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Pragmatic Two-Stage Design for Clinical Trials With Treatment Selection
1H. Milton Stewart School of Industrial and Systems Engineering, Georgia Institute of Technology, Atlanta, GA, USA.
Statistics in Medicine
|July 28, 2026
Summary
This study introduces a two-stage clinical trial design for efficient treatment selection and evaluation. The pragmatic approach balances sample size and practicality, improving drug development cost-effectiveness.
Area of Science:
- Biostatistics
- Clinical Trial Design
- Pharmaceutical Development
Background:
- Clinical trials face complexity in selecting optimal treatments and evaluating efficacy simultaneously.
- Existing designs may lack efficiency or practicality in multi-treatment scenarios.
Purpose of the Study:
- To develop a pragmatic, two-stage sequential clinical trial design.
- To balance sample efficiency and practicality in treatment selection and efficacy evaluation.
- To minimize costs and improve the practicality of pharmaceutical development.
Main Methods:
- A two-stage sequential approach with adaptive arm selection and early termination in the first stage.
- Optimized sample allocation in the second stage.
- Stochastic optimization to solve a framework minimizing expected patients, bad arm selection probability, and Type II error, subject to Family-Wise Error Rate and maximum sample size constraints.
Main Results:
- The proposed method offers an efficient solution inspired by the Kiefer-Weiss problem.
- Performance comparisons against fixed and multi-arm multi-stage designs demonstrate improvements.
- The design enhances cost-effectiveness and practicality for clinical trials.
Conclusions:
- The developed two-stage sequential design provides a pragmatic and efficient approach for clinical trials.
- This methodology optimizes resource allocation and decision-making in treatment selection and evaluation.
- The findings contribute to more practical and cost-effective pharmaceutical development.
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