论文标题
用R包装的复合端点的试验设计
Design of Trials with Composite Endpoints with the R Package CompAREdesign
论文作者
论文摘要
在临床试验中，复合端点被广泛用作主要终点。使用时间到事件端点的试验设计可能特别具有挑战性，因为即使使用复合端点时，即使前提对于它们的组件仍然是正确的，通常不存在比例危险假设。因此，用于样本量计算的常规公式不再适用。我们呈现了R套件的效果，可以根据复合端点组件的信息来计算试验设计的关键要素（例如样本量和效应尺寸）。与Sampesign提供了评估设计计算对初始值和假设变化的敏感性和鲁棒性的功能。此外，我们描述了包装的其他功能，例如设计具有二进制复合端点的试验的功能，以及在各种场景下使用复合端点模拟试验的功能。
Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a composite endpoint, even when the premise remains true for their components. Consequently, the conventional formulae for sample size calculation do not longer apply. We present the R package CompAREdesign by means of which the key elements of trial designs, such as the sample size and effect sizes, can be computed based on the information on the composite endpoint components. CompAREdesign provides the functions to assess the sensitivity and robustness of design calculations to variations in initial values and assumptions. Furthermore, we describe other features of the package, such as functions for the design of trials with binary composite endpoints, and functions to simulate trials with composite endpoints under a wide range of scenarios.
