评估晚期癌症的整体生存益处:外部比较对象队列研究与现实世界数据的作用
Francesco Pignatti1, Tarec Christoffer El-Galaly2, Martin Kaiser3
1The European Medicines Agency, Amsterdam, The Netherlands.
Clinical pharmacology and therapeutics
|February 11, 2026
概括
使用真实世界数据 (RWD) 的外部比较者队列 (ECC) 研究被认为是癌症治疗单臂试验 (SAT) 的更强有力的证据. 更高质量的RWD ECC研究显示出更大的整体生存 (OS) 益处,获得了更高的证据强度评级.
科学领域:
- 医疗保健服务研究 医疗服务研究
- 临床试验设计 临床试验设计
- 现实世界的证据 (RWE)
背景情况:
- 使用真实世界数据 (RWD) 的外部比较者队列 (ECC) 研究为治疗评估提供了与单臂试验 (SAT) 相比的潜在优势.
- 然而,ECC研究面临挑战,包括选择偏差和数据异质性,影响其发现的可靠性.
- 随机对照试验 (RCT) 仍然是黄金标准,但在某些临床环境中,它们的适用性和可行性可能受到限制.
研究的目的:
- 在医疗保健专业人员 (HCP) 和监管机构 (RA) 评估人员中,评估ECC研究与SAT和RCT相比的认知证据的强度.
- 评估RWD质量和观察到的整体存活率 (OS) 改善等因素如何影响ECC研究的证据评级的强度.
- 了解证据的强度与新癌症治疗中最大可接受的毒性风险之间的关系.
主要方法:
- 联合分析被用来评估证据评级的强度,用于涉及新癌症治疗的假设情景.
- 参与者包括来自欧洲血液学会 (EHA) 和欧洲医学瘤学会 (ESMO) 的医疗保健专家,以及来自国际监管机构 (RA) 的评估员.
- 根据研究设计 (ECC与ECC对比) 情况不同. SAT与RCT),RWD质量,以及没有有效治疗的晚期疾病的OS改善的大小.
主要成果:
- 医疗保健专家参与者对RWD研究的评价比RA参与者 (12.9%) 更为有利 (47.6%).
- 高质量的RWD ECC研究显示1.5个月和3个月的OS改善,与SAT相比,获得更高证据强度评级的几率明显更高 (OR分别为2.7和14.7).
- 与SAT相比,RCT获得证据强度评级的几率要高得多 (OR分别为36.4和358.4).
结论:
- 使用真实数据的外部比较者队列研究被认为比单臂试验更有说服力,用于评估新疗法的整体存活率.
- 对于ECC研究的感知证据的强度随着RWD质量的提高和更大的证明OS益处而增加.
- 证据强度评级受到可接受的毒性值的影响,突出了药物评估中复杂的决策过程.
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