Cabrera Juan R, Taylor Jennie W, Molinaro Annette M
University of California San Francisco, Department of Neurological Surgery, 505 Parnassus Ave. Rm. M779, San Francisco, CA 94143 (J.R.C.; A.M.M.); University of California San Francisco, Department of Epidemiology and Biostatistics, 1450 3rd Street, San Francisco, CA 94158 (A.M.M.); University of California San Francisco, Department of Neurological Surgery, 400 Parnassus Ave. Rm. A808, San Francisco, CA 94143 (J.W.T.).
Neurooncol Pract. 2017 Mar;4(1):67-72. doi: 10.1093/nop/npw014. Epub 2016 Aug 26.
An efficient phase I trial is a crucial step in developing a new drug in a safe and timely manner. The main objective of a phase I trial is to determine the maximum tolerated dose in order to recommend the dose for a phase II trial. There are many designs that are implemented in phase I trials. Rule-based designs such as the traditional 3 + 3 method and rolling six design are easy to implement and assess for safety using a conservative approach. Model-based designs such as the continual reassessment method and the time-to-event continual reassessment method use mathematical models to increase the precision of dose estimation. The advantages and shortcomings of these designs, along with other designs, are reviewed.