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In this paper, we consider aggregated functional data composed by a linear combination of component curves and the problem of estimating these component curves. We propose the application of a bayesian wavelet shrinkage rule based on a mixture of a point mass function at zero and the logistic distribution as prior to wavelet coefficients to estimate mean curves of components. This procedure has the advantage of estimating component functions with important local characteristics such as discontinuities, spikes and oscillations for example, due the features of wavelet basis expansion of functions. Simulation studies were done to evaluate the performance of the proposed method, and its results are compared with a spline-based method. An application on the so-called Tecator dataset is also provided. [ABSTRACT FROM AUTHOR]

Randomized controlled trials (RCTs) admit unconfounded design-based inference – randomization largely justifies the assumptions underlying statistical effect estimates – but often have limited sample sizes. However, researchers may have access to big observational data on covariates and outcomes from RCT nonparticipants. For example, data from A/B tests conducted within an educational technology platform exist alongside historical observational data drawn from student logs. We outline a design-based approach to using such observational data for variance reduction in RCTs. First, we use the observational data to train a machine learning algorithm predicting potential outcomes using covariates and then use that algorithm to generate predictions for RCT participants. Then, we use those predictions, perhaps alongside other covariates, to adjust causal effect estimates with a flexible, design-based covariate-adjustment routine. In this way, there is no danger of biases from the observational data leaking into the experimental estimates, which are guaranteed to be exactly unbiased regardless of whether the machine learning models are “correct” in any sense or whether the observational samples closely resemble RCT samples. We demonstrate the method in analyzing 33 randomized A/B tests and show that it decreases standard errors relative to other estimators, sometimes substantially.

Leveraging external controls – relevant individual patient data under control from external trials or real-world data – has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial patients given access to novel treatments. However, due to lack of randomization, RCT patients and external controls may differ with respect to covariates that may or may not have been measured. Hence, after controlling for measured covariates, for instance by matching, testing for treatment effect using external controls may still be subject to unmeasured biases. In this article, we propose a sensitivity analysis approach to quantify the magnitude of unmeasured bias that would be needed to alter the study conclusion that presumed no unmeasured biases are introduced by employing external controls. Whether leveraging external controls increases power or not depends on the interplay between sample sizes and the magnitude of treatment effect and unmeasured biases, which may be difficult to anticipate. This motivates a combined testing procedure that performs two highly correlated analyses, one with and one without external controls, with a small correction for multiple testing using the joint distribution of the two test statistics. The combined test provides a new method of sensitivity analysis designed for data fusion problems, which anchors at the unbiased analysis based on RCT only and spends a small proportion of the type I error to also test using the external controls. In this way, if leveraging external controls increases power, the power gain compared to the analysis based on RCT only can be substantial; if not, the power loss is small. The proposed method is evaluated in theory and power calculations, and applied to a real trial.

We derive a novel stochastic representation of exchangeable Marshall–Olkin distributions based on their death-counting processes. We show that these processes are Markov. Furthermore, we provide a numerically stable approximation of their infinitesimal generator matrices in the extendible case. This approach uses integral representations of Bernstein functions to calculate the generator’s first row, and then uses a recursion to calculate the remaining rows. Combining the Markov representation with the numerically stable approximation of corresponding generators allows us to sample extendible Marshall–Olkin distributions with a flexible simulation algorithm derived from known Markov sampling strategies. Finally, we benchmark an implementation of this Markov-based simulation algorithm against alternative simulation algorithms based on the Lévy frailty model, the Arnold model, and the exogenous shock model.