关于癌症临床试验中患者报告结果的估计值的思考
Rachael Lawrance1,2, Konstantina Skaltsa1,3, Antoine Regnault1,4
1Members of the EFSPI/PSI Estimands in Oncology Special Interest Group, PRO Task Force.
Journal of biopharmaceutical statistics
|November 19, 2023
概括
该估计和框架澄清了癌症试验中患者报告的结果 (PRO),改善了与健康相关的生活质量 (HRQoL) 和患者治疗益处的分析.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 患者报告的结果
背景情况:
- 在癌症临床试验中,患者对与健康相关的生活质量 (HRQoL) 的看法至关重要.
- 目前对癌症试验中的PRO的统计分析往往缺乏清晰度,阻碍了可靠的结论和试验可比性.
- ICH E9 (R1) 指南附录引入了一个估计和框架,以解决这些模两可的问题.
研究的目的:
- 在癌症临床试验中,为患者报告的结果 (PRO) 应用ICH E9 (R1) 估计和框架提供统计学家的观点.
- 倡导系统地使用估计和框架来定义明确的PRO研究问题.
- 突出估计和框架对PRO最相关的关键属性,并讨论相关的统计挑战.
主要方法:
- 专注于将ICH E9 (R1) 估计和框架应用于来自癌症临床试验的PRO数据.
- 讨论估计和框架的五个属性,强调终点定义,间流事件和PROs的人口层次摘要.
- 确定最佳实践,并讨论统计分析中的挑战,特别是关于缺失数据和死亡的挑战.
主要成果:
- 估计和框架需要前性定义明确的PRO研究问题.
- 对于PRO的关键估计和属性包括终点定义,间流动事件的管理 (例如,疾病进展,死亡) 和适当的统计总结.
- 在PRO分析中的挑战包括处理缺失/无法观察到的数据以及死亡的影响.
结论:
- 评估框架的系统应用提高了癌症试验中PRO分析的清晰度和可靠性.
- 该框架有助于确保从患者的角度准确量化真正的治疗益处.
- 虽然不是"统计魔法",但估计框架提供了一个结构化的方法来回答关于治疗对HRQoL的影响的关键问题.
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