Early-Phase Oncology Trials: Why So Many Designs?

Area of Science:

• Biostatistics
• Clinical Trial Design
• Oncology Research

Background:

• Significant statistical efforts over 30 years aimed to enhance early-phase oncology trial design and accuracy.
• Clinicians exhibit reluctance towards adopting efficient model-based approaches due to complexity and understanding barriers.
• The standard 3+3 design is widely used, but modern alternatives present challenges in selection and implementation.

Purpose of the Study:

• To review and categorize 15 proposed and currently used early-phase oncology trial designs.
• To clarify the properties and differences among various model-based designs.
• To guide clinicians in selecting the most appropriate design for their specific research needs.

Main Methods:

• A comprehensive review of 15 distinct early-phase oncology trial designs.
• Analysis of operational similarities and differences among the reviewed designs.
• Classification of the 15 designs into broader, functionally equivalent categories.

Main Results:

• Many of the 15 reviewed designs are operationally identical.
• The 15 designs can be consolidated into three main classes based on their core principles.
• Certain designs necessitate ad hoc modifications to achieve satisfactory performance.

Conclusions:

• Consolidating 15 designs into three classes simplifies the selection process for clinicians.
• Understanding the fundamental properties of these design classes is crucial for optimal trial implementation.
• Further refinement of specific designs may be required to ensure robust performance in oncology trials.