In the absence of data from a randomized trial, researchers may aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the treatment-specific survival curves, that is, the survival curves were the population under study to be assigned to receive the treatment or not. Under certain conditions, including that all confounders of the treatment-outcome relationship are observed, the treatment-specific survival curve can be identified with a covariate-adjusted survival curve. In this article, we propose a novel cross-fitted doubly-robust estimator that incorporates data-adaptive (e.g. machine learning) estimators of the conditional survival functions. We establish conditions on the nuisance estimators under which our estimator is consistent and asymptotically linear, both pointwise and uniformly in time. We also propose a novel ensemble learner for combining multiple candidate estimators of the conditional survival estimators. Notably, our methods and results accommodate events occurring in discrete or continuous time, or an arbitrary mix of the two. We investigate the practical performance of our methods using numerical studies and an application to the effect of a surgical treatment to prevent metastases of parotid carcinoma on mortality.
more »
« less
A nonparametric doubly robust test for a continuous treatment effect
The vast majority of literature on evaluating the significance of a treatment effect based on observational data has been confined to discrete treatments. These methods are not applicable to drawing inference for a continuous treatment, which arises in many important applications. To adjust for confounders when evaluating a continuous treatment, existing inference methods often rely on discretizing the treatment or using (possibly misspecified) parametric models for the effect curve. Recently, Kennedy et al. (J. R. Stat. Soc. Ser. B. Stat. Methodol. 79 (2017) 1229–1245) proposed nonparametric doubly robust estimation for a continuous treatment effect in observational studies. However, inference for the continuous treatment effect is a harder problem. To the best of our knowledge, a completely nonparametric doubly robust approach for inference in this setting is not yet available. We develop such a nonparametric doubly robust procedure in this paper for making inference on the continuous treatment effect curve. Using empirical process techniques for local U- and V-processes, we establish the test statistic’s asymptotic distribution. Furthermore, we propose a wild bootstrap procedure for implementing the test in practice. In addition, we define a version of the test procedure based on sample splitting. We illustrate the new method(s) via simulations and a study of a constructed dataset relating the effect of nurse staffing hours on hospital performance. We implement our doubly robust dose response test in the R package DRDRtest on CRAN.
more »
« less
- Award ID(s):
- 2210312
- PAR ID:
- 10629036
- Publisher / Repository:
- Institute of Mathematical Statistics
- Date Published:
- Journal Name:
- The Annals of Statistics
- Volume:
- 52
- Issue:
- 4
- ISSN:
- 0090-5364
- Subject(s) / Keyword(s):
- Causal inference , double robustness , hypothesis test , nonparametric , U-statistics
- Format(s):
- Medium: X
- Sponsoring Org:
- National Science Foundation
More Like this
-
-
Abstract Post-treatment variables often complicate causal inference. They appear in many scientific problems, including non-compliance, truncation by death, mediation, and surrogate endpoint evaluation. Principal stratification is a strategy to address these challenges by adjusting for the potential values of the post-treatment variables, defined as the principal strata. It allows for characterizing treatment effect heterogeneity across principal strata and unveiling the mechanism of the treatment’s impact on the outcome related to post-treatment variables. However, the existing literature has primarily focused on binary post-treatment variables, leaving the case with continuous post-treatment variables largely unexplored. This gap persists due to the complexity of infinitely many principal strata, which present challenges to both the identification and estimation of causal effects. We fill this gap by providing nonparametric identification and semiparametric estimation theory for principal stratification with continuous post-treatment variables. We propose to use working models to approximate the underlying causal effect surfaces and derive the efficient influence functions of the corresponding model parameters. Based on the theory, we construct doubly robust estimators and implement them in the R package continuousPCE.more » « less
-
Abstract 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 reliable interval estimates for counterfactuals and individual treatment effects under the potential outcome framework. For completely randomized or stratified randomized experiments with perfect compliance, the intervals have guaranteed average coverage in finite samples regardless of the unknown data generating mechanism. For randomized experiments with ignorable compliance and general observational studies obeying the strong ignorability assumption, the intervals satisfy a doubly robust property which states the following: the average coverage is approximately controlled if either the propensity score or the conditional quantiles of potential outcomes can be estimated accurately. Numerical studies on both synthetic and real data sets empirically demonstrate that existing methods suffer from a significant coverage deficit even in simple models. In contrast, our methods achieve the desired coverage with reasonably short intervals.more » « less
-
Abstract Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces the covariate balance between the RCT and OS, therefore improving the trial-based estimator's generalizability. Exploiting semiparametric efficiency theory, we propose a doubly robust augmented calibration weighting estimator that achieves the efficiency bound derived under the identification assumptions. A nonparametric sieve method is provided as an alternative to the parametric approach, which enables the robust approximation of the nuisance functions and data-adaptive selection of outcome predictors for calibration. We establish asymptotic results and confirm the finite sample performances of the proposed estimators by simulation experiments and an application on the estimation of the treatment effect of adjuvant chemotherapy for early-stage non-small-cell lung patients after surgery.more » « less
-
Estimating the effect of treatments from natural experiments, where treatments are pre-assigned, is an important and well-studied problem. We introduce a novel natural experiment dataset obtained from an early childhood literacy nonprofit. Surprisingly, applying over 20 established estimators to the dataset produces inconsistent results in evaluating the nonprofits efficacy. To address this, we create a benchmark to evaluate estimator accuracy using synthetic outcomes, whose design was guided by domain experts. The benchmark extensively explores performance as real world conditions like sample size, treatment correlation, and propensity score accuracy vary. Based on our benchmark, we observe that the class of doubly robust treatment effect estimators, which are based on simple and intuitive regression adjustment, generally outperform other more complicated estimators by orders of magnitude. To better support our theoretical understanding of doubly robust estimators, we derive a closed form expression for the variance of any such estimator that uses dataset splitting to obtain an unbiased estimate. This expression motivates the design of a new doubly robust estimator that uses a novel loss function when fitting functions for regression adjustment. We release the dataset and benchmark in a Python package; the package is built in a modular way to facilitate new datasets and estimators. https://github.com/rtealwitter/naturalexperiments.more » « less
An official website of the United States government

