Ban Cynthia, Graham Jamie, Le Palaire Lyne, Persaud Priya, Brehme Franziska, Faure Olivier, Rameau Allison, Silva Ana Luisa
Sanofi, Global Regulatory CMC, Toronto, Canada
Sanofi, Global Regulatory CMC, Toronto, Canada.
PDA J Pharm Sci Technol. 2025 Apr 29. doi: 10.5731/pdajpst.2024-003023.1.
Post-approval changes (PACs) are integral to pharmaceutical product lifecycle management ensuring that the product remains safe, effective, and compliant with evolving standards. However, managing these changes across multiple regulatory jurisdictions remains a challenging endeavor due to diverse regulatory requirements and timelines across national regulatory authorities (NRAs). This results in delays obtaining approval from NRAs, impacting global supply chains, and ultimately jeopardizing timely access of essential medical products to patients. In 2021, the WHO issued the Good Reliance Practices (GReIP) guidance to encourage streamlined PAC review and approval process while maintaining access to quality-assured, safe, and effective medicinal products. NRAs are encouraged to rely on the assessment completed by a reference authority that agrees to provide the outcomes of its regulatory expertise. The ultimate objective of this guidance is to accelerate the overall process for PACs, ultimately fostering more equitable and timely access of medical products to populations who need them. This approach was tested in a CMC PAC pilot to determine the feasibility of using the principles of regulatory reliance based on the recommendations outlined in the GReIP with the goal of establishing a predictable, 6-month approval timeframe across multiple NRAs. The design and management of this pilot is described in Gastineau et al.This paper describes the outcomes of the pilot which demonstrates that regulatory reliance is feasible. Of the 21 Regulatory NRAs that agreed to participate, 55% were able to complete the review within 6 months; within 10 months, 95% of approvals were received and, after 16 months, all participating countries had approved the PAC. The use of a Q&A SharePoint Tool allowed for visibility of the questions raised and company responses amongst NRAs. Feedback on this reliance pilot was solicited from the participating NRAs and provides further support for future CMC PAC reliance cases.
批准后变更（PACs）是药品生命周期管理的重要组成部分，可确保产品始终安全、有效，并符合不断发展的标准。然而，由于各国监管机构（NRAs）的监管要求和时间线各不相同，在多个监管辖区管理这些变更仍然是一项具有挑战性的工作。这导致从NRAs获得批准出现延迟，影响全球供应链，并最终危及基本医疗产品及时供应给患者。2021年，世界卫生组织发布了《良好信赖实践》（GReIP）指南，以鼓励简化PAC审查和批准流程，同时确保能够获得质量有保证、安全有效的药品。鼓励NRAs依赖参考当局完成的评估，该参考当局同意提供其监管专业知识的结果。本指南的最终目标是加快PAC的整体流程，最终促进医疗产品更公平、及时地供应给有需要的人群。这种方法在一个CMC PAC试点中进行了测试，以确定根据GReIP中概述的建议使用监管信赖原则的可行性，目标是在多个NRAs建立一个可预测的6个月批准时间框架。Gastineau等人描述了该试点的设计和管理。本文描述了该试点的结果，证明监管信赖是可行的。在同意参与的21个监管NRAs中，55%能够在6个月内完成审查；在10个月内，95%的申请获得批准，16个月后，所有参与国家都批准了PAC。使用问答SharePoint工具可以让NRAs之间看到提出的问题和公司的回复。从参与的NRAs征求了对该信赖试点的反馈意见，为未来的CMC PAC信赖案例提供了进一步支持。
