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 six 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 six healthy volunteers in the UK 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.
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 language | English |
|---|---|
| Pages (from-to) | 100-106 |
| Number of pages | 7 |
| Journal | Pharmaceutical Statistics |
| Volume | 16 |
| Issue number | 2 |
| Early online date | 16 Feb 2017 |
| DOIs | |
| Publication status | Published - 15 Mar 2017 |
Keywords
- BIAL 10-2474
- Protocol
- Design
- Statistical issues
- Combined approvals
Access to Document
- Bird_2017_Statistical_PharmaStat_AAM
Copyright © 2017 John Wiley & Sons, Ltd. This work has been made available online in accordance with the publisher’s policies. This is the author created, accepted version manuscript following peer review and may differ slightly from the final published version. The final published version of this work is available at: https://doi.org/10.1002/pst.1801