A flexible dose-response modeling framework based on continuous toxicity outcomes in phase I cancer clinical trials

Se Yoon Lee

Abstract: The past few decades have seen remarkable developments in dose-finding designs for phase I cancer clinical trials. While many of these designs rely on a binary toxicity response, there is an increasing focus on leveraging continuous toxicity responses. A continuous toxicity response pertains to a quantitative measure represented by real numbers. A higher value corresponds not only to an elevated likelihood of side effects for patients but also to an increased probability of treatment efficacy. This relationship between toxicity and dose is often nonlinear, necessitating flexibility in the quest to find an optimal dose.