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Comparing Bayesian early stopping boundaries for phase II clinical trials
Liyun Jiang1,2, Fangrong Yan1, Peter F Thall2
1Research Center of Biostatistics and Computational Pharmacy, China Pharmaceutical University, Nanjing, China.
Pharmaceutical Statistics
|July 29, 2020
Summary
Designing phase II clinical trials requires balancing early stopping rules with a high true positive probability (TPP). An exponential boundary shape effectively maintains TPP while minimizing patient exposure to ineffective or unsafe treatments.
Area of Science:
- Clinical Trials Methodology
- Biostatistics
- Drug Development
Background:
- Phase II clinical trials are crucial for evaluating treatment efficacy and safety.
- Designing effective interim monitoring rules is essential to balance early stopping for futility/safety with maintaining a high true positive probability (TPP).
Purpose of the Study:
- To define and compare methods for specifying early stopping boundaries in phase II trials.
- To optimize trial designs by balancing TPP and the mean number of enrolled patients.
Main Methods:
- Utilized Bayesian posterior probabilities as decision criteria for early stopping.
- Compared constant, linear, and exponential boundary shapes as functions of interim sample size.
- Employed simulations to evaluate operating characteristics under various scenarios.
Main Results:
- No single boundary shape demonstrated universal optimality.
- Exponential boundary shapes, when calibrated, maintained high TPP.
- Exponential boundaries effectively limited patient enrollment in trials with inferior treatments or excessive toxicity.
Conclusions:
- Appropriately calibrated exponential early stopping boundaries offer an effective strategy for phase II trial design.
- This approach enhances the balance between reliable early termination and preserving the probability of detecting effective treatments.
- Optimized trial designs can reduce patient exposure to suboptimal therapies.
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