An adaptive seamless phase II/III design for oncology trials with subpopulation selection using correlated survival endpoints

Martin Jenkins, Andrew Stone, Christopher Jennison

Research output: Contribution to journalArticle

99 Citations (Scopus)

Abstract

Although the statistical methods enabling efficient adaptive seamless designs are increasingly well established, it is important to continue to use the endpoints and specifications that best suit the therapy area and stage of development concerned when conducting such a trial. Approaches exist that allow adaptive designs to continue seamlessly either in a subpopulation of patients or in the whole population on the basis of data obtained from the first stage of a phase II/III design: our proposed design adds extra flexibility by also allowing the trial to continue in all patients but with both the subgroup and the full population as co-primary populations. Further, methodology is presented which controls the Type-I error rate at less than 2.5% when the phase II and III endpoints are different but correlated time-to-event endpoints. The operating characteristics of the design are described along with a discussion of the practical aspects in an oncology setting.
Original languageEnglish
Pages (from-to)347-356
Number of pages10
JournalPharmaceutical Statistics
Volume10
Issue number4
Early online date8 Dec 2010
DOIs
Publication statusPublished - 1 Jul 2011

Keywords

  • targeted therapy
  • subpopulation selection
  • oncology
  • time-to-event endpoints
  • phase II/III
  • seamless design

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