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.
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A propensity score-integrated approach for leveraging external data in a randomized controlled trial with time-to-event endpoints.
Pharm Stat
November 2024
Division of Biostatistics, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
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.
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Division of Biostatistics, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
The propensity score-integrated composite likelihood (PSCL) method is one method that can be utilized to design and analyze an application when real-world data (RWD) are leveraged to augment a prospectively designed clinical study. In the PSCL, strata are formed based on propensity scores (PS) such that similar subjects in terms of the baseline covariates from both the current study and RWD sources are placed in the same stratum, and then composite likelihood method is applied to down-weight the information from the RWD. While PSCL was originally proposed for a fixed design, it can be extended to be applied under an adaptive design framework with the purpose to either potentially claim an early success or to re-estimate the sample size.
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