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A gated group sequential design for seamless Phase II/III trial with subpopulation selection.
Guanhong Miao1, Jason J Z Liao2, Jing Yang3
1Department of Biostatistics, University of Florida, Gainesville, FL, 32611, USA.
BMC Medical Research Methodology
|January 3, 2023
Summary
This study introduces a gated group sequential design for seamless Phase II/III trials, improving efficiency by adapting population selection. This approach offers greater power and resource savings compared to traditional methods.
Area of Science:
- Clinical trial design
- Biostatistics
- Pharmaceutical development
Background:
- Phase III trials are costly and have high failure rates, often using group sequential designs (GSD).
- Seamless Phase II/III designs enhance trial efficiency by incorporating a proof-of-concept stage before full Phase III commitment.
- Adaptive population selection focuses investment on patient subgroups most likely to benefit.
Purpose of the Study:
- To develop an adaptive population selection method for seamless Phase II/III trials.
- To evaluate a gated group sequential design for trials with one or two primary endpoints.
Main Methods:
- Proposed a gated group sequential design for seamless Phase II/III trials.
- Incorporated adaptive population selection and multiple interim analyses.
- Controlled the familywise error rate.
Main Results:
- The gated group sequential design demonstrated higher power than traditional group sequential and adaptive designs.
- Simulations indicated reduced time and resource requirements.
- An illustrative example supported the design's effectiveness.
Conclusions:
- The gated group sequential design integrates benefits of group sequential and adaptive designs.
- This approach increases power and efficiency while controlling error rates.
- Potential to reduce drug development costs and accelerate meeting medical needs.
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