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谁想先来?一项关于独立试验与启动平台试验的累积效果的模拟研究。

Who wants to go first? A simulation study of accrual in a stand-alone trial versus starting a platform trial.

机构信息

AstraZeneca, Oncology Statistical Innovation, Gaithersburg, MD, USA.

Janssen, Statistical Modeling and Methodology, Gothenburg, Sweden.

出版信息

Contemp Clin Trials. 2022 Dec;123:107000. doi: 10.1016/j.cct.2022.107000. Epub 2022 Nov 11.

Abstract

Others have quantified the efficiency of the platform approach as compared to a sequence of independent two-arm trials and have shown the platform approach more efficiently evaluates a set of candidate therapies. However, a practical barrier to initiating a platform trial is incentivizing the first candidate therapies to enter the platform. A platform trial is more complex and will take longer to design and operationalize than a traditional trial. For the first therapy, this additional up-front planning time must be considered along with the ability to enroll. There is a common concern that accrual in a platform setting would take longer than for a single stand-alone trial because intuition suggests that a two-arm trial with a smaller total sample size should complete accrual more quickly than a multi-armed trial. We focus on the accrual duration for the first therapy as a particular barrier to initiating a platform trial strategy. We simulate accrual into a platform trial versus a stand-alone trial in the setting of a large clinical trial network. Accrual duration in the platform strategy dominates that of a single stand-alone trial if the platform leverages a large enough fraction of the site network. Patient preference for a particular stand-alone trial has little impact on the ability of a stand-alone trial to enroll more quickly.

摘要

其他人已经将平台方法的效率与一系列独立的双臂试验进行了量化,并表明平台方法更有效地评估了一组候选疗法。然而,启动平台试验的一个实际障碍是激励第一批候选疗法进入平台。与传统试验相比,平台试验的设计和实施更为复杂,需要更长的时间。对于第一种疗法,必须考虑到这一额外的前期规划时间以及招募的能力。人们普遍担心,由于直觉认为总样本量较小的双臂试验应该比多臂试验更快地完成入组,因此平台环境中的入组时间会更长。我们关注的是第一个疗法的入组持续时间,这是启动平台试验策略的一个特殊障碍。我们在大型临床试验网络的环境中模拟了平台试验与独立试验的入组情况。如果平台利用了足够大的站点网络的一部分,那么平台策略的入组持续时间将主导单独立项试验的入组持续时间。患者对特定单独立项试验的偏好对单独立项试验更快招募的能力几乎没有影响。

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