Office of New Drug III, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Office of Standards and Compliance for Medical Devices, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Clin Pharmacol Ther. 2022 Jan;111(1):35-43. doi: 10.1002/cpt.2410. Epub 2021 Sep 18.
There is growing interest in the utilization of real-world data (RWD) and real-world evidence (RWE) for regulatory purposes. However, there are challenges in the practical utilization of RWD to provide RWE as a basis for regulatory decision making. This article presents the regulatory initiatives in Japan and efforts taken to promote the utilization of RWD/RWE for regulatory decision making at the pre- and postapproval stages of a drug. There has been a rapid increase in the number of RWD cases evaluated for drug safety assessment in Japan. Nevertheless, more regulatory experiences and considerations are necessary for the utilization of RWD in the efficacy evaluation of a drug. Based on past experiences, data reliability and appropriateness of the methodology for analysis are the major discussion points in utilizing RWD and RWE for regulatory decision making. International harmonization of regulatory requirements is another important area in utilizing RWD and sharing the RWE globally. We describe our perspective on providing RWE, which is useful for regulatory decision making throughout a drug's life cycle.
人们对利用真实世界数据（RWD）和真实世界证据（RWE）来进行监管越来越感兴趣。然而，在实际利用 RWD 提供 RWE 作为监管决策依据方面仍存在挑战。本文介绍了日本的监管举措，以及为促进在药物的审批前和审批后阶段利用 RWD/RWE 进行监管决策所做的努力。在日本，用于药物安全性评估的 RWD 案例数量迅速增加。然而，在药物疗效评估中利用 RWD 还需要更多的监管经验和考虑因素。基于过去的经验，在利用 RWD 和 RWE 进行监管决策时，数据可靠性和分析方法的适当性是主要的讨论点。监管要求的国际协调是利用 RWD 和全球共享 RWE 的另一个重要领域。我们描述了我们对提供 RWE 的看法，这对于药物整个生命周期的监管决策是有用的。
