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

Author:

Chen Wei‐Chen1ORCID,Lu Nelson1ORCID,Wang Chenguang2,Li Heng1ORCID,Song Changhong1ORCID,Tiwari Ram3,Xu Yunling1ORCID,Yue Lilly Q.1

Affiliation:

1. Division of Biostatistics Center for Devices and Radiological Health, U.S. Food and Drug Administration Silver Spring Maryland USA

2. Regeneron Pharmaceuticals Tarrytown New York USA

3. Bristol Myers Squibb Lawrence Township New Jersey USA

Abstract

AbstractIn 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.

Publisher

Wiley

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