Statistical issues in first-in-human studies on BIA 10-2474: Neglected comparison of protocol against practice

Sheila M. Bird*, Rosemary A. Bailey, Andrew P. Grieve, Stephen Senn

*Corresponding author for this work

Research output: Contribution to journalComment/debate

6 Citations (Scopus)

Abstract

By setting the regulatory-approved protocol for a suite of first-in-human studies on BIA 10-2474 against the subsequent French investigations, we highlight 6 key design and statistical issues, which reinforce recommendations by a Royal Statistical Society Working Party, which were made in the aftermath of cytokine release storm in 6 healthy volunteers in the United Kingdom in 2006. The 6 issues are dose determination, availability of pharmacokinetic results, dosing interval, stopping rules, appraisal by safety committee, and clear algorithm required if combining approvals for single and multiple ascending dose studies.

Original languageEnglish
Pages (from-to)100-106
Number of pages7
JournalPharmaceutical Statistics
Volume16
Issue number2
DOIs
Publication statusPublished - 1 Mar 2017

Keywords

  • combined approvals
  • first-in-human
  • healthy volunteer
  • protocol
  • recommendations
  • study design

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