关于使用现实世界数据和现实世界证据作为外部控制的PMDA观点:最近的例子和考虑
Junichi Asano1, Hiromi Sugano1, Hiroyuki Murakami2
1Biostatistics Group, Center for Product Evaluation, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
Clinical pharmacology and therapeutics
|January 3, 2025
概括
在日本,现实世界数据 (RWD) 和现实世界证据 (RWE) 越来越多地用于药物批准,特别是在罕见疾病方面. 在所有研究阶段仔细考虑RWD/RWE对于监管审查和决策至关重要.
科学领域:
- 药监和药物开发 药物监督和药物开发
- 监管科学 监管科学
- 医疗信息学 医疗信息学
背景情况:
- 关于真实世界数据 (RWD) 和真实世界证据 (RWE) 的讨论往往优先考虑药物开发而不是营销后监测.
- 在日本,RWD/RWE已被实际应用为药物批准的外部控制,特别是对于孤儿疾病,随机对照试验的可行性较低.
研究的目的:
- 介绍日本对RWD/RWE的监管审查经验,用于在新药应用中使用外部控制的疗效评估.
- 描述在药物评估和批准中使用RWD/RWE的监管考虑因素.
- 促进适当的药物开发,并与PMDA等监管机构就RWD/RWE进行有关RWD/RWE的讨论.
主要方法:
- 对日本新药申请的监管提交的审查,该申请使用RWD/RWE作为有效性评估的外部控制.
- 分析与药物批准中的RWD/RWE相关的监管考虑和决策过程.
主要成果:
- 在日本,RWD/RWE已被用作药物批准的外部控制,特别是对于孤儿疾病.
- 使用RWD/RWE提供了支持监管审查的补充信息.
- 在所有研究阶段 (规划,分析,解释) 考虑RWD/RWE至关重要.
结论:
- 在日本积累RWD/RWE的监管经验将增强对药物批准的理解并改善监管决策.
- 鼓励使用RWD/RWE进行适当的药物开发,并与监管机构进行公开讨论.
- 进一步完善RWD/RWE利用将支持强大的药物评估和批准过程.
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