title: Practical Bayesian guidelines for phase IIB clinical trials.
abstract: 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 theta s 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 theta s, 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.
authors.affiliation: Department of Biomathematics, Anderson Cancer Center, University of Texas, Houston 77030.
authors.forename: P F
authors.initials: PF
authors.lastname: Thall
authors.forename: R
authors.initials: R
authors.lastname: Simon
mesh_terms: D001499:Bayes Theorem; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D006801:Humans; D011336:Probability; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D013223:Statistics as Topic