title: A Bayesian-frequentist two-stage single-arm phase II clinical trial design.
abstract: It is well-known that both frequentist and Bayesian clinical trial designs have their own advantages and disadvantages. To have better properties inherited from these two types of designs, we developed a Bayesian-frequentist two-stage single-arm phase II clinical trial design. This design allows both early acceptance and rejection of the null hypothesis ( H(0) ). The measures (for example probability of trial early termination, expected sample size, etc.) of the design properties under both frequentist and Bayesian settings are derived. Moreover, under the Bayesian setting, the upper and lower boundaries are determined with predictive probability of trial success outcome. Given a beta prior and a sample size for stage I, based on the marginal distribution of the responses at stage I, we derived Bayesian Type I and Type II error rates. By controlling both frequentist and Bayesian error rates, the Bayesian-frequentist two-stage design has special features compared with other two-stage designs.
authors.affiliation: Integrated Information Sciences (IIS) - Integrated Hospital Care (IHC), Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
authors.forename: Gaohong
authors.initials: G
authors.lastname: Dong
authors.forename: Weichung Joe
authors.initials: WJ
authors.lastname: Shih
authors.forename: Dirk
authors.initials: D
authors.lastname: Moore
authors.forename: Hui
authors.initials: H
authors.lastname: Quan
authors.forename: Stephen
authors.initials: S
authors.lastname: Marcella
mesh_terms: D001499:Bayes Theorem; D015982:Bias; D016010:Binomial Distribution; D017322:Clinical Trials, Phase II as Topic / Q000592:standards / Q000706:statistics & numerical data*; D003627:Data Interpretation, Statistical; D004305:Dose-Response Relationship, Drug; D023381:Endpoint Determination; D006801:Humans; D015233:Models, Statistical; D012107:Research Design; D018401:Sample Size