A simple, doubly robust, efficient estimator for survival functions using pseudo observations

Pharm Stat. 2018 Feb;17(1):38-48. doi: 10.1002/pst.1834. Epub 2017 Nov 1.

Abstract

Survival functions are often estimated by nonparametric estimators such as the Kaplan-Meier estimator. For valid estimation, proper adjustment for confounding factors is needed when treatment assignment may depend on confounding factors. Inverse probability weighting is a commonly used approach, especially when there is a large number of potential confounders to adjust for. Direct adjustment may also be used if the relationship between the time-to-event and all confounders can be modeled. However, either approach requires a correctly specified model for the relationship between confounders and treatment allocation or between confounders and the time-to-event. We propose a pseudo-observation-based doubly robust estimator, which is valid when either the treatment allocation model or the time-to-event model is correctly specified and is generally more efficient than the inverse probability weighting approach. The approach can be easily implemented using standard software. A simulation study was conducted to evaluate this approach under a number of scenarios, and the results are presented and discussed. The results confirm robustness and efficiency of the proposed approach. A real data example is also provided for illustration.

Keywords: Kaplan-Meier estimator; causal inference; doubly robust; inverse probability weighting; pseudo observation.

Publication types

  • Review

MeSH terms

  • Breast Neoplasms / mortality
  • Computer Simulation*
  • Data Interpretation, Statistical*
  • Female
  • Humans
  • Models, Theoretical*
  • Survival Analysis