Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial.
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| Title: | Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial. |
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| Authors: | Park, Yeonhee1 (AUTHOR) ypark56@wisc.edu |
| Source: | Statistics in Biopharmaceutical Research. Oct-Dec2025, Vol. 17 Issue 4, p557-566. 10p. |
| Subjects: | Clinical trials, Sequential analysis, Experimental design, T-test (Statistics), Simulation methods & models |
| Abstract: | Surrogate endpoints are pivotal in clinical trials, replacing true endpoints that may be challenging to measure. However, their imprecise association with true endpoints can lead to biased results, necessitating validation through trials. This article proposes a bias-corrected group sequential design in the presence of surrogate endpoints to address these challenges. We introduce bias-corrected test statistics tailored to scenarios where both true and surrogate endpoints are available or where true endpoints are limited. Our approach is exemplified through a simulation study and a case study redesign of the Pamoja Tulinde Maisha (PALM) trial, showcasing its practical implementation and benefits. The proposed design enhances the accuracy of interim analyses by mitigating biases associated with surrogate endpoints. Through rigorous evaluation and comparison with alternative designs, we demonstrate the effectiveness of the proposed methods. [ABSTRACT FROM AUTHOR] |
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| Database: | Engineering Source |
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| Abstract: | Surrogate endpoints are pivotal in clinical trials, replacing true endpoints that may be challenging to measure. However, their imprecise association with true endpoints can lead to biased results, necessitating validation through trials. This article proposes a bias-corrected group sequential design in the presence of surrogate endpoints to address these challenges. We introduce bias-corrected test statistics tailored to scenarios where both true and surrogate endpoints are available or where true endpoints are limited. Our approach is exemplified through a simulation study and a case study redesign of the Pamoja Tulinde Maisha (PALM) trial, showcasing its practical implementation and benefits. The proposed design enhances the accuracy of interim analyses by mitigating biases associated with surrogate endpoints. Through rigorous evaluation and comparison with alternative designs, we demonstrate the effectiveness of the proposed methods. [ABSTRACT FROM AUTHOR] |
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| ISSN: | 19466315 |
| DOI: | 10.1080/19466315.2024.2432906 |
