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在干预性试验中使用代用终点的定义和解释框架
Oriana Ciani1, Anthony M Manyara2, Philippa Davies3
1Centre for Research on Health and Social Care Management, SDA Bocconi School of Management, Milan, Italy.
EClinicalMedicine
|October 25, 2023
概括
这项研究制定了一个框架,以澄清干预试验中使用代用终点的使用情况. 它解决了利益相关者之间定义和解释的不一致性,以改善报告和证据使用.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 健康 结果 研究 研究 结果
背景情况:
- 干预性试验越来越多地使用代用终点来评估治疗效果.
- 定义和报告代用终点的现有框架是不一致和不清楚的.
- 替代终点取代了衡量患者福祉或生存的最终结果.
研究的目的:
- 开发一个框架来定义,解释和报告干预试验中的代用终点.
- 解决当前对替代终点的定义和利益相关者的解释不一致的问题.
- 提高临床研究中替代终点证据的理解和使用.
主要方法:
- 进行了四项相关的经验研究:范围审查,e-Delphi研究,共识会议和网络调查.
- 在各种利益相关者中检查了替代终点的定义和解释.
- 开发了CONSORT (综合报告试验标准) 和SPIRIT (标准协议项目:干预试验建议) 的替代终点扩展.
主要成果:
- 在当前的替代终点定义和解释中发现了显著的不一致性和缺乏共识.
- 发现与临床医生和监管机构相比,患者和卫生技术评估专家更倾向于将中间结果视为替代终点.
- 强调需要对替代终点定义和使用采取标准化的方法.
结论:
- 提出了一个新的框架,用于定义试验中的替代终点 (生物标志物和中间结果) 和目标结果.
- 该框架旨在提高替代终点报告的清晰度,一致性和实用性.
- 更好地理解和报告代用终点对于干预性试验证据的解释和应用至关重要.
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