Propensity score methods are widely used in observational studies for evaluating marginal treatment effects. The generalized propensity score (GPS) is an extension of the propensity score framework, historically developed in the case of binary exposures, for use with quantitative or continuous exposures. In this paper, we proposed variance esti-mators for treatment effect estimators on continuous outcomes. Dose-response functions (DRF) were estimated through weighting on the inverse of the GPS, or using stratification. Variance estimators were evaluated using Monte Carlo simulations. Despite the use of stabilized weights, the variability of the weighted estimator of the DRF was particularly high, and none of the variance estimators (a bootstrap-based estimator, a closed-form estimator especially developped to take into account the estimation step of the GPS, and a sandwich estimator) were able to adequately capture this variability, resulting in coverages below to the nominal value, particularly when the proportion of the variation in the quantitative exposure explained by the covariates was 1 large. The stratified estimator was more stable, and variance estima-tors (a bootstrap-based estimator, a pooled linearized estimator, and a pooled model-based estimator) more efficient at capturing the empirical variability of the parameters of the DRF. The pooled variance estimators tended to overestimate the variance, whereas the bootstrap estimator, which intrinsically takes into account the estimation step of the GPS, resulted in correct variance estimations and coverage rates. These methods were applied to a real data set with the aim of assessing the effect of maternal body mass index on newborn birth weight.
Recent measurement technologies enable us to analyze baseball at higher levels. There are, however, still many unclear points around the pitching strategy. The two elements make it difficult to measure the effect of pitching strategy. First, most public datasets do not include location data where the catcher demands a ball, which is essential information to obtain the battery's intent. Second, there are many confounders associated with pitching/batting results when evaluating pitching strategy. We here clarify the effect of pitching attempts to a specific location, e.g., inside or outside. We …
This paper constructs a doubly robust estimator for continuous dose-response estimation. An outcome regression model is augmented with a set of inverse generalized propensity score covariates to correct for potential misspecification bias. From the augmented model we can obtain consistent estimates of mean average potential outcomes for distinct strata of the treatment. A polynomial regression is then fitted to these point estimates to derive a Taylor approximation to the continuous dose-response function. The bootstrap is used for variance estimation. Analytical results and simulations show t…
The generalized propensity score (GPS) is an extension of the propensity score for use with quantitative or continuous exposures (e.g., dose of medication or years of education). Current GPS methods allow estimation of the dose-response relationship between a single continuous exposure and an outcome. However, in many real-world settings, there are multiple exposures occurring simultaneously that could be causally related to the outcome. We propose a multivariate GPS method (mvGPS) that allows estimation of a dose-response surface that relates the joint distribution of multiple continuous expo…
This paper develops a multiply robust (MR) dose-response estimator for causal inference problems involving multivalued treatments. We combine a family of generalised propensity score (GPS) models and a family of outcome regression (OR) models to achieve an average potential outcomes estimator that is consistent if just one of the GPS or OR models in each family is correctly specified. We provide proofs and simulations that demonstrate multiple robustness in the context of multivalued causal inference problems.
Propensity score methods have become a part of the standard toolkit for applied researchers who wish to ascertain causal effects from observational data. While they were originally developed for binary treatments, several researchers have proposed generalizations of the propensity score methodology for non-binary treatment regimes. Such extensions have widened the applicability of propensity score methods and are indeed becoming increasingly popular themselves. In this article, we closely examine the two main generalizations of propensity score methods, namely, the propensity function (P-FUNCT…
Optimal propensity score matching has emerged as one of the most ubiquitous approaches for causal inference studies on observational data; However, outstanding critiques of the statistical properties of propensity score matching have cast doubt on the statistical efficiency of this technique, and the poor scalability of optimal matching to large data sets makes this approach inconvenient if not infeasible for sample sizes that are increasingly commonplace in modern observational data. The stratamatch package provides implementation support and diagnostics for `stratified matching designs,' an …
Evidence from animal models and epidemiological studies has linked prenatal alcohol exposure (PAE) to a broad range of long-term cognitive and behavioral deficits. However, there is virtually no information in the scientific literature regarding the levels of PAE associated with an increased risk of clinically significant adverse effects. During the period from 1975-1993, several prospective longitudinal cohort studies were conducted in the U.S., in which maternal reports regarding alcohol use were obtained during pregnancy and the cognitive development of the offspring was assessed from early…
Motivated by a clinical trial conducted by Janssen Pharmaceuticals in which a flexible dosing regimen is compared to placebo, we evaluate how switchers in the treatment arm (i.e., patients who were switched to the higher dose) would have fared had they been kept on the low dose. This in order to understand whether flexible dosing is potentially beneficial for them. Simply comparing these patients' responses with those of patients who stayed on the low dose is unsatisfactory because the latter patients are usually in a better health condition. Because the available information in the considered…
Modern longitudinal studies collect feature data at many timepoints, often of the same order of sample size. Such studies are typically affected by {dropout} and positivity violations. We tackle these problems by generalizing effects of recent incremental interventions (which shift propensity scores rather than set treatment values deterministically) to accommodate multiple outcomes and subject dropout. We give an identifying expression for incremental intervention effects when dropout is conditionally ignorable (without requiring treatment positivity), and derive the nonparametric efficiency …
Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises because the time to disease progression can be censored by the time to normal tissue complications, and vice versa. Besides, much literature has examined that patients receiving the same radiotherapy dose may yield distinct responses due to their heterogeneous radiation susceptibility statuses. Therefore, the "one-dose-fit-all" strategy often fails, and it is m…
Evaluating treatment effect heterogeneity widely informs treatment decision making. At the moment, much emphasis is placed on the estimation of the conditional average treatment effect via flexible machine learning algorithms. While these methods enjoy some theoretical appeal in terms of consistency and convergence rates, they generally perform poorly in terms of uncertainty quantification. This is troubling since assessing risk is crucial for reliable decision-making in sensitive and uncertain environments. In this work, we propose a conformal inference-based approach that can produce reliabl…
The dose regimen of Warfarin is separated into two phases. Firstly a loading dose is given, which is designed to bring the International Normalisation Ratio (INR) to within therapeutic range. Then a stable maintenance dose is given to maintain the INR within therapeutic range. In the United Kingdom (UK) the loading dose is usually given as three individual daily doses, the standard loading dose being 10mg on days one and two and 5mgs on day three, which can be varied at the discretion of the clinician. However, due to the large inter-individual variation in the response to Warfarin therapy, it…
