title: Nine-year change in statistical design, profile, and success rates of Phase II oncology trials.
abstract: We investigated nine-year trends in statistical design and other features of Phase II oncology clinical trials published in 2005, 2010, and 2014 in five leading oncology journals: Cancer, Clinical Cancer Research, Journal of Clinical Oncology, Annals of Oncology, and Lancet Oncology. The features analyzed included cancer type, multicenter vs. single-institution, statistical design, primary endpoint, number of treatment arms, number of patients per treatment arm, whether or not statistical methods were well described, whether the drug was found effective based on rigorous statistical testing of the null hypothesis, and whether the drug was recommended for future studies.
authors.affiliation: a Department of Biostatistics , The University of North Carolina at Chapel Hill , Chapel Hill , North Carolina , USA.
authors.forename: Anastasia
authors.initials: A
authors.lastname: Ivanova
authors.affiliation: b Department of Medicine , The University of North Carolina at Chapel Hill , Chapel Hill , North Carolina , USA.
authors.forename: Barry
authors.initials: B
authors.lastname: Paul
authors.affiliation: c Quintiles , Morrisville , North Carolina , USA.
authors.forename: Olga
authors.initials: O
authors.lastname: Marchenko
authors.affiliation: c Quintiles , Morrisville , North Carolina , USA.
authors.forename: Guochen
authors.initials: G
authors.lastname: Song
authors.affiliation: d Department of Health Policy and Management , The University of North Carolina at Chapel Hill , Chapel Hill , North Carolina , USA.
authors.forename: Neerali
authors.initials: N
authors.lastname: Patel
authors.affiliation: b Department of Medicine , The University of North Carolina at Chapel Hill , Chapel Hill , North Carolina , USA.
authors.forename: Stergios J
authors.initials: SJ
authors.lastname: Moschos
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods* / Q000706:statistics & numerical data*; D003627:Data Interpretation, Statistical; D006801:Humans; D008495:Medical Oncology / Q000706:statistics & numerical data*; D009369:Neoplasms / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents
keywords: Fleming’s design; Phase II trials; Simon’s design; oncology; two-stage design