在随机试验中可以检测到的最大效应没有上限
Benjamin Djulbegovic1, Iztok Hozo2, Renata Iskander3
1Medical University of South Carolina, Division of Medical Hematology and Oncology, Department of Medicine, Charleston, SC, USA.
Journal of clinical epidemiology
|May 14, 2025
概括
随机对照试验 (RCT) 可以发现显著的癌症治疗效果,没有上限. 增加RCT的数量大大提高了识别突破性疗法和加速医学进步的可能性.
科学领域:
- 在瘤学瘤学.
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 随机对照试验 (RCT) 对于检测小和大治疗效益至关重要.
- 预测潜在的显著治疗效果有助于临床试验资源分配.
研究的目的:
- 预测癌症RCT中未来大治疗效应的可能性.
- 为指导对临床试验进行的资源分配.
主要方法:
- 从1955年到2022年,对716个癌症RCT进行了分析,涉及约35万名患者.
- 应用极值理论,使用通用帕雷托分布 (GPD) 预测最大治疗效应.
主要成果:
- 在GPD分析表明没有最大治疗效果的理论上限.
- 从历史上看,观察到的最大赔率比率 (OR) 是45;预计未来50年最大的OR是23.
- 在50年内检测OR>50的治疗存在20%的概率;增加RCT可以显著提高这种可能性.
结论:
- 在癌症RCT中可能没有可发现的治疗效果的绝对上限.
- 进行更多的RCT对于增加识别具有大效果的治疗方法的可能性至关重要.
- 持续或扩大试验活动,加上公众参与,对于加速治疗突破至关重要.
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