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An optimal design for screening trials
1CSIRO Mathematical and Information Sciences, Cleveland, Queensland, Australia. You-Gan.Wang@marine.csiro.au
Abstract:
Yao, Begg, and Livingston (1996, Biometrics 52, 992-1001) considered the optimal group size for testing a series of potentially therapeutic agents to identify a promising one as soon as possible for given error rates. The number of patients to be tested with each agent was fixed as the group size. We consider a sequential design that allows early acceptance and rejection, and we provide an optimal strategy to minimize the sample sizes (patients) required using Markov decision processes. The minimization is under the constraints of the two types (false positive and false negative) of error probabilities, with the Lagrangian multipliers corresponding to the cost parameters for the two types of errors. Numerical studies indicate that there can be a substantial reduction in the number of patients required.
Insights
This study introduces a sequential design for drug testing, optimizing patient sample sizes. The new method significantly reduces the number of patients needed to identify effective therapeutic agents while controlling error rates.
Area of Science:
- Biostatistics
- Clinical Trial Design
- Pharmacological Research
Background:
- Previous research (Yao, Begg, & Livingston, 1996) focused on fixed group sizes for testing therapeutic agents.
- Identifying promising agents efficiently requires minimizing patient sample sizes.
Purpose of the Study:
- To develop an optimal sequential design for testing multiple therapeutic agents.
- To minimize the total number of patients (sample size) required in clinical trials.
- To establish a strategy that allows for early acceptance or rejection of agents.
Main Methods:
- Utilized Markov decision processes to formulate an optimal strategy.
- Minimized sample size under constraints of false positive and false negative error probabilities.
- Employed Lagrangian multipliers to represent the costs associated with different error types.
Main Results:
- Developed a sequential design enabling early decisions on therapeutic agents.
- Demonstrated substantial reductions in required patient sample sizes through numerical studies.
- The optimal strategy effectively balances error rates and sample size efficiency.
Conclusions:
- Sequential designs offer significant advantages over fixed group sizes in therapeutic agent testing.
- The proposed Markov decision process approach provides an efficient method for sample size minimization.
- This strategy can lead to faster identification of effective treatments with fewer patients.