Causal inference for extreme events has many potential applications in fields such as climate science, medicine and economics. We study the extremal quantile treatment effect of a binary treatment on a continuous, heavy-tailed outcome. Existing methods are limited to the case where the quantile of interest is within the range of the observations. For applications in risk assessment, however, the most relevant cases relate to extremal quantiles that go beyond the data range. We introduce an estimator of the extremal quantile treatment effect that relies on asymptotic tail approximation, and use a new causal Hill estimator for the extreme value indices of potential outcome distributions. We establish asymptotic normality of the estimators and propose a consistent variance estimator to achieve valid statistical inference. We illustrate the performance of our method in simulation studies, and apply it to a real data set to estimate the extremal quantile treatment effect of college education on wage.
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In a completely randomized experiment, the variances of treatment effect estimators in the finite population are usually not identifiable and hence not estimable. Although some estimable bounds of the variances have been established in the literature, few of them are derived in the presence of covariates. In this paper, the difference-in-means estimator and the Wald estimator are considered in the completely randomized experiment with perfect compliance and noncompliance, respectively. Sharp bounds for the variances of these two estimators are established when covariates are available. Furthermore, consistent estimators for such bounds are obtained, which can be used to shorten the confidence intervals and improve the power of tests. Confidence intervals are constructed based on the consistent estimators of the upper bounds, whose coverage rates are uniformly asymptotically guaranteed. Simulations were conducted to evaluate the proposed methods. The proposed methods are also illustrated with two real data analyses.
Consider a $p$-dimensional population ${\mathbf x} \in\mathbb{R}^p$ with iid coordinates in the domain of attraction of a stable distribution with index $\alpha\in (0,2)$. Since the variance of ${\mathbf x}$ is infinite, the sample covariance matrix ${\mathbf S}_n=n^{-1}\sum_{i=1}^n {{\mathbf x}_i}{\mathbf x}'_i$ based on a sample ${\mathbf x}_1,\ldots,{\mathbf x}_n$ from the population is not well behaved and it is of interest to use instead the sample correlation matrix ${\mathbf R}_n= \{\operatorname{diag}({\mathbf S}_n)\}^{-1/2}\, {\mathbf S}_n \{\operatorname{diag}({\mathbf S}_n)\}^{-1/2}$. This paper finds the limiting distributions of the eigenvalues of ${\mathbf R}_n$ when both the dimension $p$ and the sample size $n$ grow to infinity such that $p/n\to \gamma \in (0,\infty)$. The family of limiting distributions $\{H_{\alpha,\gamma}\}$ is new and depends on the two parameters $\alpha$ and $\gamma$. The moments of $H_{\alpha,\gamma}$ are fully identified as sum of two contributions: the first from the classical Mar\v{c}enko-Pastur law and a second due to heavy tails. Moreover, the family $\{H_{\alpha,\gamma}\}$ has continuous extensions at the boundaries $\alpha=2$ and $\alpha=0$ leading to the Mar\v{c}enko-Pastur law and a modified Poisson distribution, respectively. Our proofs use the method of moments, the path-shortening algorithm developed in [18] and some novel graph counting combinatorics. As a consequence, the moments of $H_{\alpha,\gamma}$ are expressed in terms of combinatorial objects such as Stirling numbers of the second kind. A simulation study on these limiting distributions $H_{\alpha,\gamma}$ is also provided for comparison with the Mar\v{c}enko-Pastur law.
In this paper we obtain quantitative Bernstein-von Mises type bounds on the normal approximation of the posterior distribution in exponential family models when centering either around the posterior mode or around the maximum likelihood estimator. Our bounds, obtained through a version of Stein's method, are non-asymptotic, and data dependent; they are of the correct order both in the total variation and Wasserstein distances, as well as for approximations for expectations of smooth functions of the posterior. All our results are valid for univariate and multivariate posteriors alike, and do not require a conjugate prior setting. We illustrate our findings on a variety of exponential family distributions, including Poisson, multinomial and normal distribution with unknown mean and variance. The resulting bounds have an explicit dependence on the prior distribution and on sufficient statistics of the data from the sample, and thus provide insight into how these factors may affect the quality of the normal approximation. The performance of the bounds is also assessed with simulations.
Recently, high dimensional vector auto-regressive models (VAR), have attracted a lot of interest, due to novel applications in the health, engineering and social sciences. The presence of temporal dependence poses additional challenges to the theory of penalized estimation techniques widely used in the analysis of their iid counterparts. However, recent work (e.g., [Basu and Michailidis, 2015, Kock and Callot, 2015]) has established optimal consistency of $\ell_1$-LASSO regularized estimates applied to models involving high dimensional stable, Gaussian processes. The only price paid for temporal dependence is an extra multiplicative factor that equals 1 for independent and identically distributed (iid) data. Further, [Wong et al., 2020] extended these results to heavy tailed VARs that exhibit "$\beta$-mixing" dependence, but the rates rates are sub-optimal, while the extra factor is intractable. This paper improves these results in two important directions: (i) We establish optimal consistency rates and corresponding finite sample bounds for the underlying model parameters that match those for iid data, modulo a price for temporal dependence, that is easy to interpret and equals 1 for iid data. (ii) We incorporate more general penalties in estimation (which are not decomposable unlike the $\ell_1$ norm) to induce general sparsity patterns. The key technical tool employed is a novel, easy-to-use concentration bound for heavy tailed linear processes, that do not rely on "mixing" notions and give tighter bounds.
Investigators are increasingly using novel methods for extending (generalizing or transporting) causal inferences from a trial to a target population. In many generalizability and transportability analyses, the trial and the observational data from the target population are separately sampled, following a non-nested trial design. In practical implementations of this design, non-randomized individuals from the target population are often identified by conditioning on the use of a particular treatment, while individuals who used other candidate treatments for the same indication or individuals who did not use any treatment are excluded. In this paper, we argue that conditioning on treatment in the target population changes the estimand of generalizability and transportability analyses and potentially introduces serious bias in the estimation of causal estimands in the target population or the subset of the target population using a specific treatment. Furthermore, we argue that the naive application of marginalization-based or weighting-based standardization methods does not produce estimates of any reasonable causal estimand. We use causal graphs and counterfactual arguments to characterize the identification problems induced by conditioning on treatment in the target population and illustrate the problems using simulated data. We conclude by considering the implications of our findings for applied work.
Estimation and evaluation of individualized treatment rules have been studied extensively, but real-world treatment resource constraints have received limited attention in existing methods. We investigate a setting in which treatment is intervened upon based on covariates to optimize the mean counterfactual outcome under treatment cost constraints when the treatment cost is random. In a particularly interesting special case, an instrumental variable corresponding to encouragement to treatment is intervened upon with constraints on the proportion receiving treatment. For such settings, we first develop a method to estimate optimal individualized treatment rules. We further construct an asymptotically efficient plug-in estimator of the corresponding average treatment effect relative to a given reference rule.
A common problem faced in clinical studies is that of estimating the effect of the most effective (e.g., the one having the largest mean) treatment among $k~(\geq2)$ available treatments. The most effective treatment is adjudged based on numerical values of some statistic corresponding to the $k$ treatments. A proper design for such problems is the so-called "Drop-the-Losers Design (DLD)". We consider two treatments whose effects are described by independent Gaussian distributions having different unknown means and a common known variance. To select the more effective treatment, the two treatments are independently administered to $n_1$ subjects each and the treatment corresponding to the larger sample mean is selected. To study the effect of the adjudged more effective treatment (i.e., estimating its mean), we consider the two-stage DLD in which $n_2$ subjects are further administered the adjudged more effective treatment in the second stage of the design. We obtain some admissibility and minimaxity results for estimating the mean effect of the adjudged more effective treatment. The maximum likelihood estimator is shown to be minimax and admissible. We show that the uniformly minimum variance conditionally unbiased estimator (UMVCUE) of the selected treatment mean is inadmissible and obtain an improved estimator. In this process, we also derive a sufficient condition for inadmissibility of an arbitrary location and permutation equivariant estimator and provide dominating estimators in cases where this sufficient condition is satisfied. The mean squared error and the bias performances of various competing estimators are compared via a simulation study. A real data example is also provided for illustration purposes.
The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity in the utility of a surrogate marker. When such heterogeneity exists, existing methods that use the surrogate to test for a treatment effect while ignoring this heterogeneity may lead to inaccurate conclusions about the treatment effect, particularly when the patient population in the new study has a different mix of characteristics than the study used to evaluate the utility of the surrogate marker. In this paper, we develop a novel test for a treatment effect using surrogate marker information that accounts for heterogeneity in the utility of the surrogate. We compare our testing procedure to a test that uses primary outcome information (gold standard) and a test that uses surrogate marker information, but ignores heterogeneity. We demonstrate the validity of our approach and derive the asymptotic properties of our estimator and variance estimates. Simulation studies examine the finite sample properties of our testing procedure and demonstrate when our proposed approach can outperform the testing approach that ignores heterogeneity. We illustrate our methods using data from an AIDS clinical trial to test for a treatment effect using CD4 count as a surrogate marker for RNA.
A goodness-of-fit test for one-parameter count distributions with finite second moment is proposed. The test statistic is derived from the $L_1$-distance of a function of the probability generating function of the model under the null hypothesis and that of the random variable actually generating data, when the latter belongs to a suitable wide class of alternatives. The test statistic has a rather simple form and it is asymptotically normally distributed under the null hypothesis, allowing a straightforward implementation of the test. Moreover, the test is consistent for alternative distributions belonging to the class, but also for all the alternative distributions whose probability of zero is different from that under the null hypothesis. Thus, the use of the test is proposed and investigated also for alternatives not in the class. The finite-sample properties of the test are assessed by means of an extensive simulation study.
To estimate causal effects, analysts performing observational studies in health settings utilize several strategies to mitigate bias due to confounding by indication. There are two broad classes of approaches for these purposes: use of confounders and instrumental variables (IVs). Because such approaches are largely characterized by untestable assumptions, analysts must operate under an indefinite paradigm that these methods will work imperfectly. In this tutorial, we formalize a set of general principles and heuristics for estimating causal effects in the two approaches when the assumptions are potentially violated. This crucially requires reframing the process of observational studies as hypothesizing potential scenarios where the estimates from one approach are less inconsistent than the other. While most of our discussion of methodology centers around the linear setting, we touch upon complexities in non-linear settings and flexible procedures such as target minimum loss-based estimation (TMLE) and double machine learning (DML). To demonstrate the application of our principles, we investigate the use of donepezil off-label for mild cognitive impairment (MCI). We compare and contrast results from confounder and IV methods, traditional and flexible, within our analysis and to a similar observational study and clinical trial.
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