Adaptive Enrichment Designs for Confirmatory Randomized Trials: Methods and Software
- East Baltimore
- Summer Inst. term
- 1 credits
- Academic Year:
- 2019 - 2020
Some knowledge of clinical trials and statistics/biostatistics
Randomized trial designs that adaptively change enrollment criteria during a trial, called adaptive enrichment designs, have potential to provide improved information about which subpopulations benefit from new treatments.
Provides an overview of the strengths and limitations of randomized trial designs that adaptively change enrollment criteria during a trial (adaptive enrichment designs) and have the potential to provide improved information about which subpopulations benefit from new treatments. Explains recent advances in statistical methods for these designs, and presents adaptive design software planning tools. Discusses FDA guidance documents on adaptive designs. Examines methods for improving precision of estimators of the average treatment effect, by leveraging information in baseline variables; these methods can be used in adaptive designs as well as standard (non-adaptive) trial designs.
- Learning Objectives:
- Describe benefits and limitations of adaptive enrichment designs
- Explain new methods for improving precision in randomized trials by leveraging prognostic baseline variables
- Recognize benefits and limitations of software for planning adaptive designs
- Methods of Assessment:
In class participation -20%; in class exercises - 80%
- Instructor Consent:
No consent required
- Special Comments:
Pre-class readings will be provided to students, which will give a gentle introduction to adaptive designs. The readings will include discussion papers on the benefits and limitations of adaptive designs, and FDA draft guidances on adaptive designs; these readings are accessible to a wide audience and do not require previous knowledge of adaptive designs.