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An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment

Analytical similarity assessment of critical quality attributes (CQAs) serves as a foundation for the development of biosimilar products and facilitates an abbreviated subsequent clinical evaluation. In this study, we establish a statistical evaluation roadmap with statistical approaches for some se...

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Detalles Bibliográficos
Autores principales: Wu, Kejian, Pan, Haitao, Li, Chen, Zhao, Qingbo, Wang, Ling, Xia, Jielai
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6283468/
https://www.ncbi.nlm.nih.gov/pubmed/30521581
http://dx.doi.org/10.1371/journal.pone.0208354
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author Wu, Kejian
Pan, Haitao
Li, Chen
Zhao, Qingbo
Wang, Ling
Xia, Jielai
author_facet Wu, Kejian
Pan, Haitao
Li, Chen
Zhao, Qingbo
Wang, Ling
Xia, Jielai
author_sort Wu, Kejian
collection PubMed
description Analytical similarity assessment of critical quality attributes (CQAs) serves as a foundation for the development of biosimilar products and facilitates an abbreviated subsequent clinical evaluation. In this study, we establish a statistical evaluation roadmap with statistical approaches for some selected CQAs from Tier 1, because they are most relevant to clinical outcomes and require the most rigorous statistical methods. In the roadmap, we incorporate 3 methods—ranking and tier assignment of quality attributes, the equivalence test, and the Mann–Whitney test for equivalence—that are important to determine analytical similarity between the reference and biosimilar products. For the equivalence test, we develop a power calculation formula based on the two one-sided tests procedure. Exact sample sizes can be numerically calculated. Then, we propose a flexible idea for selecting the number of reference lots (n(R)) and the number of biosimilar lots (n(T)) to adjust for serious unbalanced sample sizes. From results of extensive simulations under various parameter settings, we obtain a workable strategy to determine the optimum sample size combination (n(T), n(R)) for the equivalence test of CQAs from Tier 1. R codes are provided to facilitate implementation of the roadmap and corresponding methods in practice.
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spelling pubmed-62834682018-12-20 An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment Wu, Kejian Pan, Haitao Li, Chen Zhao, Qingbo Wang, Ling Xia, Jielai PLoS One Research Article Analytical similarity assessment of critical quality attributes (CQAs) serves as a foundation for the development of biosimilar products and facilitates an abbreviated subsequent clinical evaluation. In this study, we establish a statistical evaluation roadmap with statistical approaches for some selected CQAs from Tier 1, because they are most relevant to clinical outcomes and require the most rigorous statistical methods. In the roadmap, we incorporate 3 methods—ranking and tier assignment of quality attributes, the equivalence test, and the Mann–Whitney test for equivalence—that are important to determine analytical similarity between the reference and biosimilar products. For the equivalence test, we develop a power calculation formula based on the two one-sided tests procedure. Exact sample sizes can be numerically calculated. Then, we propose a flexible idea for selecting the number of reference lots (n(R)) and the number of biosimilar lots (n(T)) to adjust for serious unbalanced sample sizes. From results of extensive simulations under various parameter settings, we obtain a workable strategy to determine the optimum sample size combination (n(T), n(R)) for the equivalence test of CQAs from Tier 1. R codes are provided to facilitate implementation of the roadmap and corresponding methods in practice. Public Library of Science 2018-12-06 /pmc/articles/PMC6283468/ /pubmed/30521581 http://dx.doi.org/10.1371/journal.pone.0208354 Text en © 2018 Wu et al http://creativecommons.org/licenses/by/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Research Article
Wu, Kejian
Pan, Haitao
Li, Chen
Zhao, Qingbo
Wang, Ling
Xia, Jielai
An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title_full An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title_fullStr An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title_full_unstemmed An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title_short An evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
title_sort evaluation roadmap for critical quality attributes from tier 1 in analytical similarity assessment
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6283468/
https://www.ncbi.nlm.nih.gov/pubmed/30521581
http://dx.doi.org/10.1371/journal.pone.0208354
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