The randomized placebo-phase design for clinical trials
The randomized placebo-phase design for clinical trials
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DOI：
10.1016/s0895-4356(00)00357-7
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发表时间：
2001-06-01
影响因子：
7.2
通讯作者：
Szalai, JP
中科院分区：
文献类型：
--
作者：
Feldman, B;Wang, E;Szalai, JP
随机对照试验是评价药物治疗效果的标准方法。然而，在某些情况下，潜在受试者或其医生无法接受在随机对照试验中进入对照组的可能性。这种不被接受的情况是权责发生制不佳的一个原因。我们开发并验证了一种新的生存数据临床试验设计，该设计可能会减轻对未接受研究药物的担忧，并且应该更容易接受。被称为随机安慰剂期设计，这个新设计询问，平均而言，那些开始积极治疗的受试者是否比那些开始较晚的受试者反应更快。使用Monte Carlo计算机模拟，我们证明了该设计是有效的，在某些情况下可能比传统的随机对照试验提供优势。随机安慰剂期设计可能特别有用时，高效力的治疗罕见疾病的测试或当招募可能是困难的。(C)2001 Elsevier Science Inc. All rights reserved.
Randomized controlled trials are the criterion standard method for evaluating the effectiveness of medical treatments. There are situations, however, where the possibility of being in the control group in a randomized controlled trial is unacceptable to potential subjects or their physicians. This lack of acceptance is a reason for poor accrual. We developed and validated a new clinical trial design for survival data that may allay concerns about not receiving an investigational product and should be more acceptable. Called the randomized placebo-phase design, this new design asks whether, on average, those subjects who begin active treatment sooner respond sooner than those who begin it later. Using Monte Carlo computer simulations, we demonstrated that the design is valid and may offer advantages over traditional randomized controlled trials in some situations. The randomized placebo-phase design may be especially useful when highly potent therapies for rare diseases are tested or when accrual may be otherwise difficult. (C) 2001 Elsevier Science Inc. All rights reserved.