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  1. Summary

    Power analyses are an important aspect of experimental design, because they help determine how experiments are implemented in practice. It is common to specify a desired level of power and compute the sample size necessary to obtain that power. Such calculations are well known for completely randomized experiments, but there can be many benefits to using other experimental designs. For example, it has recently been established that rerandomization, where subjects are randomized until covariate balance is obtained, increases the precision of causal effect estimators. This work establishes the power of rerandomized treatment-control experiments, thereby allowing for sample size calculators. We find the surprising result that, while power is often greater under rerandomization than complete randomization, the opposite can occur for very small treatment effects. The reason is that inference under rerandomization can be relatively more conservative, in the sense that it can have a lower Type-I error at the same nominal significance level, and this additional conservativeness adversely affects power. This surprising result is due to treatment effect heterogeneity, a quantity often ignored in power analyses. We find that heterogeneity increases power for large effect sizes, but decreases power for small effect sizes.

     
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  2. Abstract

    Randomized experiments are the gold standard for causal inference and enable unbiased estimation of treatment effects. Regression adjustment provides a convenient way to incorporate covariate information for additional efficiency. This article provides a unified account of its utility for improving estimation efficiency in multiarmed experiments. We start with the commonly used additive and fully interacted models for regression adjustment in estimating average treatment effects (ATE), and clarify the trade-offs between the resulting ordinary least squares (OLS) estimators in terms of finite sample performance and asymptotic efficiency. We then move on to regression adjustment based on restricted least squares (RLS), and establish for the first time its properties for inferring ATE from the design-based perspective. The resulting inference has multiple guarantees. First, it is asymptotically efficient when the restriction is correctly specified. Second, it remains consistent as long as the restriction on the coefficients of the treatment indicators, if any, is correctly specified and separate from that on the coefficients of the treatment-covariate interactions. Third, it can have better finite sample performance than the unrestricted counterpart even when the restriction is moderately misspecified. It is thus our recommendation when the OLS fit of the fully interacted regression risks large finite sample variability in case of many covariates, many treatments, yet a moderate sample size. In addition, the newly established theory of RLS also provides a unified way of studying OLS-based inference from general regression specifications. As an illustration, we demonstrate its value for studying OLS-based regression adjustment in factorial experiments. Importantly, although we analyse inferential procedures that are motivated by OLS, we do not invoke any assumptions required by the underlying linear models.

     
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  3. Summary

    Complete randomization balances covariates on average, but covariate imbalance often exists in finite samples. Rerandomization can ensure covariate balance in the realized experiment by discarding the undesired treatment assignments. Many field experiments in public health and social sciences assign the treatment at the cluster level due to logistical constraints or policy considerations. Moreover, they are frequently combined with re-randomization in the design stage. We define cluster rerandomization as a cluster-randomized experiment compounded with rerandomization to balance covariates at the individual or cluster level. Existing asymptotic theory can only deal with rerandomization with treatments assigned at the individual level, leaving that for cluster rerandomization an open problem. To fill the gap, we provide a design-based theory for cluster rerandomization. Moreover, we compare two cluster rerandomization schemes that use prior information on the importance of the covariates: one based on the weighted Euclidean distance and the other based on the Mahalanobis distance with tiers of covariates. We demonstrate that the former dominates the latter with optimal weights and orthogonalized covariates. Last but not least, we discuss the role of covariate adjustment in the analysis stage, and recommend covariate-adjusted procedures that can be conveniently implemented by least squares with the associated robust standard errors.

     
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  4. Abstract

    Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyse them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level. Standard analytic strategies are regressions based on individual data, cluster averages and cluster totals, which differ when the cluster sizes vary. These methods are often motivated by models with strong and unverifiable assumptions, and the choice among them can be subjective. Without any outcome modelling assumption, we evaluate these regression estimators and the associated robust standard errors from the design-based perspective where only the treatment assignment itself is random and controlled by the experimenter. We demonstrate that regression based on cluster averages targets a weighted average treatment effect, regression based on individual data is suboptimal in terms of efficiency and regression based on cluster totals is consistent and more efficient with a large number of clusters. We highlight the critical role of covariates in improving estimation efficiency and illustrate the efficiency gain via both simulation studies and data analysis. The asymptotic analysis also reveals the efficiency-robustness trade-off by comparing the properties of various estimators using data at different levels with and without covariate adjustment. Moreover, we show that the robust standard errors are convenient approximations to the true asymptotic standard errors under the design-based perspective. Our theory holds even when the outcome models are misspecified, so it is model-assisted rather than model-based. We also extend the theory to a wider class of weighted average treatment effects.

     
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  5. Free, publicly-accessible full text available April 1, 2025
  6. Measuring the effect of peers on individuals' outcomes is a challenging problem, in part because individuals often select peers who are similar in both observable and unobservable ways. Group formation experiments avoid this problem by randomly assigning individuals to groups and observing their responses; for example, do first‐year students have better grades when they are randomly assigned roommates who have stronger academic backgrounds? In this paper, we propose randomization‐based permutation tests for group formation experiments, extending classical Fisher Randomization Tests to this setting. The proposed tests are justified by the randomization itself, require relatively few assumptions, and are exact in finite samples. This approach can also complement existing strategies, such as linear‐in‐means models, by using a regression coefficient as the test statistic. We apply the proposed tests to two recent group formation experiments.

     
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  7. This paper provides a critical review of the Bayesian perspective of causal inference based on the potential outcomes framework. We review the causal estimands, assignment mechanism, the general structure of Bayesian inference of causal effects and sensitivity analysis. We highlight issues that are unique to Bayesian causal inference, including the role of the propensity score, the definition of identifiability, the choice of priors in both low- and high-dimensional regimes. We point out the central role of covariate overlap and more generally the design stage in Bayesian causal inference. We extend the discussion to two complex assignment mechanisms: instrumental variable and time-varying treatments. We identify the strengths and weaknesses of the Bayesian approach to causal inference. Throughout, we illustrate the key concepts via examples. This article is part of the theme issue ‘Bayesian inference: challenges, perspectives, and prospects’. 
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  8. Summary Point processes are probabilistic tools for modelling event data. While there exists a fast-growing literature on the relationships between point processes, how such relationships connect to causal effects remains unexplored. In the presence of unmeasured confounders, parameters from point process models do not necessarily have causal interpretations. We propose an instrumental variable method for causal inference with point process treatment and outcome. We define causal quantities based on potential outcomes and establish nonparametric identification results with a binary instrumental variable. We extend the traditional Wald estimation to deal with point process treatment and outcome, showing that it should be performed after a Fourier transform of the intention-to-treat effects on the treatment and outcome, and thus takes the form of deconvolution. We refer to this approach as generalized Wald estimation and propose an estimation strategy based on well-established deconvolution methods. 
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  9. Nearest neighbor (NN) matching is widely used in observational studies for causal effects. Abadie and Imbens (2006) provided the first large‐sample analysis of NN matching. Their theory focuses on the case with the number of NNs,Mfixed. We reveal something new out of their study and show that once allowingMto diverge with the sample size an intrinsic statistic in their analysis constitutes a consistent estimator of the density ratio with regard to covariates across the treated and control groups. Consequently, with a divergingM, the NN matching with Abadie and Imbens' (2011) bias correction yields a doubly robust estimator of the average treatment effect and is semiparametrically efficient if the density functions are sufficiently smooth and the outcome model is consistently estimated. It can thus be viewed as a precursor of the double machine learning estimators.

     
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  10. One dominant approach to evaluate the causal effect of a treatment is through panel data analysis, whereby the behaviors of multiple units are observed over time. The information across time and units motivates two general approaches: (i) horizontal regression (i.e., unconfoundedness), which exploits time series patterns, and (ii) vertical regression (e.g., synthetic controls), which exploits cross‐sectional patterns. Conventional wisdom often considers the two approaches to be different. We establish this position to be partly false for estimation but generally true for inference. In the absence of any assumptions, we show that both approaches yield algebraically equivalent point estimates for several standard estimators. However, the source of randomness assumed by each approach leads to a distinct estimand and quantification of uncertainty even for the same point estimate. This emphasizes that researchers should carefully consider where the randomness stems from in their data, as it has direct implications for the accuracy of inference.

     
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