Adaptive design methods in clinical trials - a review.
Adaptive design methods in clinical trials - a review.
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DOI：
10.1186/1750-1172-3-11
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发表时间：
2008-05-02
影响因子：
3.7
通讯作者：
Chang M
中科院分区：
文献类型：
--
作者：
Chow SC;Chang M
近年来，基于累积数据的自适应设计方法在临床研究和开发中的应用由于其灵活性和效率而变得非常流行。根据所应用的适应性，适应性设计可分为三类：前瞻性、并发（临时）和回顾性适应性设计。适应性设计允许对正在进行的临床试验的试验和/或统计程序进行修改。然而，令人担忧的是，调整后的实际患者人群可能偏离最初的目标患者人群，因此可能无法控制总体I类错误（错误地声称感染性药物的疗效）率。此外，对正在进行的试验和/或统计程序的重大调整可能导致完全不同的试验，无法解决试验打算回答的科学/医学问题。在这篇文章中，几个常见的考虑适应性设计在临床试验中进行了审查。描述了特别调整（方案修订）的影响、设计（前瞻性）调整的挑战和回顾性调整的障碍。在罕见病的临床开发中使用自适应设计的策略进行了讨论。文中给出了一些关于万珂用于多发性骨髓瘤和非霍奇金淋巴瘤的开发实例。还讨论了在临床试验中实施自适应设计方法时经常遇到的实际问题。
In recent years, the use of adaptive design methods in clinical research and development based on accrued data has become very popular due to its flexibility and efficiency. Based on adaptations applied, adaptive designs can be classified into three categories: prospective, concurrent (ad hoc), and retrospective adaptive designs. An adaptive design allows modifications made to trial and/or statistical procedures of ongoing clinical trials. However, it is a concern that the actual patient population after the adaptations could deviate from the originally target patient population and consequently the overall type I error (to erroneously claim efficacy for an infective drug) rate may not be controlled. In addition, major adaptations of trial and/or statistical procedures of on-going trials may result in a totally different trial that is unable to address the scientific/medical questions the trial intends to answer. In this article, several commonly considered adaptive designs in clinical trials are reviewed. Impacts of ad hoc adaptations (protocol amendments), challenges in by design (prospective) adaptations, and obstacles of retrospective adaptations are described. Strategies for the use of adaptive design in clinical development of rare diseases are discussed. Some examples concerning the development of Velcade intended for multiple myeloma and non-Hodgkin's lymphoma are given. Practical issues that are commonly encountered when implementing adaptive design methods in clinical trials are also discussed.
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影响因子：
1.7
作者：
Brannath, W;König, F;Bauer, P
通讯作者：
Bauer, P
影响因子：
1.5
作者：
Friede, T;Kieser, M
通讯作者：
Kieser, M
影响因子：
2
作者：
LAN, KKG;DEMETS, DL
通讯作者：
DEMETS, DL
影响因子：
1.1
作者：
Gallo, Paul;Chuang-Stein, Christy;Pinheiro, Jose
通讯作者：
Pinheiro, Jose
影响因子：
2
作者：
BAUER, P;ROHMEL, J
通讯作者：
ROHMEL, J