A Phase IIB clinical trial typically is a single-arm study aimed at deciding whether a new treatment E is sufficiently promising, relative to a standard therapy, S, to include in a large-scale randomized trial. Thus, Phase IIB trials are inherently comparative even though a standard therapy arm usually is not included. Uncertainty regarding the response rate Θs of S is rarely made explicit, either in planning the trial or interpreting its results. We propose practical Bayesian guidelines for deciding whether E is promising relative to S in settings where patient response is binary and the data are monitored continuously. The design requires specification of an informative prior for Θs, a targeted improvement for E, and bounds on the allowed sample size. No explicit specification of a loss function is required. Sampling continues until E is shown to be either promising or not promising relative to S with high posterior probability, or the maximum sample size is reached. The design provides decision boundaries, a probability distribution for the sample size at termination, and operating characteristics under fixed response probabilities with E.
Biometrics is a scientific journal emphasizing the role of statistics and mathematics in the biological sciences. Its object is to promote and extend the use of mathematical and statistical methods in pure and applied biological sciences by describing developments in these methods and their applications in a form readily assimilable by experimental scientists. JSTOR provides a digital archive of the print version of Biometrics. The electronic version of Biometrics is available at http://www.blackwell-synergy.com/servlet/useragent?func=showIssues&code;=biom. Authorized users may be able to access the full text articles at this site.
The International Biometric Society is an international society for the advancement of biological science through the development of quantitative theories and the application, development and dissemination of effective mathematical and statistical techniques. The Society welcomes as members biologists, mathematicians, statisticians, and others interested in applying similar techniques.
This item is part of JSTOR collection
For terms and use, please refer to our Terms and Conditions
Biometrics
© 1994 International Biometric Society
Request Permissions
