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基于生理学的生物药剂学模型报告模板的开发:鉴于监管提交要求,对提高质量的考量

Development of a Physiologically Based Biopharmaceutics Model Report Template: Considerations for Improved Quality in View of Regulatory Submissions.

作者信息

Arora Sumit, Pepin Xavier, Jamei Masoud, Sharma Pradeep, Heimbach Tycho, Wagner Christian, Bransford Philip, Kollipara Sivacharan, Ahmed Tausif, Hingle Martin, Dallmann André, Scherholz Megerle, Stamatis Stephen D, Cano-Vega Mario, Mistry Nena, Tannergren Christer, Borges Luiza, Lindahl Anders, Rullo Gregory, Mackie Claire, Mitra Amitava, Kushner Joseph, Harish Jain Krutika Meena, Polli James E

机构信息

Janssen Pharmaceutica NV, Turnhoutseweg 30, 2340 Beerse, Belgium.

Simulations Plus Inc., 42505 10th Street West, Lancaster, California 93534, United States.

出版信息

Mol Pharm. 2025 Jun 2;22(6):2735-2746. doi: 10.1021/acs.molpharmaceut.5c00225. Epub 2025 Apr 29.

Abstract

Pharmaceutical innovators and generic companies use Physiologically Based Biopharmaceutics Models (PBBMs) to guide drug product development and potentially waive clinical pharmacokinetic studies for both pre- and postapproval changes. This modeling approach can assist with biopharmaceutics risk assessment and the establishment of patient centric, clinically relevant drug product specifications. However, the variability of possible model strategies and the existence of gaps in scientific knowledge associated with the lack of standardized regulatory expectations for model parametrization, data requirements for model development, and criteria for fit-for-purpose model validation leads to varied acceptance rates and frequent requests for additional information and deficiencies in PBBM submissions across regulatory agencies. During the 2023 Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) PBBM Best Practices for Drug Product Quality: Regulatory and Industry Perspectives workshop, it was identified that a PBBM report template summarizing model considerations and proposing a structure for presenting question(s) of interest, model context, input data, a modeling plan, and validation would be beneficial for both industry and regulatory agencies. The present work is not a regulatory guideline but rather a summary of current best practices and considerations for PBBM submissions. The associated template can be downloaded directly from the Supporting Information to guide one in the preparation of PBBM reports. The current paper discusses the critical elements of the PBBM report template, which were identified during the industry-regulator scientific collaboration and interactions.

摘要

制药创新企业和仿制药公司使用基于生理的生物药剂学模型(PBBM)来指导药品开发,并可能免除批准前和批准后变更的临床药代动力学研究。这种建模方法有助于生物药剂学风险评估,并建立以患者为中心、与临床相关的药品规格。然而,可能的模型策略存在变异性,并且由于对模型参数化缺乏标准化的监管期望、模型开发的数据要求以及适用目的模型验证的标准,导致科学知识存在空白,这使得各监管机构对PBBM提交文件的接受率各不相同,并经常要求提供更多信息以及指出PBBM提交文件存在缺陷。在2023年马里兰卓越监管科学与创新中心(M-CERSI)关于药品质量的PBBM最佳实践:监管与行业视角研讨会上,确定了一个PBBM报告模板,该模板总结模型考量因素,并提出一种结构来呈现感兴趣的问题、模型背景、输入数据、建模计划和验证,这对行业和监管机构都将是有益的。本工作并非监管指南,而是PBBM提交文件的当前最佳实践和考量因素的总结。相关模板可直接从补充信息中下载,以指导撰写PBBM报告。本文讨论了PBBM报告模板的关键要素,这些要素是在行业与监管机构的科学合作与互动过程中确定的。

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