In this article, we derive and compare methods to derive \textit{p}-values and sets of confidence intervals with strong control of the family-wise error rates and coverage for estimates of treatment effects in cluster randomised trials with multiple outcomes. There are few methods for \textit{p}-value corrections and deriving confidence intervals, limiting their application in this setting. We discuss the methods of Bonferroni, Holm, and Romano \& Wolf (2005) and adapt them to cluster randomised trial inference using permutation-based methods with different test statistics. We develop a novel search procedure for confidence set limits using permutation tests to produce a set of confidence intervals under each method of correction. We conduct a simulation-based study to compare family-wise error rates, coverage of confidence sets, and the efficiency of each procedure in comparison to no correction using both model-based standard errors and permutation tests. We show that the Romano-Wolf type procedure has nominal error rates and coverage under non-independent correlation structures and is more efficient than the other methods in a simulation-based study. We also compare results from the analysis of a real-world trial.
Long-run covariance matrix estimation is the building block of time series inference problems. The corresponding difference-based estimator, which avoids detrending, has attracted considerable interest due to its robustness to both smooth and abrupt structural breaks and its competitive finite sample performance. However, existing methods mainly focus on estimators for the univariate process while their direct and multivariate extensions for most linear models are asymptotically biased. We propose a novel difference-based and debiased long-run covariance matrix estimator for functional linear models with time-varying regression coefficients, allowing time series non-stationarity, long-range dependence, state-heteroscedasticity and their mixtures. We apply the new estimator to i) the structural stability test, overcoming the notorious non-monotonic power phenomena caused by piecewise smooth alternatives for regression coefficients, and (ii) the nonparametric residual-based tests for long memory, improving the performance via the residual-free formula of the proposed estimator. The effectiveness of the proposed method is justified theoretically and demonstrated by superior performance in simulation studies, while its usefulness is elaborated by means of real data analysis.
We propose a novel Monte-Carlo based ab-initio algorithm for directly computing the statistics for quantities of interest in an immiscible two-phase compressible flow. Our algorithm samples the underlying probability space and evolves these samples with a sharp interface front-tracking scheme. Consequently, statistical information is generated without resorting to any closure assumptions and information about the underlying microstructure is implicitly included. The proposed algorithm is tested on a suite of numerical experiments and we observe that the ab-initio procedure can simulate a variety of flow regimes robustly and converges with respect of refinement of number of samples as well as number of bubbles per volume. The results are also compared with a state-of-the-art discrete equation method to reveal the inherent limitations of existing macroscopic models.
Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials allows for the combination of datasets with unconfounded treatment assignment to improve the power to estimate heterogeneous treatment effects. This paper discusses several non-parametric approaches for estimating heterogeneous treatment effects using data from multiple trials. We extend single-study methods to a scenario with multiple trials and explore their performance through a simulation study, with data generation scenarios that have differing levels of cross-trial heterogeneity. The simulations demonstrate that methods that directly allow for heterogeneity of the treatment effect across trials perform better than methods that do not, and that the choice of single-study method matters based on the functional form of the treatment effect. Finally, we discuss which methods perform well in each setting and then apply them to four randomized controlled trials to examine effect heterogeneity of treatments for major depressive disorder.
Causal discovery from observational data is a very challenging, often impossible, task. However, estimating the causal structure is possible under certain assumptions on the data-generating process. Many commonly used methods rely on the additivity of the noise in the structural equation models. Additivity implies that the variance or the tail of the effect, given the causes, is invariant; the cause only affects the mean. In many applications, it is desirable to model the tail or other characteristics of the random variable since they can provide different information about the causal structure. However, models for causal inference in such cases have received only very little attention. It has been shown that the causal graph is identifiable under different models, such as linear non-Gaussian, post-nonlinear, or quadratic variance functional models. We introduce a new class of models called the Conditional Parametric Causal Models (CPCM), where the cause affects the effect in some of the characteristics of interest.We use the concept of sufficient statistics to show the identifiability of the CPCM models, focusing mostly on the exponential family of conditional distributions.We also propose an algorithm for estimating the causal structure from a random sample under CPCM. Its empirical properties are studied for various data sets, including an application on the expenditure behavior of residents of the Philippines.
In this paper we study the finite sample and asymptotic properties of various weighting estimators of the local average treatment effect (LATE), several of which are based on Abadie's (2003) kappa theorem. Our framework presumes a binary treatment and a binary instrument, which may only be valid after conditioning on additional covariates. We argue that one of the Abadie estimators, which is weight normalized, is preferable in many contexts. Several other estimators, which are unnormalized, do not generally satisfy the properties of scale invariance with respect to the natural logarithm and translation invariance, thereby exhibiting sensitivity to the units of measurement when estimating the LATE in logs and the centering of the outcome variable more generally. On the other hand, when noncompliance is one-sided, certain unnormalized estimators have the advantage of being based on a denominator that is bounded away from zero. To reconcile these findings, we demonstrate that when the instrument propensity score is estimated using an appropriate covariate balancing approach, the resulting normalized estimator also shares this advantage. We use a simulation study and three empirical applications to illustrate our findings. In two cases, the unnormalized estimates are clearly unreasonable, with "incorrect" signs, magnitudes, or both.
In the past decade, the technology industry has adopted online randomized controlled experiments (a.k.a. A/B testing) to guide product development and make business decisions. In practice, A/B tests are often implemented with increasing treatment allocation: the new treatment is gradually released to an increasing number of units through a sequence of randomized experiments. In scenarios such as experimenting in a social network setting or in a bipartite online marketplace, interference among units may exist, which can harm the validity of simple inference procedures. In this work, we introduce a widely applicable procedure to test for interference in A/B testing with increasing allocation. Our procedure can be implemented on top of an existing A/B testing platform with a separate flow and does not require a priori a specific interference mechanism. In particular, we introduce two permutation tests that are valid under different assumptions. Firstly, we introduce a general statistical test for interference requiring no additional assumption. Secondly, we introduce a testing procedure that is valid under a time fixed effect assumption. The testing procedure is of very low computational complexity, it is powerful, and it formalizes a heuristic algorithm implemented already in industry. We demonstrate the performance of the proposed testing procedure through simulations on synthetic data. Finally, we discuss one application at LinkedIn, where a screening step is implemented to detect potential interference in all their marketplace experiments with the proposed methods in the paper.
Estimands can help clarify the interpretation of treatment effects and ensure that estimators are aligned to the study's objectives. Cluster randomised trials require additional attributes to be defined within the estimand compared to individually randomised trials, including whether treatment effects are marginal or cluster specific, and whether they are participant or cluster average. In this paper, we provide formal definitions of estimands encompassing both these attributes using potential outcomes notation and describe differences between them. We then provide an overview of estimators for each estimand that are asymptotically unbiased under minimal assumptions. Then, through a reanalysis of a published cluster randomised trial, we demonstrate that estimates corresponding to the different estimands can vary considerably. Estimated odds ratios corresponding to different estimands varied by more than 30 percent, from 3.69 to 4.85. We conclude that careful specification of the estimand, along with appropriate choice of estimator, are essential to ensuring that cluster randomised trials are addressing the right question.
In this paper, we consider a new approach for semi-discretization in time and spatial discretization of a class of semi-linear stochastic partial differential equations (SPDEs) with multiplicative noise. The drift term of the SPDEs is only assumed to satisfy a one-sided Lipschitz condition and the diffusion term is assumed to be globally Lipschitz continuous. Our new strategy for time discretization is based on the Milstein method from stochastic differential equations. We use the energy method for its error analysis and show a strong convergence order of nearly $1$ for the approximate solution. The proof is based on new H\"older continuity estimates of the SPDE solution and the nonlinear term. For the general polynomial-type drift term, there are difficulties in deriving even the stability of the numerical solutions. We propose an interpolation-based finite element method for spatial discretization to overcome the difficulties. Then we obtain $H^1$ stability, higher moment $H^1$ stability, $L^2$ stability, and higher moment $L^2$ stability results using numerical and stochastic techniques. The nearly optimal convergence orders in time and space are hence obtained by coupling all previous results. Numerical experiments are presented to implement the proposed numerical scheme and to validate the theoretical results.
Informative cluster size (ICS) arises in situations with clustered data where a latent relationship exists between the number of participants in a cluster and the outcome measures. Although this phenomenon has been sporadically reported in statistical literature for nearly two decades now, further exploration is needed in certain statistical methodologies to avoid potentially misleading inferences. For inference about population quantities without covariates, inverse cluster size reweightings are often employed to adjust for ICS. Further, to study the effect of covariates on disease progression described by a multistate model, the pseudo-value regression technique has gained popularity in time-to-event data analysis. We seek to answer the question: "How to apply pseudo-value regression to clustered time-to-event data when cluster size is informative?" ICS adjustment by the reweighting method can be performed in two steps; estimation of marginal functions of the multistate model and fitting the estimating equations based on pseudo-value responses, leading to four possible strategies. We present theoretical arguments and thorough simulation experiments to ascertain the correct strategy for adjusting for ICS. A further extension of our methodology is implemented to include informativeness induced by the intra-cluster group size. We demonstrate the methods in two real-world applications: (i) to determine predictors of tooth survival in a periodontal study, and (ii) to identify indicators of ambulatory recovery in spinal cord injury patients who participated in locomotor-training rehabilitation.
This paper focuses on the expected difference in borrower's repayment when there is a change in the lender's credit decisions. Classical estimators overlook the confounding effects and hence the estimation error can be magnificent. As such, we propose another approach to construct the estimators such that the error can be greatly reduced. The proposed estimators are shown to be unbiased, consistent, and robust through a combination of theoretical analysis and numerical testing. Moreover, we compare the power of estimating the causal quantities between the classical estimators and the proposed estimators. The comparison is tested across a wide range of models, including linear regression models, tree-based models, and neural network-based models, under different simulated datasets that exhibit different levels of causality, different degrees of nonlinearity, and different distributional properties. Most importantly, we apply our approaches to a large observational dataset provided by a global technology firm that operates in both the e-commerce and the lending business. We find that the relative reduction of estimation error is strikingly substantial if the causal effects are accounted for correctly.