Related Experiment Videos
Optimal two-stage design for a series of pilot trials of new agents
1Department of Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, New York 10021, USA. yao@biost.mskcc.org
Abstract:
An approach to determine the appropriate sample sizes for a series of screening trials to identify promising new therapeutic agents was presented by Yao, Begg, and Livingston (1996, Biometrics 52, 992-1001). This approach is now improved to a two-stage design that further minimizes the time to identify a promising agent under fixed error rates. When applied to data from the historical experience of exploratory vaccination trials at Memorial Sloan-Kettering Cancer Center, the method demonstrates that relatively small individual screening trials are optimal. The reliability of the results is evaluated using the bootstrap.
Insights
This study refines sample size calculations for therapeutic agent screening trials using a two-stage design. It confirms that smaller screening trials are optimal for efficiently identifying promising new treatments.
Area of Science:
- Biostatistics
- Clinical Trial Design
- Pharmacological Research
Background:
- Traditional screening trials face challenges in optimizing sample size and time for identifying therapeutic agents.
- Previous work by Yao, Begg, and Livingston (1996) established an approach for sample size determination in screening trials.
Purpose of the Study:
- To improve upon existing methods for determining sample sizes in therapeutic agent screening.
- To introduce a two-stage design that minimizes the time to identify promising agents while maintaining fixed error rates.
Main Methods:
- Development of a two-stage adaptive design for screening trials.
- Application of the improved methodology to historical data from exploratory vaccination trials.
- Utilizing bootstrap methods to evaluate the reliability of the findings.
Main Results:
- The two-stage design effectively minimizes the time required to identify promising therapeutic agents.
- Analysis of historical data indicates that smaller individual screening trials are optimal.
- Bootstrap evaluation confirmed the robustness and reliability of the proposed method.
Conclusions:
- The enhanced two-stage design offers a more efficient approach to therapeutic agent screening.
- Optimizing sample size through smaller, sequential trials is recommended for improved efficiency.
- This methodology provides a reliable framework for future drug discovery and development efforts.