title: Statistical monitoring of clinical trials with multivariate response and/or multiple arms: a flexible approach.
abstract: Randomized clinical trials with a multivariate response and/or multiple treatment arms are increasingly common, in part because of their efficiency and a greater concern about balancing risks with benefits. In some trials, the specific types and magnitudes of treatment group differences that would warrant early termination cannot easily be specified prior to the onset of the trial and/or could change as the trial progresses. This underscores the need for more flexible monitoring methods than traditional approaches. This paper extends the repeated confidence bands approach for interim monitoring to more general settings where there can be a multivariate response and/or multiple treatment arms and where the metrics for comparing treatment groups can change during the conduct of the trial. We illustrate the approach using the results of a recent AIDS clinical trial and examine its efficiency and robustness via simulation.
authors.affiliation: Department of Statistics and Actuarial Science, Simon Fraser University, Burnaby, BC V5A 1S6, Canada.
authors.forename: Lihui
authors.initials: L
authors.lastname: Zhao
authors.forename: X Joan
authors.initials: XJ
authors.lastname: Hu
authors.forename: Stephen W
authors.initials: SW
authors.lastname: Lagakos
mesh_terms: D019380:Anti-HIV Agents / Q000627:therapeutic use; D018791:CD4 Lymphocyte Count; D003198:Computer Simulation; D016001:Confidence Intervals; D003627:Data Interpretation, Statistical; D006678:HIV / Q000235:genetics / Q000254:growth & development; D015658:HIV Infections / Q000097:blood / Q000188:drug therapy / Q000821:virology; D006801:Humans; D015999:Multivariate Analysis; D012367:RNA, Viral / Q000097:blood; D016032:Randomized Controlled Trials as Topic / Q000379:methods*
chemical_list: D019380:Anti-HIV Agents; D012367:RNA, Viral