Warwick Medical School, The University of Warwick, Coventry, UK.
Stat Methods Med Res. 2011 Dec;20(6):623-34. doi: 10.1177/0962280210379035. Epub 2010 Aug 19.
In recent years, there has been a drive to save development costs and shorten time-to-market of new therapies. Research into novel trial designs to facilitate this goal has led to, amongst other approaches, the development of methodology for seamless phase II/III designs. Such designs allow treatment or dose selection at an interim analysis and comparative evaluation of efficacy with control, in the same study. Methods have gained much attention because of their potential advantages compared to conventional drug development programmes with separate trials for individual phases. In this article, we review the various approaches to seamless phase II/III designs based upon the group-sequential approach, the combination test approach and the adaptive Dunnett method. The objective of this article is to describe the approaches in a unified framework and highlight their similarities and differences to allow choice of an appropriate methodology by a trialist considering conducting such a trial.
近年来，人们一直致力于降低新药研发成本并缩短其上市时间。为实现这一目标，人们对新的试验设计方法进行了研究，其中包括用于无缝 II/III 期设计的方法。此类设计允许在中期分析时进行治疗或剂量选择，并在同一研究中对疗效进行与对照的比较评估。与针对各个阶段单独进行试验的传统药物开发方案相比，这些方法具有潜在优势，因此备受关注。本文基于群组序贯设计、联合检验设计和自适应 Dunnett 方法，对各种无缝 II/III 期设计方法进行了综述。本文的目的是在统一的框架内描述这些方法，并突出它们的异同，以便考虑进行此类试验的试验人员选择合适的方法。
