在肿瘤学试验主方案中使用非同步共同对照：美国统计协会生物制药部分公开论坛讨论报告
Use of Non-concurrent Common Control in Master Protocols in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion
摘要：
本文总结了美国统计协会（ASA）生物制药（BIOP）部分开放论坛的讨论，该论坛于2020年12月10日举行，由ASA BIOP肿瘤学统计方法科学工作组与美国FDA肿瘤学卓越中心协调组织。包括国际监管机构专家、学者和制药行业代表在内的不同利益相关者参与了关于在肿瘤学试验主方案中使用非并行对照的讨论。虽然使用非同期对照与同期对照可能会增加检测治疗和对照之间治疗差异的能力，但小组成员对非同期对照和同期对照建模的统计方法有不同意见。一些人更关心非同期控制的时间性和与时间相关的不同混杂因素引入的偏倚，例如，标准治疗的变化、患者人群的变化、招募策略的变化、终点评估的变化。然而，在某些情况下，例如罕见疾病的招募极具挑战性时，专家组成员得出结论，使用非同期对照是合理的。
英文摘要：
This article summarizes the discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum that took place on December 10, 2020 and was organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group, in coordination with the US FDA Oncology Center of Excellence. Diverse stakeholders including experts from international regulatory agencies, academicians, and representatives of the pharmaceutical industry engaged in a discussion on the use of non-concurrent control in Master Protocols for oncology trials. While the use of non-concurrent control with the concurrent control may increase the power of detecting the therapeutic difference between a treatment and the control, the panelists had diverse opinion on the statistical approaches for modeling non-concurrent and concurrent controls. Some were more concerned about the temporality of the non-concurrent control and bias introduced by different confounders related to time, e.g., changes in standard of care, changes in patient population, changes in recruiting strategies, changes in assessment of endpoints. Nevertheless, in some situations such as when the recruitment is extremely challenging for a rare disease, the panelists concluded that the use of a non-concurrent control can be justified.
机构、总结、中文摘要均由AI分析而来，可能存在错误，请以论文正文为准。
