导读：Q:Since gene therapy products are complex and may have
Q:Since gene therapy products are complex and may have limited manufacturing data, what are the expectations around setting commercial specifications with limited experience?由于基因疗法产品复杂且可能具有有限的制造数据，因此在有限的经验下设置商业规格的期望是什么？A:Thanks, Steven. We generally recommend that commercial lot release criteria should be based on lots that are shown to be safe and effective during the clinical study. And this approach will allow you to evaluate correlations between product attributes and clinical outcome. And to this end, we really recommend that you use multiple lots during your clinical study, if possible, even if one lot would be able to treat everybody in those pivotal studies.谢谢，史蒂文。我们通常建议商业批次放行标准应基于在临床研究中证明是安全和有效的批次。这种方法将允许您评估产品属性和临床结果之间的相关性。为此，我们建议您，如果可能的话，即使一个批次能够治疗所有关键性研究中的患者，也在临床研究期间使用多个批次。And so one way to facilitate this would be to conduct PPQ runs and use those PPQ runs during the pivotal study. And so then you’re really getting more bang for your buck with those lots. These data should be used to determine the commercial lot release criteria and to ensure product consistency and quality.
因此，促进这一点的一种方式是实施PPQ，并在关键性研究中使用这些PPQ。这样，您可以真正充分利用这些批次。这些数据应用于确定商业批次放行标准并确保产品一致性和质量。And so that’s really describing the ideal situation. And we realized that this may not be possible for all products, especially those made for rare diseases. And so this is where our conversation becomes much more product-specific. And as Denise mentioned, you can consider leveraging data from multiple drug substance lots in relation to your drug product or design your manufacturing process to allow more drug product lots to be used during the clinical study.
因此，这实际上描述了理想情况。我们意识到这可能不适用于所有产品，特别是那些为罕见疾病制造的产品。这就是我们的谈话变得更加产品特定的地方。正如丹尼斯所提到的，您可以考虑利用与您的药物相关的多个药物物质批次的数据，或者设计您的制造过程以允许在临床研究中使用更多的药物产品批次。And really, if you believe that the amount of data that’s going to be available to set the commercial lot release criteria is going to be a problem, particularly for your product, and we encourage you to come in early and talk to your review team so that we can formulate a plan on how to move forward to get the most information possible in order to set the commercial lot release criteria.
如果您认为将可用的数据量用于设置商业批次放行标准将成为一个问题，特别是针对您的产品，则我们真诚地鼓励您早日来与审评团队交流，以便我们制定计划，以获取最可能的信息，以设置商业批次放行标准。
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