美国基因治疗产品临床试验监管体系及对我国的启示

American regulatory system for clinical trials of gene therapy products and its enlightenment to China

基因治疗是以改变人类基因治疗疾病的技术,已成为近年来全球药物研发的热点与前沿领域。作为风险程度高、成功率低的前沿技术研发,基因治疗产品临床试验的监管是各国药品监管部门面临的新问题。本文梳理全球基因治疗产品临床试验现状,分析美国管理基因治疗产品的监管部门和法律法规体系,并在中美比较的基础上,根据我国的实际情况提出联合监管形成合力、构建动态监管体系、提高法律法规效力层级衔接性、细化规范性文件等针对性的建议。

Gene therapy,by changing human gene to treat disease,has become the hot spot and frontier field of global drug research and development in recent years.As a frontier technology with high risk and low success rate,the supervision of clinical trials of gene therapy products is a new problem faced by drug administration departments in various countries.Through presenting the clinical trials of global gene therapy products,the supervision departments and laws and regulations system of gene therapy products in the United States were analyzed.Based on the comparison between China and the United States and the actual situation of our country,some pertinent suggestions were put forward in this paper,such as the formation of joint supervision for joint force,the construction of a dynamic supervision system,the improvement of effectiveness cohesion level of laws and regulations and the refinement of normative documents.