title: A comparison of methods for phase II cancer clinical trials: advantages of the triangular test, a group sequential method.
abstract: In cancer, the purpose of Phase II studies is to determine whether the response rate p to a new treatment is greater than a prespecified value p0, defined as the largest response rate for which Phase III studies are not worthwhile. It concerns, for example, determining whether the response rate to a new drug is greater than 20%. The main problem is of a decision making nature, and amounts to the comparison of an observed percentage with a theoretical percentage. One way of resolving it is to perform a single statistical analysis after the inclusion of a predetermined number of patients N, but it is not always possible because of high values of N. Furthermore, this approach presents ethical problems when elements in favor of inefficacy or efficacy are available early in the trial. For these reasons, several authors have developed methods which allow to perform repeated analyses and possibly to reach an early conclusion of the study: two-stage, multistage, sequential and group sequential methods. This article considers the main decision making methods proposed in the literature for Phase II studies in oncology. The bibliographic study, which highlights the interest of using group sequential methods, and especially the Triangular Test, is confirmed by a comparative study of the statistical properties of the different methods.
authors.affiliation: Département de Biostatistique et Informatique Médicale, Hôpital Saint-Louis, Paris, France.
authors.forename: E
authors.initials: E
authors.lastname: Bellissant
authors.forename: J
authors.initials: J
authors.lastname: Benichou
authors.forename: C
authors.initials: C
authors.lastname: Chastang
mesh_terms: D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003657:Decision Making; D003661:Decision Support Techniques; D004992:Ethics, Medical; D006801:Humans; D009369:Neoplasms / Q000628:therapy; D011336:Probability; D012107:Research Design; D016896:Treatment Outcome