在非癌症医疗技术评估中使用外部控制臂的可接受性 提交的卫生技术评估文件
Kamal Kant Mangla1, Spyros Kolovos2, Ana Lisica3
1Novo Nordisk Service Center India Pvt. Ltd.
Journal of comparative effectiveness research
|December 12, 2025
概括
在非瘤医疗技术评估 (HTA) 中使用外部控制臂 (ECA) 在欧洲机构之间有所不同. ECA的可接受性取决于理由,数据和方法,NICE比IQWiG更开放.
科学领域:
- 卫生技术评估 卫生技术评估
- 相对有效性研究研究比较
- 证据综合 证据综合
背景情况:
- 在医疗技术评估 (HTA) 中,越来越多地考虑外部控制臂 (ECA).
- 在欧洲HTA机构中,ECA在非瘤学提交中的可接受性需要进一步调查.
- 了解HTA机构指导和感知的变化对于ECA实施至关重要.
研究的目的:
- 评估外部控制臂 (ECA) 在欧洲非瘤医疗技术评估 (HTA) 提交中的可接受性.
- 分析关键的欧洲HTA机构关于ECA方法的指导和实践.
- 确定影响ECA在HTA接受的驱动因素和障碍.
主要方法:
- 一个连续的混合方法研究,结合文档审查和专家采访.
- 对NICE (英格兰),HAS (法国) 和IQWiG (德国) 发布的指南进行分析.
- 对英国,法国,德国和意大利的非瘤学HTA评估进行了审查,随后与HTA专家进行了六次半结构面试.
主要成果:
- 尼斯和HAS提供一些ECA指导,而IQWiG的指导是有限的.
- ECA主要用于瘤学;在审查的机构中,非瘤学应用很少见.
- 与IQWiG对传统的受控试验的偏好相比,NICE对补充临床试验数据的ECA更开放.
结论:
- 当有强有力的理由,可靠的数据和严格的方法来减轻偏见时,ECA是最可接受的.
- 适合ECA使用的情景包括单臂试验,有限的比较数据或不断变化的护理标准.
- 需要提高对HTA机构的认识和早期参与,以促进ECA作为随机对照试验补充剂的价值.
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