4 papers
A Utility Score Framework for Dose Optimization Studies with Binary Efficacy-Safety Endpoints: Sample Size Determination and Bias Characterization
Xuemin Gu, Cong Xu, Lei Xu +1
The FDA's Project Optimus initiative emphasizes patient-centered dose selection in oncology that balances efficacy and safety. We develop a framework for randomized dose optimizati…
ROSE: Randomized Optimal Selection Design for Dose Optimization
Shuqi Wang, Ying Yuan, Suyu Liu
The U.S. Food and Drug Administration (FDA) launched Project Optimus to shift the objective of dose selection from the maximum tolerated dose to the optimal biological dose (OBD),…
BARD: A seamless two-stage dose optimization design integrating backfill and adaptive randomization
Yixuan Zhao, Rachael Liu, Jianchang Lin +1
One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose (MTD), followed by a randomization…
BOP2-TE: Bayesian Optimal Phase 2 Design for Jointly Monitoring Efficacy and Toxicity with Application to Dose Optimization
Kai Chen, Heng Zhou, J. Jack Lee +1
We propose a Bayesian optimal phase 2 design for jointly monitoring efficacy and toxicity, referred to as BOP2-TE, to improve the operating characteristics of the BOP2 design propo…