Decision-making in a phase II clinical trial: a new approach combining Bayesian and frequentist concepts

Stallard, N., Whitehead, J. and Cleall, S. (2005) Decision-making in a phase II clinical trial: a new approach combining Bayesian and frequentist concepts. Pharmaceutical Statistics, 4 (2). pp. 119-128. ISSN 1539-1612 doi: 10.1002/pst.164

Abstract/Summary

The aim of a phase H clinical trial is to decide whether or not to develop an experimental therapy further through phase III clinical evaluation. In this paper, we present a Bayesian approach to the phase H trial, although we assume that subsequent phase III clinical trials will hat,e standard frequentist analyses. The decision whether to conduct the phase III trial is based on the posterior predictive probability of a significant result being obtained. This fusion of Bayesian and frequentist techniques accepts the current paradigm for expressing objective evidence of therapeutic value, while optimizing the form of the phase II investigation that leads to it. By using prior information, we can assess whether a phase II study is needed at all, and how much or what sort of evidence is required. The proposed approach is illustrated by the design of a phase II clinical trial of a multi-drug resistance modulator used in combination with standard chemotherapy in the treatment of metastatic breast cancer. Copyright (c) 2005 John Wiley & Sons, Ltd.