估计数是否正确使用? 审查英国的研究方案
Timothy P Clark1, Richard H Wicentowski2, Suzie Cro3
1MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL, 90 High Holborn, 2 Floor, London, WC1V 6LJ, UK. tim.clark@ucl.ac.uk.
Trials
|August 27, 2025
概括
国际协调会议E9(R1) 附录增加了临床试验的估计和使用,但报告仍然不足. 需要进一步改进,以充分发挥估计的潜力,以澄清研究问题.
科学领域:
- 临床试验方法
- 制药研究
- 生物统计学
背景情况:
- 2019年11月,国际协调会议关于估计的E9附录 (ICH E9 ((R1) 附录) 正式确定了临床试验中的估计和使用情况.
- ICH E9 ((R1) 附录旨在提高临床试验所涉及的具体研究问题的清晰度和透明度.
- 之前的审查表明,公布的试验方案中对初级估值的明确定义有限.
研究的目的:
- 根据ICH E9 ((R1) 附录,评估临床试验中估计和使用的演变.
- 评估临床试验协议中的估计和属性定义的正确性.
- 在临床试验设计中确定用于管理相互流动事件的策略.
主要方法:
- 分析了向英国卫生研究局提交的29212份原始研究方案.
- 包括标准要求协议提到"估计"并尝试定义至少一个主要的估计属性.
- 提取的数据包括试验随机化,治疗领域,估计和属性定义的正确性以及交互事件处理策略.
主要成果:
- 在ICH E9 (R1) 附录公布后,确定初级估值的协议数量显著增加.
- 估计和描述往往是不理想的,其中忽略了关键属性 (例如人口,治疗条件).
- 估计和分析种群的错误定义是常见的.
结论:
- 虽然ICH E9 ((R1) 附录促进了临床试验的估计和采用,但报告质量需要提高.
- 持续努力是必要的,以确保有效地澄清和集中临床试验研究问题.
- 解决估计和属性的非最佳报告对于最大限度地提高其效益至关重要.
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