Optimal Two-Stage Designs Based on Restricted Mean Survival Time for a Single-Arm Study

Document Type

Article

Publication Date

1-23-2021

Publication Title

Contemporary Clinical Trials Communications

Volume

21

First page number:

1

Last page number:

7

Abstract

© 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.

Keywords

Optimal designs; Proportional hazards; Restricted mean survival time; Two-stage designs

Disciplines

Biostatistics | Clinical Trials

Language

English

UNLV article access

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