Sample size and screening size trade-off in the presence of subgroups with different expected treatment effects

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Abstract

When designing clinical trials, sometimes we may expect a larger treatment effect in one group while others exhibit an attenuated effect. In these settings there can be a trade-off between a smaller average treatment effect with broader enrollment criteria and a larger effect with restricted criteria but longer enrollment duration. Identification of subgroups will often use a clinical decision rule, for example, biomarker cutoff, but may be imprecise, that is, with sensitivity and specificity not simultaneously 100%. We evaluate the impact of including attenuated subgroups on design operating characteristics and illustrate scenarios where overall trial duration may be shorter by not being restrictive.

Original languageEnglish (US)
Pages (from-to)344-358
Number of pages15
JournalJournal of Biopharmaceutical Statistics
Volume24
Issue number2
DOIs
StatePublished - Mar 4 2014

Bibliographical note

Funding Information:
This work was supported in part by grant 1UL1RR033183 from the National Center for Research Resources and by grant 8 UL1 TR000114-02 from the National Center for Advancing Translational Sciences of the National Institutes of Health.

Keywords

  • Biomarker
  • Clinical trial design
  • Enrichment
  • Heterogeneous effects
  • Operating characteristics
  • Sample size

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