Optimal Two-Stage Designs Based on Restricted Mean Survival Time for a Single-Arm Study
Contemporary Clinical Trials Communications
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© 2021 The Author(s) Restricted mean survival time is an alternative measure of treatment effect to hazard ratio in clinical trials with time-to-event outcome. The current methods have been focused on one-stage designs. In this article, we propose optimal two-stage designs for a single-arm study with the smallest expected sample size. We compare the performance of the new optimal two-stage designs with the existing one-stage design with regards to the expected sample size and the expected total study length. The simulation results indicate that the new two-stage designs can save the expected sample size substantially as compared to the one-stage design. We use a non-small cell lung cancer trial to illustrate the application of the proposed designs. The proposed optimal two-stage designs are recommended for use when time for patient accrual is longer than the pre-specified follow-up time.
Optimal designs; Proportional hazards; Restricted mean survival time; Two-stage designs
Biostatistics | Clinical Trials
Optimal Two-Stage Designs Based on Restricted Mean Survival Time for a Single-Arm Study.
Contemporary Clinical Trials Communications, 21