现实世界的证据和观察性研究:临床研究中的方法论挑战
Carmine Zoccali1,2,3, Giovanni Tripepi4
1Renal Research Institute, New York, New York, USA.
European journal of clinical investigation
|December 8, 2025
概括
从常规护理中的真实世界数据 (RWD) 收集的真实世界证据 (RWE) 提供了超越临床试验的宝贵见解. 观察性研究是RWE的关键,但需要谨慎设计,以解决混问题并确保可靠的发现.
科学领域:
- 内部医学 内部医学
- 临床研究方法论 临床研究方法论
- 医疗信息学 医疗信息学
背景情况:
- 现实世界的证据 (RWE) 扩大了临床研究的范围,超出了随机对照试验 (RCT).
- RWE是从常规临床实践中收集的真实世界数据 (RWD) 中得出的.
- 登记册和电子健康记录 (EHR) 是大规模纵向RWD的关键来源.
研究的目的:
- 探索观察性研究在产生RWE方面的作用和挑战.
- 强调RWE在内科医学中的重要性,以了解患者的治疗结果和流行病学.
- 讨论提高RWE可靠性和有效性的方法.
主要方法:
- 使用观察性研究设计 (队列,病例对照,横截面) 来生成RWE.
- 从注册表和电子健康记录中获取数据,以获得全面的患者信息.
- 采用先进的统计技术 (例如,倾向性得分匹配) 来解决混.
- 遵守标准化报告准则 (例如,STROBE) 以确保透明度.
主要成果:
- 观察性研究提供了对患者结果,治疗模式和疾病流行病学的见解.
- 方法方面的挑战包括混,偏见和数据质量问题 (缺少数据,错误分类).
- 先进的统计方法可以减轻但不能消除与非随机数据固有的局限性.
- 透明的报告和严格的分析对于可信的RWE至关重要.
结论:
- 观察性研究对于回答RCT未解决的临床问题是不可或缺的.
- RWE在临床实践和医疗保健政策决策方面提供了重要的信息.
- 为了最大限度地提高RWE的影响,需要仔细的研究设计,严格的分析和透明的报告.
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