Critical aspects of the Bayesian approach to phase I cancer trials

• Published in: Statistics in medicine Vol. 27; no. 13; pp. 2420 - 2439
• Main Authors: Neuenschwander, Beat, Branson, Michael, Gsponer, Thomas
• Format: Journal Article
• Language: English
• Published: Chichester, UK John Wiley & Sons, Ltd 15-06-2008; Wiley Subscription Services, Inc
• Subjects: Antineoplastic Agents - administration & dosage; Antineoplastic Agents - adverse effects; Bayes Theorem; Bayesian analysis; Cancer; Clinical trials; Clinical Trials, Phase I as Topic - methods; Computer Simulation; continual reassessment method; Drug dosages; Humans; logistic model; maximum-tolerated dose; Medical statistics; Methods; Models, Statistical; Neoplasms - drug therapy
• ISSN: 0277-6715; 1097-0258
• DOI: 10.1002/sim.3230

The Bayesian approach to finding the maximum‐tolerated dose in phase I cancer trials is discussed. The suggested approach relies on a realistic dose–toxicity model, allows one to include prior information, and supports clinical decision making by presenting within‐trial information in a transparent way. The modeling and decision‐making components are flexible enough to be extendable to more complex settings. Critical aspects are emphasized and a comparison with the continual reassessment method (CRM) is performed with data from an actual trial and a simulation study. The comparison revealed similar operating characteristics while avoiding some of the difficulties encountered in the actual trial when applying the CRM. Copyright © 2008 John Wiley & Sons, Ltd.