLocation ID:
- 1 to 13
- Format(s):
- Medium: X
- Sponsoring Org:
- National Science Foundation
More Like this
-
-
Inverse probability of treatment weighting (IPTW), which has been used to estimate average treatment effects (ATE) using observational data, tenuously relies on the positivity assumption and the correct specification of the treatment assignment model, both of which are problematic assumptions in many observational studies. Various methods have been proposed to overcome these challenges, including truncation, covariate‐balancing propensity scores, and stable balancing weights. Motivated by an observational study in spine surgery, in which positivity is violated and the true treatment assignment model is unknown, we present the use of optimal balancing by kernel optimal matching (KOM) to estimate ATE. By uniformly controlling the conditional mean squared error of a weighted estimator over a class of models, KOM simultaneously mitigates issues of possible misspecification of the treatment assignment model and is able to handle practical violations of the positivity assumption, as shown in our simulation study. Using data from a clinical registry, we apply KOM to compare two spine surgical interventions and demonstrate how the result matches the conclusions of clinical trials that IPTW estimates spuriously refute.more » « less
-
High-throughput target trial emulation for Alzheimer’s disease drug repurposing with real-world dataTarget trial emulation is the process of mimicking target randomized trials using real-world data, where effective confounding control for unbiased treatment effect estimation remains a main challenge. Although various approaches have been proposed for this challenge, a systematic evaluation is still lacking. Here we emulated trials for thousands of medications from two large-scale real-world data warehouses, covering over 10 years of clinical records for over 170 million patients, aiming to identify new indications of approved drugs for Alzheimer’s disease. We assessed different propensity score models under the inverse probability of treatment weighting framework and suggested a model selection strategy for improved baseline covariate balancing. We also found that the deep learning-based propensity score model did not necessarily outperform logistic regression-based methods in covariate balancing. Finally, we highlighted five top-ranked drugs (pantoprazole, gabapentin, atorvastatin, fluticasone, and omeprazole) originally intended for other indications with potential benefits for Alzheimer’s patients.more » « less
-
Abstract The generalized semiparametric mixed varying‐coefficient effects model for longitudinal data can accommodate a variety of link functions and flexibly model different types of covariate effects, including time‐constant, time‐varying and covariate‐varying effects. The time‐varying effects are unspecified functions of time and the covariate‐varying effects are nonparametric functions of a possibly time‐dependent exposure variable. A semiparametric estimation procedure is developed that uses local linear smoothing and profile weighted least squares, which requires smoothing in the two different and yet connected domains of time and the time‐dependent exposure variable. The asymptotic properties of the estimators of both nonparametric and parametric effects are investigated. In addition, hypothesis testing procedures are developed to examine the covariate effects. The finite‐sample properties of the proposed estimators and testing procedures are examined through simulations, indicating satisfactory performances. The proposed methods are applied to analyze the AIDS Clinical Trial Group 244 clinical trial to investigate the effects of antiretroviral treatment switching in HIV‐infected patients before and after developing the T215Y antiretroviral drug resistance mutation.The Canadian Journal of Statistics47: 352–373; 2019 © 2019 Statistical Society of Canadamore » « less
-
Abstract Background Substance use disorders (SUDs) represent major public health concerns and are linked to enhanced risk of legal consequences. Unresolved legal issues may prevent individuals with SUD from completing treatment. Interventions aimed at improving SUD treatment outcomes are limited. Filling that gap, this randomized controlled trial (RCT) tests the ability of a technology-assisted intervention to increase SUD treatment completion rates and improve post-treatment health, economic, justice-system, and housing outcomes. Methods A randomized controlled trial with a two-year administrative follow-up period will be conducted. Eight hundred Medicaid eligible and uninsured adults receiving SUD treatment will be recruited at community-based non-profit health care clinics in Southeast, Michigan, USA. Using an algorithm embedded in a community-based case management system, we randomly assign all eligible adults to one of two groups. The treatment/intervention group will receive hands-on assistance with a technology aimed at resolving unaddressed legal issues and the control group receives no treatment. Upon enrollment into the intervention, both treatment ( n = 400) and control groups ( n = 400) retain traditional options to resolve unaddressed legal issues, such as hiring an attorney, but only the treatment group is targeted the technology and offered personalized assistance in navigating the online legal platform. To develop baseline and historical contexts for participants, we collect life course history reports from all participants and intend to link those in each group to administrative data sources. In addition to the randomized controlled trial (RCT), we used an exploratory sequential mixed methods and participatory-based design to develop, test, and administer our life course history instruments to all participants. The primary objective is to test whether targeting no-cost online legal resources to those experiencing SUD improves their long-term recovery and decreases negative health, economic, justice-system, and housing outcomes. Discussion Findings from this RCT will improve our understanding of the acute socio-legal needs faced by those experiencing SUD and provide recommendations to help target resources toward the areas that best support long-term recovery. The public health impact includes making publicly available a deidentified, longitudinal dataset of uninsured and Medicaid eligible clients in treatment for SUD. Data include an overrepresentation of understudied groups including African American and American Indian Alaska Native persons documented to experience heightened risk for SUD-related premature mortality and justice-system involvement. Within these data, several intended outcome measures can inform the health policy landscape: (1) health, including substance use, disability, mental health diagnosis, and mortality; (2) financial health, including employment, earnings, public assistance receipt, and financial obligations to the state; (3) justice-system involvement, including civil and criminal legal system encounters; (4) housing, including homelessness, household composition, and homeownership. Trial registration Retrospectively registered # NCT05665179 on December 27, 2022.more » « less
An official website of the United States government

