title: Making efficient use of patients in designing phase III trials investigating simultaneously a set of targeted therapies with different targets.
abstract: Targeted therapies are a recent development in cancer treatment research. As these therapies can only be administered to patients with certain individual characteristics, it is a straightforward idea to investigate several of such therapies simultaneously in a given patient population in order to compare each targeted therapy with the current standard therapy. This raises the question how patients satisfying several characteristics should be handled. We consider in this paper several designs to allocate treatments in a random manner to these patients, such that the evaluation of each single targeted therapy can be based on a simple comparison of patients receiving the targeted therapies versus those receiving the standard therapy within a well defined subgroup of patients satisfying the corresponding characteristic. We show how one can ensure that patients with several characteristics can contribute simultaneously to the evaluation of several targeted therapies and that this is the key point for an efficient use of the patients available. We further discuss some ethical and practical issues in applying the new designs and outline strategies to evaluate the overall effect of all targeted therapies together.
authors.affiliation: Department of Statistics, University of Southern Denmark. werner.v@stat.sdu.dk
authors.forename: Werner
authors.initials: W
authors.lastname: Vach
authors.forename: Rene
authors.initials: R
authors.lastname: dePont Christensen
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001699:Biometry / Q000379:methods*; D017326:Clinical Trials, Phase III as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D016503:Drug Delivery Systems / Q000379:methods* / Q000706:statistics & numerical data*; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy* / Q000453:epidemiology; D018579:Patient Selection; D012107:Research Design
chemical_list: D000970:Antineoplastic Agents