title: Issues in the design of clinical trials for safety in HIV-infected children.
abstract: The use of newly approved drugs for human immunodeficiency virus (HIV)-infected adults is limited in children until child-specific pharmacokinetic and safety data can be submitted to the Food and Drug Administration (FDA) to fulfill labeling requirements. Design of a clinical trial to assess drug safety in children requires a different set of issues from standard Phase II and III efficacy trials in adults. For example, Phase II trials in adults are done to collect preliminary efficacy information. In children, efficacy of the drug is assumed and only safety needs to be shown. As a result, control groups may not be required and it may not be necessary to show superiority of the new drug over a control. This paper describes these issues in some detail and gives examples of designs appropriate in different scenarios.
authors.affiliation: Center for Biostatistics in AIDS Research, Harvard School of Public Health, Boston, Massachusetts 02115, USA.
authors.forename: J C
authors.initials: JC
authors.lastname: Lindsey
authors.forename: M W
authors.initials: MW
authors.lastname: Kline
mesh_terms: D000328:Adult; D002648:Child; D002651:Child Advocacy; D002986:Clinical Trials as Topic; D016001:Confidence Intervals; D004341:Drug Evaluation; D016903:Drug Monitoring; D015658:HIV Infections / Q000188:drug therapy*; D006801:Humans; D012107:Research Design; D012449:Safety; D014481:United States; D014486:United States Food and Drug Administration