Biomarker-Driven Oncology Clinical Trials: Key Design Elements, Types, Features, and Practical Considerations
1Johns Hopkins University, Baltimore, MD.
JCO Precision Oncology
|September 14, 2020
Summary
Biomarker-driven clinical trials are evolving, with new master protocols and platform trials offering advantages but also unique challenges. This overview guides the selection and adaptation of trial designs for personalized medicine.
Area of Science:
- Oncology
- Clinical Trial Design
- Biomarker Development
Background:
- Precision oncology relies on molecular profiling for personalized disease management.
- Biomarker-driven trials require careful design considerations based on biomarker type and utility.
- Master protocols and platform trials represent a paradigm shift in biomarker-driven research.
Purpose of the Study:
- To provide an overview of design options for biomarker-driven clinical trials.
- To discuss considerations for single-biomarker/single-disease and multi-biomarker/multi-disease settings.
- To explain the rationale and practical aspects of adaptive trial designs.
Main Methods:
- Review of design options for biomarker-driven clinical trials.
- Explanation of adaptive design components for flexibility and efficiency.
- Inclusion of lessons learned from real-world clinical trials.
Main Results:
- Overview of design choices for single- and multi-biomarker/disease trials.
- Guidance on incorporating adaptive elements into trial designs.
- Practical considerations for operationalizing complex trial structures.
Conclusions:
- Adaptive designs enhance flexibility, efficiency, and resource allocation in biomarker-driven trials.
- Understanding design nuances is crucial for optimizing personalized medicine strategies.
- Platform trials offer logistical benefits but require careful operational planning.
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