Quality risk management and data integrity in R&D laboratories supporting CMC lifecycle of biological products.

IF 3.7 3区 医学 Q2 CHEMISTRY, MEDICINAL Journal of pharmaceutical sciences Pub Date : 2024-09-21 DOI:10.1016/j.xphs.2024.09.013
Brent S Kendrick, John P Gabrielson, Deanna Hunt, Merry Christie, Steven Bowen, Christina Vessely, Richard S Rogers, Chad Cleveland, Karl Maluf, Shawn Roach, Nadine Ritter
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Abstract

The development of pharmaceutical products is the critical bridge that moves a potential new medicine from academic discovery to applied treatment of patients. It translates an idea for a new drug to bench-level research on how it can be manufactured, formulated, characterized and controlled for use in non-clinical and early clinical trials. From pre-clinical R&D discovery work through the commercial launch, substantial R&D CMC data is generated to develop and optimize cGMP manufacturing and testing operations, while also supporting product comparability, elucidating product / impurity structures, assessing critical quality attributes, developing the drug delivery mode, and developing the product formulation for long-term stability. Significant R&D CMC work continues post-approval to support continuous improvement and market expansion of the commercial product. These activities are crucial elements of Product Lifecycle Management, and taken together, they comprise Pharmaceutical Quality or Chemistry, Manufacturing and Controls (CMC). The objective of this paper is to mitigate the regulatory ambiguity of R&D quality systems with practical, risk-based examples and recommendations when conducting supportive CMC studies for biological products. Making sound strategic CMC decisions under any circumstances assumes data from R&D studies are reliable, traceable, and complete. While there are specific regulatory guidelines on phase-appropriate cGMP activities, none exist for quality practices in R&D CMC laboratories conducting non-cGMP studies. Hindsight is not the time to discover that R&D studies lack key elements that would otherwise have allowed the data to be directly presented to regulators, if needed. There is a strong prospective business interest in protecting considerable investments made for CMC R&D studies. Therefore, establishment of a robust and stage-appropriate R&D laboratory quality system is essential for companies seeking to capitalize on prior knowledge, protect investments, and be prepared for accelerated approval pathways.

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支持生物产品 CMC 生命周期的研发实验室的质量风险管理和数据完整性。
医药产品的开发是将潜在新药从学术发现转化为应用治疗病人的关键桥梁。它将新药的想法转化为如何生产、配制、表征和控制新药的台式研究,以便用于非临床和早期临床试验。从临床前研发发现工作到商业投放市场,都会产生大量的研发 CMC 数据,用于开发和优化 cGMP 生产和测试操作,同时还支持产品可比性、阐明产品/杂质结构、评估关键质量属性、开发给药模式以及开发长期稳定的产品配方。大量的研发 CMC 工作将在批准后继续进行,以支持商业产品的持续改进和市场拓展。这些活动是产品生命周期管理的关键要素,它们共同构成了药品质量或化学、制造和控制 (CMC)。本文旨在通过实用的、基于风险的示例和建议,在对生物产品进行支持性 CMC 研究时,减轻研发质量体系在监管方面的模糊性。要在任何情况下做出合理的 CMC 战略决策,研发研究的数据必须可靠、可追溯且完整。虽然有关于阶段性适当 cGMP 活动的具体监管指南,但没有任何指南适用于进行非 cGMP 研究的研发 CMC 实验室的质量实践。如果发现研发研究缺乏关键要素,而这些要素本可以在需要时直接向监管机构提交数据,那么现在就不能事后诸葛亮了。保护对 CMC 研发研究的大量投资,是未来企业的重大利益所在。因此,建立健全的、与阶段相适应的研发实验室质量体系,对于希望利用先前知识、保护投资并为加速审批途径做好准备的公司来说至关重要。
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来源期刊
CiteScore
7.30
自引率
13.20%
发文量
367
审稿时长
33 days
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.
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