[专题PRO] 根据估计和框架,使用患者报告考虑癌症治疗比较耐受性的终点
John Devin Peipert1, Monique Breslin2, Ethan Basch3
1Medical Sciences, Northwestern University Feinberg School of Medical Sciences, Chicago, Illinois, USA.
Journal of biopharmaceutical statistics
|February 15, 2024
概括
监管机构在癌症试验中优先考虑患者报告的结果 (PRO),以评估治疗耐受性. 本文探讨了使用PROs来比较治疗副作用,解决当前的方法论挑战.
科学领域:
- 瘤学临床试验 临床试验
- 患者报告的结果 (PROs)
- 监管科学是一种监管科学.
背景情况:
- 监管机构越来越多地使用系统方法来处理癌症试验中的患者体验数据.
- 临床医生报告不足突出了患者报告的耐受性与疗效的必要性.
- 目前分析患者报告耐受性的方法是描述性的,正在开发中.
研究的目的:
- 讨论在临床试验中比较患者报告的癌症治疗耐受性的考虑因素.
- 探索ICH E9 (R1) 估计和框架对耐受性终点的应用.
- 为了解决调整试验设计,目标和分析以用于PRO解释的挑战.
主要方法:
- 对实施,分析和解释患者报告的耐受性数据的当前方法的审查.
- 讨论现有的PRO指南对疗效终点与耐受性终点的适用性.
- 在PRO可容忍性背景下考虑ICH E9 (R1) 估计和框架.
主要成果:
- 作为疗效终点的PRO指南已经存在,但它们用于耐受性终点的使用需要进一步开发.
- 在PRO分析中的异质性和不明确的目标对解释耐受性结果提出了挑战.
- ICH E9 (R1) 估计框架为调整试验设计和分析提供了一个潜在的解决方案.
结论:
- 系统地收集和分析患者报告的耐受性对于瘤学的监管决策至关重要.
- 需要进一步的方法开发,以可靠地比较使用PROs的治疗耐受性.
- 调整试验设计,目标和统计分析,可能使用estimatand框架,是可靠的耐受性评估的关键.
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