A propensity score-integrated approach for leveraging external data in a randomized controlled trial with time-to-event endpoints

Pharm Stat. 2024 Sep-Oct;23(5):645-661. doi: 10.1002/pst.2377. Epub 2024 Mar 5.

Abstract

In a randomized controlled trial with time-to-event endpoint, some commonly used statistical tests to test for various aspects of survival differences, such as survival probability at a fixed time point, survival function up to a specific time point, and restricted mean survival time, may not be directly applicable when external data are leveraged to augment an arm (or both arms) of an RCT. In this paper, we propose a propensity score-integrated approach to extend such tests when external data are leveraged. Simulation studies are conducted to evaluate the operating characteristics of three propensity score-integrated statistical tests, and an illustrative example is given to demonstrate how these proposed procedures can be implemented.

Keywords: PSKM; RMST; log‐rank test; survival function; time‐to‐event.

MeSH terms

  • Computer Simulation*
  • Data Interpretation, Statistical
  • Endpoint Determination* / methods
  • Endpoint Determination* / statistics & numerical data
  • Humans
  • Models, Statistical
  • Propensity Score*
  • Randomized Controlled Trials as Topic* / methods
  • Randomized Controlled Trials as Topic* / statistics & numerical data
  • Research Design
  • Survival Analysis
  • Time Factors