No Arabic abstract
The Consent-to-Contact (C2C) registry at the University of California, Irvine collects data from community participants to aid in the recruitment to clinical research studies. Self-selection into the C2C likely leads to bias due in part to enrollees having more years of education relative to the US general population. Salazar et al. (2020) recently used the C2C to examine associations of race/ethnicity with participant willingness to be contacted about research studies. To address questions about generalizability of estimated associations we estimate propensity for self-selection into the convenience sample weights using data from the National Health and Nutrition Examination Survey (NHANES). We create a combined dataset of C2C and NHANES subjects and compare different approaches (logistic regression, covariate balancing propensity score, entropy balancing, and random forest) for estimating the probability of membership in C2C relative to NHANES. We propose methods to estimate the variance of parameter estimates that account for uncertainty that arises from estimating propensity weights. Simulation studies explore the impact of propensity weight estimation on uncertainty. We demonstrate the approach by repeating the analysis by Salazar et al. with the deduced propensity weights for the C2C subjects and contrast the results of the two analyses. This method can be implemented using our estweight package in R available on GitHub.
The inverse probability weighting approach is popular for evaluating treatment effects in observational studies, but extreme propensity scores could bias the estimator and induce excessive variance. Recently, the overlap weighting approach has been proposed to alleviate this problem, which smoothly down-weighs the subjects with extreme propensity scores. Although advantages of overlap weighting have been extensively demonstrated in literature with continuous and binary outcomes, research on its performance with time-to-event or survival outcomes is limited. In this article, we propose two weighting estimators that combine propensity score weighting and inverse probability of censoring weighting to estimate the counterfactual survival functions. These estimators are applicable to the general class of balancing weights, which includes inverse probability weighting, trimming, and overlap weighting as special cases. We conduct simulations to examine the empirical performance of these estimators with different weighting schemes in terms of bias, variance, and 95% confidence interval coverage, under various degree of covariate overlap between treatment groups and censoring rate. We demonstrate that overlap weighting consistently outperforms inverse probability weighting and associated trimming methods in bias, variance, and coverage for time-to-event outcomes, and the advantages increase as the degree of covariate overlap between the treatment groups decreases.
Hierarchical inference in (generalized) regression problems is powerful for finding significant groups or even single covariates, especially in high-dimensional settings where identifiability of the entire regression parameter vector may be ill-posed. The general method proceeds in a fully data-driven and adaptive way from large to small groups or singletons of covariates, depending on the signal strength and the correlation structure of the design matrix. We propose a novel hierarchical multiple testing adjustment that can be used in combination with any significance test for a group of covariates to perform hierarchical inference. Our adjustment passes on the significance level of certain hypotheses that could not be rejected and is shown to guarantee strong control of the familywise error rate. Our method is at least as powerful as a so-called depth-wise hierarchical Bonferroni adjustment. It provides a substantial gain in power over other previously proposed inheritance hierarchical procedures if the underlying alternative hypotheses occur sparsely along a few branches in the tree-structured hierarchy.
A straightforward application of semi-supervised machine learning to the problem of treatment effect estimation would be to consider data as unlabeled if treatment assignment and covariates are observed but outcomes are unobserved. According to this formulation, large unlabeled data sets could be used to estimate a high dimensional propensity function and causal inference using a much smaller labeled data set could proceed via weighted estimators using the learned propensity scores. In the limiting case of infinite unlabeled data, one may estimate the high dimensional propensity function exactly. However, longstanding advice in the causal inference community suggests that estimated propensity scores (from labeled data alone) are actually preferable to true propensity scores, implying that the unlabeled data is actually useless in this context. In this paper we examine this paradox and propose a simple procedure that reconciles the strong intuition that a known propensity functions should be useful for estimating treatment effects with the previous literature suggesting otherwise. Further, simulation studies suggest that direct regression may be preferable to inverse-propensity weight estimators in many circumstances.
The increasing prevalence of rich sources of data and the availability of electronic medical record databases and electronic registries opens tremendous opportunities for enhancing medical research. For example, controlled trials are ubiquitously used to investigate the effect of a medical treatment, perhaps dependent on a set of patient covariates, and traditional approaches have relied primarily on randomized patient sampling and allocation to treatment and control group. However, when covariate data for a large cohort group of patients have already been collected and are available in a database, one can potentially design a treatment/control sample and allocation that provides far better estimates of the covariate-dependent effects of the treatment. In this paper, we develop a new approach that uses optimal design of experiments (DOE) concepts to accomplish this objective. The approach selects the patients for the treatment and control samples upfront, based on their covariate values, in a manner that optimizes the information content in the data. For the optimal sample selection, we develop simple guidelines and an optimization algorithm that provides solutions that are substantially better than random sampling. Moreover, our approach causes no sampling bias in the estimated effects, for the same reason that DOE principles do not bias estimated effects. We test our method with a simulation study based on a testbed data set containing information on the effect of statins on low-density lipoprotein (LDL) cholesterol.
The popularity of online surveys has increased the prominence of using weights that capture units probabilities of inclusion for claims of representativeness. Yet, much uncertainty remains regarding how these weights should be employed in the analysis of survey experiments: Should they be used or ignored? If they are used, which estimators are preferred? We offer practical advice, rooted in the Neyman-Rubin model, for researchers producing and working with survey experimental data. We examine simple, efficient estimators for analyzing these data, and give formulae for their biases and variances. We provide simulations that examine these estimators as well as real examples from experiments administered online through YouGov. We find that for examining the existence of population treatment effects using high-quality, broadly representative samples recruited by top online survey firms, sample quantities, which do not rely on weights, are often sufficient. We found that Sample Average Treatment Effect (SATE) estimates did not appear to differ substantially from their weighted counterparts, and they avoided the substantial loss of statistical power that accompanies weighting. When precise estimates of Population Average Treatment Effects (PATE) are essential, we analytically show post-stratifying on survey weights and/or covariates highly correlated with the outcome to be a conservative choice. While we show these substantial gains in simulations, we find limited evidence of them in practice.