中国食品药品监管Issue(5)：68-81,14.DOI:10.3969/j.issn.1673-5390.2025.05.007
以加速创新药品可及性为目的的药学研发及监管工具介绍与案例分析
Regulatory Landscape and Case Studies on CMC Tools Supporting Early Access Programs
杨萌 1迟蕾 2席加乐 3张亦璇 4连敏 5王雅英 6吕玉真1
作者信息
- 1. 罗氏(中国)投资有限公司
- 2. 阿斯利康全球研发(中国)有限公司
- 3. 第一三共(中国)投资有限公司
- 4. 葛兰素史克(中国)投资有限公司
- 5. 默沙东研发(中国)有限公司
- 6. 武田亚太生物医药研发有限公司
- 折叠
摘要
Abstract
To accelerate the development of innovative medicines and address unmet medical needs,regulatory authorities in China,the United States,and the European Union have established expedited pathways.These programs have enhanced public access to medicines,ensured emergency responses,and improved efficiency of health authorities.However,products under accelerated pathways often face challenges in completing the full scope of Chemistry,Manufacturing,and Controls(CMC)studies and data requirements within constrained timelines.In response,the U.S.Food and Drug Administration(FDA)and European Medicines Agency(EMA)have collaborated with industry to develop flexible regulatory tools,publishing key guidelines that offer flexibilities such as strategic deferral of certain studies or data submissions.These tools,including but not limited to prior knowledge,risk assessment,innovative process validation strategies,and Post-Approval Change Management Protocols(PACMPs),enable earlier market access while ensuring product quality and managing residual risks.This article reviews two important guidelines from the EMA and FDA and presents case studies involving small molecules,biologics(antibodies),and antibody-drug conjugates.It illustrates that regulatory flexibility does not mean reducing the scope of CMC studies or data packages for submission,but rather applying science-based tools to balance product quality risks and clinical benefits.The journey of China joining globally simultaneous submissions of innovative drugs has only just begun.We hope this article offers insights to inform the development of technical guidelines for accelerated pharmaceutical programs,supporting earlier access to innovative medicines for patients in China.关键词
加速路径/工艺验证/先验知识/药学研究/风险评估/药学/全球同步递交Key words
accelerated pathways/process validation/prior knowledge/pharmaceutical development/risk assessment/chemistry,manufacturing and controls(CMC)/global simultaneous submission分类
药学引用本文复制引用
杨萌,迟蕾,席加乐,张亦璇,连敏,王雅英,吕玉真..以加速创新药品可及性为目的的药学研发及监管工具介绍与案例分析[J].中国食品药品监管,2025,(5):68-81,14.
