用实用元素进行临床试验:使用案例和现实世界数据利用情况的审查
Le Su1, Lei Chen1, Seema Betigeri1
1Merck & Co., Inc., Rahway, New Jersey, USA.
Clinical pharmacology and therapeutics
|August 19, 2025
概括
实用性临床试验通过结合真实世界数据 (RWD) 和灵活的设计来提高效率和通用性. 本综述描述了它们的使用,强调了长期结果证据生成的好处.
科学领域:
- 临床试验设计和方法论
- 临床研究中的真实世界数据 (RWD)
- 医疗保健服务研究 医疗服务研究
背景情况:
- 实用性临床试验提供了诸如多样化的患者群体和日常护理的反映等优势.
- 了解它们的设计,特性和使用现实世界数据 (RWD) 是有限的.
- 关于实用因素和RWD如何被整合到试验设计和长期跟进中,知识有限.
研究的目的:
- 描述临床试验使用案例,在设计和实施过程中包含实用元素.
- 确定使用RWD的临床试验,用于扩展阶段的长期随访.
- 在实用试验中了解设计特征,特性和RWD集成.
主要方法:
- 对PubMed和EMBASE数据库进行了有针对性的文献搜索 (2016年1月至2024年7月).
- 描述了27个使用案例,重点关注实用元素和RWD利用.
- 分析包括试验设计 (随机化,盲目化),比较器,结果和RWD来源.
主要成果:
- 22个用例包含实用元素,主要使用随机化 (95.5%) 和开放式设计 (90.9%).
- 共同的务实因素包括广泛的资格,灵活的待遇,最小的后续和简化的终点.
- 在一半的案例中,RWD被用于丰富数据或将试验嵌入医疗保健系统;在5个案例中,RWD被用于扩展阶段的随访.
结论:
- 实用元素越来越多地用于提高临床试验的效率和患者群体的概括性.
- 现实世界的数据整合有助于产生有效性,安全性和长期结果的证据.
- 本次审查强调了实用性试验设计和RWD对于强有力的临床证据日益重要的重要性.
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