Modern Approaches to Clinical Trials Using SAS: Classical, Adaptive, and Bayesian Methods

5.1 Dose Finding in Oncology

In cancer drug development, phase I clinical trials are mainly designed to determine the maximum tolerated dose (MTD), which is typically defined as the maximum dosage that can be administered to patients without exceeding the unacceptable level of toxicity. In practice, the investigators specify a target toxicity rate so that the toxicity probability of the identified MTD should be closest to the target among all the doses under investigation. Phase I trials, often known as dose-escalation studies, involve only a single arm. The collected data are typically binary, indicating whether patients have experienced the dose-limiting toxicity (DLT). The DLTs are predefined drug-related toxicities that would prohibit continuing ...