A propensity score-integrated approach for leveraging external data in a randomized controlled trial with...

Area of Science:

• Biostatistics
• Clinical Trials
• Survival Analysis

Background:

• Standard statistical tests for survival analysis in randomized controlled trials (RCTs) face challenges when external data are integrated.
• Existing methods may not directly apply to augmented RCT arms, limiting comprehensive survival difference assessments.

Purpose of the Study:

• To propose and evaluate a propensity score-integrated approach for extending commonly used statistical tests in RCTs augmented with external data.
• To provide a framework for robust survival analysis when combining trial and external patient data.

Main Methods:

• Development of propensity score-integrated statistical tests to accommodate external data in RCTs.
• Conducting simulation studies to assess the performance and operating characteristics of the proposed tests.
• Illustrative example demonstrating the practical implementation of the new procedures.

Main Results:

• The proposed propensity score-integrated approach effectively extends standard statistical tests for survival analysis.
• Simulation studies validated the operating characteristics of the developed statistical tests.
• The illustrative example confirmed the practical utility and implementation of the method.

Conclusions:

• The propensity score-integrated approach offers a viable solution for survival analysis in RCTs utilizing external data.
• This methodology enhances the ability to accurately test survival differences in complex trial designs.
• The proposed tests provide valuable tools for biostatisticians and clinical researchers working with augmented RCT data.