Innovative Study Designs and Approaches for Dose Optimization Across Multiple Indications

Music City Center 

The FDA's Project Optimus has redefined dose optimization by recommending randomized, parallel dose-response trials to compare multiple doses. Traditionally, dose optimization follows a two-stage design: starting with dose escalation to identify the maximum tolerated dose, followed by a randomization stage where patients are assigned to different doses to assess and compare their risk-benefit profiles. However, this method often requires a large sample size, posing a significant limitation.

In this presentation, I will introduce innovative seamless dose optimization designs that incorporate adaptive randomization. These designs aim to reduce sample sizes, shorten trial durations, and improve the accuracy of identifying optimal doses. Additionally, recognizing that different dosages may be required for various indications, I will explore the basket trial design, which allows for the simultaneous assessment of a new treatment across multiple cancer types.

Dose Optimization

Basket Trial Design