FDA对基因治疗产品原材料放行的观点

偷偷溜

FDA在问答培训视频中的回复建议

What is theFDA's position on performing identity testing as a requirement for raw materialrelease to manufacture gene therapy products in clinical phases? Can riskassessment be used to support not performing identity tests for some materials?

FDA对于在临床阶段制造基因治疗产品所需的原材料放行进行鉴别测试的立场是什么？风险评估能否用于支持某些材料不进行鉴别测试？

DR. GAVIN:

That’s a good question. And we had several versionsof that question come in. And so I’ll try to address that.

GAVIN博士：

这是一个很好的问题，我们收到了几个类似的问题，我来回答一下。

During phase 1 studies and early investigationalstudies, sponsors may follow the phase 1 GMP guidance,which outlines CBER expectations for phase 1 studies. This guidance gives youinformation to focus on how the manufacturer’s quality unit controls anddocuments operations in the facility, as well as how product quality ismaintained. In this case, a risk assessment may be used to qualify materials.However, if critical materials such as plasmids or cell banks, which really arenot verified — it could jeopardize the manufacturing process and delay clinicaldevelopment, so mix-ups do occur, and we can trust, but we recommend that youalso verify.

在一期临床和早期调查研究中，申请者可以遵循一期GMP指南，该指南概述了CBER对一期研究的期望。该指南提供了关于制造商质量部门如何控制和记录设施操作以及如何维护产品质量的信息。在这种情况下，可以使用风险评估来评估物料是否合格。然而，如果像质粒或细胞库这样的关键材料没有经过验证，可能会危及制造过程并延误临床开发，因此混淆是可能的，我们可以信任，但我们建议您也进行验证。

By the time you start your phase 2 studies, there’san expectation that manufacturers will operate under the GMPs outlined in the21 CFR 211. Compliance with GMP requires that materials such as components andcontainer starting materials — raw materials used in the manufacture of thedrug product — when they come into the GMP facility, are tested to verifyidentity. This is 211 84-D:1, to use Anna’s vernacularand conformity to specifications that for purity, strength, and quality; thisis 211 84-D:2. Specifications for raw materials and plasmids and such aredetermined by the nature of the material and the needs of the manufacturer.

当进入二期研究时，我们期望制造商遵守21 CFR211中GMP体现要求。符合GMP要求意味着在原材料进入GMP设施时，诸如组分和容器起始材料等用于药品生产的原料必须经过鉴别验证测试。这是211 84-D:1的要求。符合纯度、含量和质量的规格；这是211 84-D:2的要求。原材料和质粒等的标准是由材料的性质和制造商的需求决定的。

As an alternative to some of the conformity of thespecifications in the regulations, a sponsor may rely on vendor information forconfirmation of purity, strength, and quality. And this is if they perform aspecific identity test, so if you do identity testing, and that themanufacturer has confidence in the supplier.

作为符合规范的替代方案，申办者可以依赖供应商提供的纯度、含量和质量确认信息。前提是供应商进行了特定的鉴别测试，也就是说，如果您进行了鉴别测试，并且制造商对供应商有信心。

How is confidence gained? Confidence is gainedthrough a prior history of quality assurance and testing verification ofspecific vendors. Manufacturers may perform a risk assessment as part of yourvendor qualification, but please keep in mind that you still need to verifyidentity. Thank you.

如何获得信心？信心是通过供应商的质量保证和测试验证的先前记录获得的。制造商可以作为供应商资格认证的一部分进行风险评估，但请记住，您仍然需要进行鉴别验证。谢谢。

xqliu

学习了，谢谢提供分享。

3812288

学习了，谢谢提供分享。

sd2789390

习了，谢谢提供分享。

深海屿鹿

学习了，谢谢！