美国、欧盟和日本基因治疗产品的监管考量
Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan.
作者信息
Halioua-Haubold Celine-Lea, Peyer James G, Smith James A, Arshad Zeeshaan, Scholz Matthew, Brindley David A, MacLaren Robert E
机构信息
Department of Paediatrics, University of Oxford, Oxford, UK.
Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
出版信息
Yale J Biol Med. 2017 Dec 19;90(4):683-693. eCollection 2017 Dec.
Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of traditional small-molecule therapeutics, due to the unique mechanism-of-action of GTPs and the subsequent novel risks arisen. We have provided herein a summary of the regulatory structure under which GTPs fall in the United States, the European Union, and Japan, and a comprehensive overview of the regulatory guidance applicable to the developer of GTP. Understanding the regulatory requirements for seeking GTP market approval in these major jurisdictions is crucial for an effective and expedient path to market. The novel challenges facing GTP developers is highlighted by a case study of alipogene tiparvovec (Glybera).
由于基因治疗产品（GTPs）独特的作用机制以及随之产生的新风险，GTPs的开发者必须遵守超出传统小分子疗法的额外监管规定。我们在此总结了美国、欧盟和日本针对GTPs的监管架构，并全面概述了适用于GTP开发者的监管指南。了解在这些主要司法管辖区寻求GTP市场批准的监管要求，对于实现有效且迅速的上市途径至关重要。通过脂蛋白脂肪酶基因转导载体（Glybera）的案例研究，突出了GTP开发者面临的新挑战。
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