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

doi:10.1002/pst.1801

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

Research output: Contribution to journal › Article › peer-review

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.

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	https://doi.org/10.1002/pst.1801
Publication status	Published - 15 Mar 2017

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

BIAL 10-2474
Protocol
Design
Statistical issues
Combined approvals

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10.1002/pst.1801

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
Accepted author manuscript, 159 KB

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AU - Grieve, Andrew P.

AU - Senn, Stephen

N1 - Funding information: European Union's FP7 programme, Grant/Award Number: 602552

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KW - Protocol

KW - Design

KW - Statistical issues

KW - Combined approvals

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ER -

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

Abstract

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Keywords

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