Improved precision in the analysis of randomized trials with survival outcomes, without assuming proportional hazards
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Publication:2274689
Abstract: We present a new estimator of the restricted mean survival time in randomized trials where there is right censoring that may depend on treatment and baseline variables. The proposed estimator leverages prognostic baseline variables to obtain equal or better asymptotic precision compared to traditional estimators. Under regularity conditions and random censoring within strata of treatment and baseline variables, the proposed estimator has the following features: (i) it is interpretable under violations of the proportional hazards assumption; (ii) it is consistent and at least as precise as the Kaplan-Meier estimator under independent censoring; (iii) it remains consistent under violations of independent censoring (unlike the Kaplan-Meier estimator) when either the censoring or survival distributions are estimated consistently; and (iv) it achieves the nonparametric efficiency bound when both of these distributions are consistently estimated. We illustrate the performance of our method using simulations based on resampling data from a completed, phase 3 randomized clinical trial of a new surgical treatment for stroke; the proposed estimator achieves a 12% gain in relative efficiency compared to the Kaplan-Meier estimator. The proposed estimator has potential advantages over existing approaches for randomized trials with time-to-event outcomes, since existing methods either rely on model assumptions that are untenable in many applications, or lack some of the efficiency and consistency properties (i)-(iv). We focus on estimation of the restricted mean survival time, but our methods may be adapted to estimate any treatment effect measure defined as a smooth contrast between the survival curves for each study arm. We provide R code to implement the estimator.
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Cites work
- A Distributional Approach for Causal Inference Using Propensity Scores
- Adjusting for Nonignorable Drop-Out Using Semiparametric Nonresponse Models
- Asymptotic expansions for general statistical models. With the assist. of W. Wefelmeyer
- Asymptotic Statistics
- Bootstrap methods: another look at the jackknife
- Bounded, efficient and doubly robust estimation with inverse weighting
- Causal inference on the difference of the restricted mean lifetime between two groups
- Doubly Robust Estimation in Missing Data and Causal Inference Models
- Enhanced precision in the analysis of randomized trials with ordinal outcomes
- Estimation of Regression Coefficients When Some Regressors Are Not Always Observed
- scientific article; zbMATH DE number 1181283 (Why is no real title available?)
- scientific article; zbMATH DE number 1487686 (Why is no real title available?)
- scientific article; zbMATH DE number 2140075 (Why is no real title available?)
- scientific article; zbMATH DE number 3385132 (Why is no real title available?)
- Improved double-robust estimation in missing data and causal inference models
- Improving efficiency and robustness of the doubly robust estimator for a population mean with incomplete data
- Improving Efficiency of Inferences in Randomized Clinical Trials Using Auxiliary Covariates
- Landmark estimation of survival and treatment effect in a randomized clinical trial
- On the restricted mean survival time curve in survival analysis
- On the Role of the Propensity Score in Efficient Semiparametric Estimation of Average Treatment Effects
- Optimal treatment regimes for survival endpoints using a locally-efficient doubly-robust estimator from a classification perspective
- Robust methods to improve efficiency and reduce bias in estimating survival curves in randomized clinical trials
- Semiparametric estimation of treatment effect with time-lagged response in the presence of informative censoring
- Super Learner
- Unified methods for censored longitudinal data and causality
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- Rejoinder: Improving precision and power in randomized trials for COVID‐19 treatments using covariate adjustment, for binary, ordinal, and time‐to‐event outcomes
- Efficient t0$$ {t}_0 $$‐year risk regression using the logistic model
- Assumption-Lean Cox Regression
- Statistical inference for data-adaptive doubly robust estimators with survival outcomes
- Comment on: ``Target estimands for population-adjusted indirect comparisons
- Combining covariate adjustment with group sequential, information-adaptive designs to improve randomized trial efficiency
- Debiased learning of the causal net benefit with censored event time data
- A nonparametric framework for treatment effect modifier discovery in high dimensions
- Optimising precision and power by machine learning in randomised trials with ordinal and time-to-event outcomes with an application to COVID-19
- Robust methods to improve efficiency and reduce bias in estimating survival curves in randomized clinical trials
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