在血液学中使用现实世界的证据
Francesco Passamonti1, Giovanni Corrao2, Gastone Castellani3
1Università Degli Stu di di Milano, Milan, Italy; Fondazione I.R.C.C.S. Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy.
Best practice & research. Clinical haematology
|March 15, 2024
概括
现实世界数据 (RWD) 和人工智能可以生成现实世界的证据 (RWE),以补充随机临床试验 (RCT). 这种方法可能会加强血液学中药物批准决策.
科学领域:
- 血液学 血液学 血液学
- 临床研究 临床研究
- 医疗信息学 医疗信息学
背景情况:
- 随机临床试验 (RCT) 是药物批准的标准,但有局限性.
- 可限制RCT研究结果的通用性,从而与现实实践形成差距.
- 类似RCT的相互矛盾的结论凸显了对补充数据源的需求.
研究的目的:
- 探索现实世界数据 (RWD) 在血液学中的潜力.
- 评估来自RWD的现实世界证据 (RWE) 如何支持监管决策.
- 突出人工智能在RWD处理RWE生成中的作用.
主要方法:
- 对药物批准中的RCT,RWD和RWE现有文献的审查.
- 讨论人工智能在RWD分析中的应用.
- 探索RCT结论与现实世界观察之间的差距.
主要成果:
- 来自注册表的真实世界数据 (RWD) 为RCT数据提供了有价值的补充.
- 人工智能可以有效地处理RWD以产生强大的现实世界证据 (RWE).
- 从RWD衍生出的RWE显示了在弥合临床试验结果和临床实践之间的差距方面的潜力.
结论:
- 从真实世界数据 (RWD) 获得的真实世界证据 (RWE) 可以补充用于监管提交的RCT数据.
- 整合RWD和AI有望提高血液学中药物批准决策的准确性和适用性.
- 对RWE方法的进一步研究对于其在监管科学中的最佳利用至关重要.
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