A comparison of two phase I trial designs

TLDR

Phase I cancer chemotherapy trials aim to quickly identify the maximum tolerated dose of a new agent, and a recently proposed Bayesian continual reassessment method has been suggested to improve upon the standard design. The study proposes improvements to both the standard design and the Bayesian continual reassessment method. The authors suggest modifications to the standard design and the Bayesian method to enhance trial efficiency. The authors found that prior comparisons were incomplete, and that using the continual reassessment method results in more patients receiving very high doses and longer trial durations.

Abstract

Abstract Phase I cancer chemotherapy trials are designed to determine rapidly the maximum tolerated dose of a new agent for further study. A recently proposed Bayesian method, the continual reassessment method, has been suggested to offer an improvement over the standard design of such trials. We find the previous comparisons did not completely address the relative performance of the designs as they would be used in practice. Our results indicate that with the continual reassessment method, more patients will be treated at very high doses and the trials will take longer to complete. We offer some suggested improvements to both the standard design and the Bayesian method.