Regulatory Environment and Approvals in Cell and Gene Therapy Products Between Japan, the USA, and the EU
Regulatory Environment and Approvals in Cell and Gene Therapy Products Between Japan, the USA, and the EU
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日本、美国和欧盟之间细胞和基因治疗产品的监管环境和批准
DOI：
10.1007/s43441-022-00455-4
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发表时间：
2022
影响因子：
1.5
通讯作者：
Shunsuke Ono
中科院分区：
文献类型：
--
作者：
Yuya Sato;Shunsuke Ono
背景本研究旨在通过比较日本、美国和欧盟的法规和成功推出的产品来展示细胞和基因治疗(CGT)产品的开发和审批方式的差异，并检查这些差异的背景。方法从公共来源收集相关法规和批准的CGT产品的信息，并按地区进行比较。结果尽管这些地区对CGT产品的法规基本一致，但一些差异可能会对CGT产品的定义、遵守处理转基因生物的法规所需的反应时间以及良好的制造实践法规下可接受的验证过程产生重大影响。虽然所有地区都给予CGT产品一定的优惠待遇，但不同地区给予CGT产品的优惠待遇不同。在每个地区推出的CGT产品在类型、适应症、开发商的性质和提交的临床证据方面也有很大差异。虽然在日本推出的所有蜂窝产品都是基于小型非对照试验获得批准，但美国和欧盟的大多数蜂窝产品是基于对照研究获得批准的。企业进入国内市场的趋势。结论我们的研究显示，对CGT产品的监管和批准产品的特性以及它们进入国内市场的趋势存在差异，这可能是由与传统药品不同的背景所驱动的。
BackgroundThis study aimed to demonstrate the differences in the way cell and gene therapy (CGT) products have been developed and reviewed for approval in Japan, the USA, and the EU by comparing regulations and successfully launched products in each region, and to examine the background to such differences.MethodsInformation on relevant regulations and approved CGT products were collected from the public source and compared by region.ResultsWhile regulations on CGT products are largely consistent among these regions, some differences could have a substantial impact on the practices defining CGT products, the timing of responses required to comply with the regulations for handling gene-modified organisms, and the acceptable validation processes under good manufacturing practice regulations. Although CGT products are given some preferential status in all regions, the preferential treatment given to CGT products varies across regions. The CGT products launched in each region also differ significantly in type, indications, the nature of the developers, and the clinical evidence submitted. While all the cellular products launched in Japan were approved based on small uncontrolled trials, most cellular products in the USA and EU were approved based on controlled studies. A trend was observed for companies to enter their home markets.ConclusionOur study showed differences of regulations on CGT products and of features in approved products as well as the trend of their home market entries, which may have been driven by a different context than that of traditional pharmaceuticals.
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