Statistical issues in first-in-human studies on BIA 10-2474 : neglected comparison of protocol against practice
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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.
Bird , S M , Bailey , R A , Grieve , A P & Senn , S 2017 , ' Statistical issues in first-in-human studies on BIA 10-2474 : neglected comparison of protocol against practice ' Pharmaceutical Statistics , vol 16 , no. 2 , pp. 100-106 . DOI: 10.1002/pst.1801
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
Funding information: European Union's FP7 programme, Grant/Award Number: 602552
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