IPRP关于基因治疗产品生物分布研究的相关考虑

IPRP considerations on the biodistribution of gene therapy products

目前各监管机构对基因治疗产品的生物分布研究可能有不同的要求,为基因治疗产品的开发带来了挑战。国际药物监管计划(IPRP)的基因治疗工作组(GTWG)于2018年6月编写了Expectations for Biodistribution Assessments for Gene Therapy Products意见书,该文件代表了当前不同地区的药品监管机构对支持基因治疗产品进入临床试验和上市申请时需要开展的非临床生物分布研究的认识,重点讨论了支持首次临床试验的生物分布研究、试验设计需考虑因素以及允许在首次临床试验后开展生物分布研究的情况。本文拟通过对该意见书的介绍,概述基因治疗药物生物分布研究的一般考虑,希望对我国基因治疗产品的生物分布研究相关的研发和审评工作提供参考。

There may be different requirements and expectations among various regulatory authorities which creates challenges in gene therapy(GT)products development.The Gene Therapy Working Group(GTWG)of the International Pharmaceutical Regulators Programme(IPRP)proposed the reflection paper on《Expectations for Biodistribution Assessments for Gene Therapy Products》in June 2018.This paper represents the current view of regulatory authorities from various regions on non-clinical BD data expectations to support clinical trials and marketing authorization applications,and discusses the considerations for the design of BD studies to support FIH trials and circumstances that may trigger BD studies during clinical trials.By introducing this reflection paper,we hope to summarize the general considerations on BD studies of GT products and provide some references for the study design and review of non-clinical BD data in China.