Optimal planning of phase II/III programs for clinical trials with multiple endpoints
Optimal planning of phase II/III programs for clinical trials with multiple endpoints
复制标题
多终点临床试验II/III期方案的优化规划
DOI：
10.1002/pst.1861
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发表时间：
2018
影响因子：
1.5
通讯作者：
Götte
中科院分区：
文献类型：
--
作者：
Kieser;Kirchner;Dölger;Götte
由于成本增加和竞争压力，药物开发变得越来越具有挑战性。因此，迫切需要通过开发和应用定量决策方法来提高临床研究的效率。在这种情况下，II/III期项目的综合规划发挥着重要作用，因为许多数量可能会有所不同，这对成本、效益和项目成功至关重要。最近，已经提出了一个基于效用的框架，用于II/III期项目的最佳规划，该框架将决策边界和II期样本量的选择置于定量基础上。但是，该方法仅限于具有单一至事件时间终点的研究。我们将此程序推广到具有多个终点和（渐近）正态分布检验统计量的临床试验。针对“全有或全无”和“至少一个”获胜标准提供了最佳第二阶段样本量和通过/不通过决策规则。所提出的方法的应用说明阿尔茨海默病和肿瘤学领域的药物开发计划。
Owing to increased costs and competition pressure, drug development becomes more and more challenging. Therefore, there is a strong need for improving efficiency of clinical research by developing and applying methods for quantitative decision making. In this context, the integrated planning for phase II/III programs plays an important role as numerous quantities can be varied that are crucial for cost, benefit, and program success. Recently, a utility‐based framework has been proposed for an optimal planning of phase II/III programs that puts the choice of decision boundaries and phase II sample sizes on a quantitative basis. However, this method is restricted to studies with a single time‐to‐event endpoint. We generalize this procedure to the setting of clinical trials with multiple endpoints and (asymptotically) normally distributed test statistics. Optimal phase II sample sizes and go/no‐go decision rules are provided for both the “all‐or‐none” and “at‐least‐one” win criteria. Application of the proposed method is illustrated by drug development programs in the fields of Alzheimer disease and oncology.
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DOI：
10.1016/j.dadm.2016.02.006
发表时间：
2016
期刊：
Alzheimer's & dementia (Amsterdam, Netherlands)
影响因子：
--
作者：
Siemers E;Holdridge KC;Sundell KL;Liu-Seifert H
通讯作者：
Liu-Seifert H
影响因子：
8.4
作者：
Amir, Eitan;Seruga, Bostjan;Ocana, Alberto
通讯作者：
Ocana, Alberto
DOI：
10.1176/ajp.141.11.1356
发表时间：
1984-11
期刊：
The American journal of psychiatry
影响因子：
--
作者：
W. Rosen;R. Mohs;K. Davis
通讯作者：
W. Rosen;R. Mohs;K. Davis
2012 年伦敦。
影响因子：
--
作者：
Carolyn N. Orson;R. Larson
通讯作者：
R. Larson
影响因子：
2
作者：
Ren, Shijie;Oakley, Jeremy E.
通讯作者：
Oakley, Jeremy E.