Multi-arm multi-stage survival trial design with arm-specific stopping rule

Traditional two-arm randomized trial designs have played a pivotal role in establishing the efficacy of medical interventions. However, their efficiency is often compromised when confronted with multiple experimental treatments or limited resources. In response to these challenges, the multi-arm multi-stage designs have emerged, enabling the simultaneous evaluation of multiple treatments within a single trial. In such an approach, if an arm meets efficacy success criteria at an interim stage, the whole trial stops and the arm is selected for further study. However when multiple treatment arms are active, stopping the trial at the moment one arm achieves success diminishes the probability of selecting the best arm. To address this issue, we have developed a group sequential multi-arm multi-stage survival trial design with an arm-specific stopping rule. The proposed method controls the familywise type I error in a strong sense and selects the best promising treatment arm with a high probability.

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Publication Details

Journal
Journal of Biopharmaceutical Statistics
Published
2024-09-16
DOI
https://doi.org/10.1080/10543406.2024.2398036
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
0.00

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article

Multi-arm multi-stage survival trial design with arm-specific stopping rule

Liang Zhu, Jianrong Wu, Yimei Li, Tushar Panti
Journal of Biopharmaceutical Statistics
Statistical Methods in Clinical Trials
article

Multi-arm multi-stage survival trial design with arm-specific stopping rule

Liang Zhu, Jianrong Wu, Yimei Li, Tushar Panti
article en

Abstract

Traditional two-arm randomized trial designs have played a pivotal role in establishing the efficacy of medical interventions. However, their efficiency is often compromised when confronted with multiple experimental treatments or limited resources. In response to these challenges, the multi-arm multi-stage designs have emerged, enabling the simultaneous evaluation of multiple treatments within a single trial. In such an approach, if an arm meets efficacy success criteria at an interim stage, the whole trial stops and the arm is selected for further study. However when multiple treatment arms are active, stopping the trial at the moment one arm achieves success diminishes the probability of selecting the best arm. To address this issue, we have developed a group sequential multi-arm multi-stage survival trial design with an arm-specific stopping rule. The proposed method controls the familywise type I error in a strong sense and selects the best promising treatment arm with a high probability.

Journal of Biopharmaceutical StatisticsVol. 36(2)
St. Jude Children's Research Hospital (US), University of New Mexico (US), New Mexico Cancer Center (US), UNM Comprehensive Cancer Center, The University of Texas Health Science Center at Houston (US)
American Lebanese Syrian Associated Charities, National Cancer Institute
Openalex Percentile: Top 84%
Statistical Methods in Clinical Trials
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Multi-arm multi-stage survival trial design with arm-specific stopping rule — Liang Zhu, Jianrong Wu, et al. · Journal of Biopharmaceutical Statistics (2024) | TGRS Research Map | TGRS