A COMPARATIVE PHASE-II CLINICAL-TRIALS PROCEDURE FOR CHOOSING THE BEST OF 3 TREATMENTS
A COMPARATIVE PHASE-II CLINICAL-TRIALS PROCEDURE FOR CHOOSING THE BEST OF 3 TREATMENTS
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DOI：
10.1002/sim.4780100902
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发表时间：
1991-09-01
影响因子：
2
通讯作者：
PALMER, CR
中科院分区：
文献类型：
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作者：
PALMER, CR
在一些临床试验中，可以有利地采用自适应设计，尽管在实践中很少使用这种技术，部分原因是其固有的复杂性。 被认为是一个简单而实用的决策理论方法的情况下，三个治疗与二进制响应，使用平等分配给剩余的治疗，一旦消除，治疗不能重新使用。 在规定了试验比较阶段内和比较阶段外接受治疗的患者总数后，目标是最大限度地增加预期的成功治疗患者总数。 对该方法的研究涉及一个可以处理任意大量患者的计算机程序。 经验表明，决策程序的行为只是稍微差于如果真正的上级治疗已经知道，并已给予所有患者。 该方法的实现使用了极大极小化方法，消除了对先验参数的依赖。 与传统的假设检验方法相比，这种方法的一个优点是可以检测到治疗效果的微小改善。 预期的应用是在II期试验期间协助治疗选择，特别是快速反应，以及当所涉及的疾病足够严重时，设计动机的伦理考虑变得至关重要。
In some clinical trials one can employ adaptive designs advantageously, although in practice such techniques are rarely used, in part due to their inherent complexity. A simple and practicable decision-theoretic approach for the case of three treatments with binary responses is considered, using equal allocation to remaining treatments and, once eliminated, a treatment cannot be re-employed. Having specified the overall number of patients treated within and beyond the comparative stages of the trial, the goal is to maximize the expected total number of those successfully treated. Investigation of the method involves a computer program that can handle arbitrarily large numbers of patients. It is shown empirically that the decision procedure behaves only marginally worse than if the truly superior treatment had been known and had been given to all patients. Implementation of the method uses a minimax approach that removes dependence on prior parameters. Primarily an identification procedure, one advantage of this approach over traditional hypothesis testing methods is the potential to detect small improvements in treatment efficacy. The intended application is to assist in treatment selection during phase II trials, especially with rapid responses and when the disease involved is serious enough that design-motivating ethical considerations become paramount.