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An automation-based adaptive seamless design for dose selection and confirmation with improved power and efficiency
Lu Cui1, Tianyu Zhan2, Lanju Zhang2
1Statistical Science and Innovation, UCB Biosciences, Raleigh, NC, USA.
Statistical Methods in Medical Research
|January 18, 2021
Summary
This study introduces an efficient adaptive design for drug development, integrating dose finding and confirmation into a single pivotal trial. This approach improves efficiency by utilizing response adaptive randomization and sample size modification.
Area of Science:
- Clinical Trials
- Biostatistics
- Drug Development
Background:
- Traditional drug development uses separate Phase 2b dose-ranging and Phase 3 confirmation studies.
- This sequential approach is inefficient, prolonging development and excluding early data from final efficacy confirmation.
Purpose of the Study:
- To propose a novel adaptive design that integrates dose-finding and confirmation into one pivotal study.
- To enhance efficiency in drug development by overcoming limitations of traditional two-step approaches.
Main Methods:
- Implementing a new adaptive design combining response adaptive randomization, sample size modification, and multiple testing techniques.
- Utilizing an automated randomization process for seamless integration of study phases.
Main Results:
- The proposed design achieves greater efficiency compared to existing adaptive seamless designs.
- It allows for the selection of targeted doses with confirmed effectiveness and controlled family-wise error rate.
Conclusions:
- The novel adaptive design offers a more efficient and integrated approach to drug development.
- This method streamlines the process, ensuring robust efficacy confirmation while optimizing resource utilization.
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