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Adaptive Enrichment Designs in Clinical Trials
1Department of Biostatistics, M.D. Anderson Cancer Center, University of Texas, Houston, Texas 77030, USA.
Annual Review of Statistics and Its Application
|October 10, 2022
Summary
Adaptive enrichment designs in clinical trials use interim data to tailor treatments for specific patient subgroups, improving precision medicine and treatment efficacy. These designs optimize trial efficiency by identifying effective therapies for targeted populations.
Area of Science:
- Clinical Trials
- Biostatistics
- Genomic Medicine
Background:
- Adaptive enrichment designs offer flexibility in clinical trials by utilizing interim data.
- These designs are crucial for optimizing treatment strategies in the era of precision medicine.
- Identifying treatment-sensitive patient subgroups is key to maximizing therapeutic benefit.
Purpose of the Study:
- To provide a comprehensive overview of adaptive enrichment designs in clinical trials.
- To discuss the fundamental concepts and various types of enrichment strategies.
- To illustrate the application of these designs with numerical examples and case reviews.
Main Methods:
- Review of adaptive signature designs and Bayesian approaches for subgroup identification.
- Exploration of designs that enrich for superior treatments or make subgroup-specific decisions.
- Inclusion of outcome-adaptive randomization and sequential decision-making processes.
Main Results:
- Demonstration of how adaptive designs can identify treatment-biomarker interactions.
- Numerical illustrations for different scenarios involving patient heterogeneity and treatment effects.
- Overview of methods for optimizing sample size and treatment allocation within subgroups.
Conclusions:
- Adaptive enrichment designs are powerful tools for advancing precision medicine in clinical trials.
- These designs allow for dynamic adjustments to trial parameters based on accumulating data.
- The strategic use of enrichment designs enhances the efficiency and effectiveness of targeted therapies.
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