arXiv subjects

Nandita Mitra

Publications and source records attributed to Nandita Mitra.

25 records · Page 2Linked to original sources

arXiv2020

Doubly Robust Nonparametric Instrumental Variable Estimators for Survival Outcomes

Instrumental variable (IV) methods allow us the opportunity to address unmeasured confounding in causal inference. However, most IV methods are only applicable to discrete or continuous outcomes with very few IV methods for censored survival outcomes. In this work we propose nonparametric estimators for the local average treatment effect on survival probabilities under both nonignorable and ignorable censoring. We provide an efficient influence function-based estimator and a simple estimation procedure when the IV is either binary or continuous. The proposed estimators possess double-robustness properties and can easily incorporate nonparametric estimation using machine learning tools. In simulation studies, we demonstrate the flexibility and efficiency of our proposed estimators under various plausible scenarios. We apply our method to the Prostate, Lung, Colorectal, and Ovarian Cancer Screening Trial for estimating the causal effect of screening on survival probabilities and investigate the causal contrasts between the two interventions under different censoring assumptions.

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arXiv2020

Bayesian Nonparametric Cost-Effectiveness Analyses: Causal Estimation and Adaptive Subgroup Discovery

Cost-effectiveness analyses (CEAs) are at the center of health economic decision making. While these analyses help policy analysts and economists determine coverage, inform policy, and guide resource allocation, they are statistically challenging for several reasons. Cost and effectiveness are correlated and follow complex joint distributions which are difficult to capture parametrically. Effectiveness (often measured as increased survival time) and accumulated cost tends to be right-censored in many applications. Moreover, CEAs are often conducted using observational data with non-random treatment assignment. Policy-relevant causal estimation therefore requires robust confounding control. Finally, current CEA methods do not address cost-effectiveness heterogeneity in a principled way - often presenting population-averaged estimates even though significant effect heterogeneity may exist. Motivated by these challenges, we develop a nonparametric Bayesian model for joint cost-survival distributions in the presence of censoring. Our approach utilizes a joint Enriched Dirichlet Process prior on the covariate effects of cost and survival time, while using a Gamma Process prior on the baseline survival time hazard. Causal CEA estimands, with policy-relevant interpretations, are identified and estimated via a Bayesian nonparametric g-computation procedure. Finally, we outline how the induced clustering of the Enriched Dirichlet Process can be used to adaptively detect presence of subgroups with different cost-effectiveness profiles. We outline an MCMC procedure for full posterior inference and evaluate frequentist properties via simulations. We use our model to assess the cost-efficacy of chemotherapy versus radiation adjuvant therapy for treating endometrial cancer in the SEER-Medicare database.

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arXiv2019

Net benefit separation and the determination curve: a probabilistic framework for cost-effectiveness estimation

Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy and promote adequate resource allocation. The net monetary benefit aggregates information on differences in mean cost and clinical outcomes; the cost-effectiveness acceptability curve was then developed to characterize the extent to which the strength of evidence regarding net monetary benefit changes with fluctuations in the willingness-to-pay threshold. Methods to derive insights from characteristics of the cost/clinical outcomes besides mean differences remain undeveloped but may also be informative. We propose a novel probabilistic measure of cost-effectiveness based on the stochastic ordering of the individual net benefit distribution under each treatment. Our approach is able to accommodate features frequently encountered in observational data including confounding and censoring, and complements the net monetary benefit in the insights it provides. We conduct a range of simulations to evaluate finite-sample performance and illustrate our proposed approach using simulated data based on a study of endometrial cancer patients.

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arXiv2019

A Bayesian Nonparametric Model for Zero-Inflated Outcomes: Prediction, Clustering, and Causal Estimation

Researchers are often interested in predicting outcomes, conducting clustering analysis to detect distinct subgroups of their data, or computing causal treatment effects. Pathological data distributions that exhibit skewness and zero-inflation complicate these tasks - requiring highly flexible, data-adaptive modeling. In this paper, we present a fully nonparametric Bayesian generative model for continuous, zero-inflated outcomes that simultaneously predicts structural zeros, captures skewness, and clusters patients with similar joint data distributions. The flexibility of our approach yields predictions that capture the joint data distribution better than commonly used zero-inflated methods. Moreover, we demonstrate that our model can be coherently incorporated into a standardization procedure for computing causal effect estimates that are robust to such data pathologies. Uncertainty at all levels of this model flow through to the causal effect estimates of interest - allowing easy point estimation, interval estimation, and posterior predictive checks verifying positivity, a required causal identification assumption. Our simulation results show point estimates to have low bias and interval estimates to have close to nominal coverage under complicated data settings. Under simpler settings, these results hold while incurring lower efficiency loss than comparator methods. Lastly, we use our proposed method to analyze zero-inflated inpatient medical costs among endometrial cancer patients receiving either chemotherapy and radiation therapy in the SEER medicare database.

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arXiv2017

Estimating scaled treatment effects with multiple outcomes

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on multiple outcomes simultaneously. Such designs can be particularly useful in patient-centered research, where different outcomes might be more or less important to different patients. In this paper we propose scaled effect measures (via potential outcome notation) that translate effects on multiple outcomes to a common scale, using mean-variance and median-interquartile-range -based standardizations. We present efficient, nonparametric, doubly robust methods for estimating these scaled effects (and weighted average summary measures), and for testing the null hypothesis that treatment affects all outcomes equally. We also discuss methods for exploring how treatment effects depend on covariates (i.e., effect modification). In addition to describing efficiency theory for our estimands and the asymptotic behavior of our estimators, we illustrate the methods in a simulation study and a data analysis. Importantly, and in contrast to much of the literature concerning effects on multiple outcomes, our methods are nonparametric and can be used not only in randomized trials to yield increased efficiency, but also in observational studies with high-dimensional covariates to reduce confounding bias.

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arXiv2017

A causal approach to analysis of censored medical costs in the presence of time-varying treatment

There has recently been a growing interest in the development of statistical methods to compare medical costs between treatment groups. When cumulative cost is the outcome of interest, right-censoring poses the challenge of informative missingness due to heterogeneity in the rates of cost accumulation across subjects. Existing approaches seeking to address the challenge of informative cost trajectories typically rely on inverse probability weighting and target a net "intent-to-treat" effect. However, no approaches capable of handling time-dependent treatment and confounding in this setting have been developed to date. A method to estimate the joint causal effect of a treatment regime on cost would be of value to inform public policy when comparing interventions. In this paper, we develop a nested g-computation approach to cost analysis in order to accommodate time-dependent treatment and repeated outcome measures. We demonstrate that our procedure is reasonably robust to departures from its distributional assumptions and can provide unique insights into fundamental differences in average cost across time-dependent treatment regimes.

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arXiv2014

Evaluating costs with unmeasured confounding: A sensitivity analysis for the treatment effect

Estimates of the effects of treatment on cost from observational studies are subject to bias if there are unmeasured confounders. It is therefore advisable in practice to assess the potential magnitude of such biases. We derive a general adjustment formula for loglinear models of mean cost and explore special cases under plausible assumptions about the distribution of the unmeasured confounder. We assess the performance of the adjustment by simulation, in particular, examining robustness to a key assumption of conditional independence between the unmeasured and measured covariates given the treatment indicator. We apply our method to SEER-Medicare cost data for a stage II/III muscle-invasive bladder cancer cohort. We evaluate the costs for radical cystectomy vs. combined radiation/chemotherapy, and find that the significance of the treatment effect is sensitive to plausible unmeasured Bernoulli, Poisson and Gamma confounders.

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