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Optimal two-stage designs based on restricted mean survival time for a single-arm study
1Department of Epidemiology and Biostatistics, School of Public Health, University of Nevada Las Vegas, Las Vegas, NV, 89154, USA.
Contemporary Clinical Trials Communications
|February 8, 2021
Summary
New two-stage clinical trial designs using restricted mean survival time offer substantial sample size savings compared to traditional one-stage methods. These designs are particularly beneficial when patient accrual time exceeds follow-up duration.
Area of Science:
- Clinical Trials
- Biostatistics
- Survival Analysis
Background:
- Restricted mean survival time (RMST) is an alternative to hazard ratio for assessing treatment effects in time-to-event clinical trials.
- Current methodologies for RMST primarily focus on single-stage trial designs.
Purpose of the Study:
- To propose novel optimal two-stage designs for single-arm studies utilizing RMST.
- To minimize the expected sample size for these studies.
Main Methods:
- Developed and evaluated optimal two-stage designs for single-arm clinical trials.
- Compared the performance of proposed two-stage designs against existing one-stage designs.
- Assessed metrics including expected sample size and expected total study length through simulations.
Main Results:
- The proposed two-stage designs demonstrated substantial reductions in expected sample size compared to one-stage designs.
- Simulations confirmed the efficiency of the new designs.
Conclusions:
- Optimal two-stage designs are recommended for single-arm studies employing RMST, especially when patient accrual time is longer than the planned follow-up.
- These designs offer significant advantages in sample size efficiency for clinical trials.
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