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单臂试验的挑战和标准导致德国医疗技术评估的额外好处

Jörg Tomeczkowski¹, Tanja Heidbrede², Birte Eichinger³

¹EGDE-Evidence-generating Data Evaluation, Dormagen, NW, Germany. joerg@tomeczkowski.de.

PharmacoEconomics

|July 27, 2025

概括

单臂试验 (SAT) 在德国医疗技术评估中很少被认为是额外的好处. 当随机对照试验不可行时,需要更明确的SAT标准.

科学领域:

卫生技术评估卫生技术评估
临床试验设计临床试验设计
监管科学监管科学

背景情况:

随机对照试验 (RCT) 是医疗技术评估的黄金标准.
单臂试验 (SAT) 经常被用于RCT是不可行的或不道德的.

研究的目的:

检查德国医疗保健质量和效率研究所 (IQWiG) 和/或联邦联合委员会 (G-BA) 接受SAT的额外收益.
确定影响SAT在医疗技术评估中的接受因素.

主要方法:

从AMNOG-Monitor数据库对健康技术评估的分析,直到2024年12月.
对SATS和其他增加福利决定的证据进行比较,按孤儿药物地位,营销许可,适应症和人群分层.
制造商声明,IQWiG建议和G-BA评估的比较.

主要成果:

在1738个G-BA决策中,13.5%的完全评估的子群体 (202/1491) 是基于SAT.
基于SAT的评估显示,12.2%的病例 (36/295) 具有额外的益处,慢性型肝炎治疗和儿科适应症的比率更高.
IQWiG仅接受G-BA认可的SAT测试的18.5% (5/27) ,其中索赔,建议和评估之间存在显著差异.

结论:

医疗技术评估中SAT的接受率仍然很低.
通常缺乏明确的标准来确定SATs的附加益处.
在与G-BA合作制定明确的,具有约束力的标准对于未来的利益评估至关重要,当RCT不可行时.

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