Gallo Paul, Chuang-Stein Christy, Dragalin Vladimir, Gaydos Brenda, Krams Michael, Pinheiro José
Novartis Pharmaceuticals, East Hanover, NJ 07936, USA.
J Biopharm Stat. 2006 May;16(3):275-83; discussion 285-91, 293-8, 311-2. doi: 10.1080/10543400600614742.
A PhRMA Working Group on adaptive clinical trial designs has been formed to investigate and facilitate opportunities for wider acceptance and usage of adaptive designs and related methodologies. A White Paper summarizing the findings of the group is in preparation; this article is an Executive Summary for that full White Paper, and summarizes the findings and recommendations of the group. Logistic, operational, procedural, and statistical challenges associated with adaptive designs are addressed. Three particular areas where it is felt that adaptive designs can be utilized beneficially are discussed: dose finding, seamless Phase II/III trials designs, and sample size reestimation.
已成立了一个药品研究和制造商协会（PhRMA）适应性临床试验设计工作组，以调查并推动适应性设计及相关方法获得更广泛接受和使用的机会。一份总结该工作组研究结果的白皮书正在编写中；本文是该完整白皮书的执行摘要，总结了工作组的研究结果和建议。文中讨论了与适应性设计相关的逻辑、操作、程序和统计方面的挑战。还探讨了认为可以有益地利用适应性设计的三个特定领域：剂量探索、无缝II/III期试验设计以及样本量重新估计。
