Till Briskot, Dominik Hiltmann, Federico Rischawy, Joey Studts, Simon Kluters
{"title":"生物制药工艺开发中机制模型的鉴定。","authors":"Till Briskot, Dominik Hiltmann, Federico Rischawy, Joey Studts, Simon Kluters","doi":"10.1016/j.xphs.2024.11.021","DOIUrl":null,"url":null,"abstract":"<div><div>Mechanistic process models play an increasingly important role in biopharmaceutical process development and manufacturing in supporting process design, characterization, and informing process control strategies. Despite the potential of mechanistic models, there is currently no clear consensus or regulatory guideline on their qualification, i.e. the processes of determining whether a model is suitable to support decision making in process development. In this work, a systematic and risk-based qualification framework for mechanistic models in biopharmaceutical process development is introduced. The framework integrates key concepts from other modeling frameworks and guidelines such as the ASME V&V 40 published by the American Society of Mechanical Engineers (ASME) and preliminary considerations in process models published by Quality Innovation Group (QIG) of the European Medicines Agency (EMA). Key concepts of the proposed framework are discussed using two case studies, including a model-informed optimization of a biopharmaceutical ultrafiltration and diafiltration process and a model-informed control strategy of a chromatography polishing step. The suggested framework can act as a foundation for dialogue and guide for other modelers in biopharmaceutical process development. It holds the capability to harmonize modeling procedures throughout the industry and establish an agreement on the qualification of mechanistic models in biopharmaceutical process development.</div></div>","PeriodicalId":16741,"journal":{"name":"Journal of pharmaceutical sciences","volume":"114 2","pages":"Pages 1095-1107"},"PeriodicalIF":3.7000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Qualification of mechanistic models in biopharmaceutical process development\",\"authors\":\"Till Briskot, Dominik Hiltmann, Federico Rischawy, Joey Studts, Simon Kluters\",\"doi\":\"10.1016/j.xphs.2024.11.021\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Mechanistic process models play an increasingly important role in biopharmaceutical process development and manufacturing in supporting process design, characterization, and informing process control strategies. Despite the potential of mechanistic models, there is currently no clear consensus or regulatory guideline on their qualification, i.e. the processes of determining whether a model is suitable to support decision making in process development. In this work, a systematic and risk-based qualification framework for mechanistic models in biopharmaceutical process development is introduced. The framework integrates key concepts from other modeling frameworks and guidelines such as the ASME V&V 40 published by the American Society of Mechanical Engineers (ASME) and preliminary considerations in process models published by Quality Innovation Group (QIG) of the European Medicines Agency (EMA). Key concepts of the proposed framework are discussed using two case studies, including a model-informed optimization of a biopharmaceutical ultrafiltration and diafiltration process and a model-informed control strategy of a chromatography polishing step. The suggested framework can act as a foundation for dialogue and guide for other modelers in biopharmaceutical process development. It holds the capability to harmonize modeling procedures throughout the industry and establish an agreement on the qualification of mechanistic models in biopharmaceutical process development.</div></div>\",\"PeriodicalId\":16741,\"journal\":{\"name\":\"Journal of pharmaceutical sciences\",\"volume\":\"114 2\",\"pages\":\"Pages 1095-1107\"},\"PeriodicalIF\":3.7000,\"publicationDate\":\"2025-02-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of pharmaceutical sciences\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0022354924005458\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CHEMISTRY, MEDICINAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of pharmaceutical sciences","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0022354924005458","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CHEMISTRY, MEDICINAL","Score":null,"Total":0}
Qualification of mechanistic models in biopharmaceutical process development
Mechanistic process models play an increasingly important role in biopharmaceutical process development and manufacturing in supporting process design, characterization, and informing process control strategies. Despite the potential of mechanistic models, there is currently no clear consensus or regulatory guideline on their qualification, i.e. the processes of determining whether a model is suitable to support decision making in process development. In this work, a systematic and risk-based qualification framework for mechanistic models in biopharmaceutical process development is introduced. The framework integrates key concepts from other modeling frameworks and guidelines such as the ASME V&V 40 published by the American Society of Mechanical Engineers (ASME) and preliminary considerations in process models published by Quality Innovation Group (QIG) of the European Medicines Agency (EMA). Key concepts of the proposed framework are discussed using two case studies, including a model-informed optimization of a biopharmaceutical ultrafiltration and diafiltration process and a model-informed control strategy of a chromatography polishing step. The suggested framework can act as a foundation for dialogue and guide for other modelers in biopharmaceutical process development. It holds the capability to harmonize modeling procedures throughout the industry and establish an agreement on the qualification of mechanistic models in biopharmaceutical process development.
期刊介绍:
The Journal of Pharmaceutical Sciences will publish original research papers, original research notes, invited topical reviews (including Minireviews), and editorial commentary and news. The area of focus shall be concepts in basic pharmaceutical science and such topics as chemical processing of pharmaceuticals, including crystallization, lyophilization, chemical stability of drugs, pharmacokinetics, biopharmaceutics, pharmacodynamics, pro-drug developments, metabolic disposition of bioactive agents, dosage form design, protein-peptide chemistry and biotechnology specifically as these relate to pharmaceutical technology, and targeted drug delivery.