title: Designing a series of decision-theoretic phase II trials in a small population.
abstract: This paper introduces a decision-theoretic design for a series of phase II trials. Instead of designing phase II trials individually, we proposed a development plan that consists of a series of phase II trials and one phase III trial such that the long-term expected utility on the whole is optimized. The phase II trials are conducted sequentially, and patients are recruited sequentially to each phase II trial. At each interim stage, a decision is made to continue recruiting patients to the current trial, to stop and recommend the treatment proceeds to a phase III trial, to stop and initiate a new phase II trial or to stop and cease the development plan. The methodology uses a hybrid approach in which it is assumed that the data from the final phase III trial will be analysed using a classical frequentist hypothesis test. The expected power of this test based on some specified prior distribution for the effect of the experimental treatment is then used in a utility function, which is used to obtain the optimal design for the whole series of trials.
authors.affiliation: Warwick Medical School, The University of Warwick, Coventry, UK. s.w.hee@warwick.ac.uk
authors.forename: Siew Wan
authors.initials: SW
authors.lastname: Hee
authors.forename: Nigel
authors.initials: N
authors.lastname: Stallard
mesh_terms: D001499:Bayes Theorem; D017322:Clinical Trials, Phase II as Topic / Q000379:methods* / Q000706:statistics & numerical data; D017326:Clinical Trials, Phase III as Topic / Q000379:methods* / Q000706:statistics & numerical data; D003662:Decision Theory; D015340:Epidemiologic Research Design; D006801:Humans; D018579:Patient Selection; D018401:Sample Size