In the era of precision medicine, it has been increasingly recognized that individuals with a certain disease are complex and different from each other. Due to the underestimation of the significant heterogeneity across participants in traditional “one-size-fits-all” trials, patient-centered trials that could provide optimal therapy customization to individuals with specific biomarkers were developed including the basket, umbrella, and platform trial designs under the master protocol framework. In recent years, the successive FDA approval of indications based on biomarker-guided master protocol designs has demonstrated that these new clinical trials are ushering in tremendous opportunities. Despite the rapid increase in the number of basket, umbrella, and platform trials, the current clinical and research understanding of these new trial designs, as compared with traditional trial designs, remains limited. The majority of the research focuses on methodologies, and there is a lack of in-depth insight concerning the underlying biological logic of these new clinical trial designs. Therefore, we provide this comprehensive review of the discovery and development of basket, umbrella, and platform trials and their underlying logic from the perspective of precision medicine. Meanwhile, we discuss future directions on the potential development of these new clinical design in view of the “Precision Pro”, “Dynamic Precision”, and “Intelligent Precision”. This review would assist trial-related researchers to enhance the innovation and feasibility of clinical trial designs by expounding the underlying logic, which be essential to accelerate the progression of precision medicine.

在精准医疗时代，人们越来越认识到患有某种疾病的个体是复杂且彼此不同的。由于低估了传统“一刀切”试验中参与者之间的显着异质性，开发了以患者为中心的试验，可以为具有特定生物标志物的个体提供最佳的治疗定制，包括篮子、伞式和平台试验设计在主协议框架下。近年来，FDA陆续批准基于生物标志物引导的主方案设计的适应症，表明这些新的临床试验正在迎来巨大的机遇。尽管篮子试验、伞式试验和平台试验的数量迅速增加，但与传统试验设计相比，目前对这些新试验设计的临床和研究理解仍然有限。大多数研究都集中在方法论上，缺乏对这些新的临床试验设计的潜在生物学逻辑的深入了解。因此，我们从精准医学的角度对篮子、伞式和平台试验的发现和发展及其底层逻辑进行了全面的回顾。同时，我们从“Precision Pro”、“Dynamic Precision”和“Intelligent Precision”的角度讨论了这些新临床设计的未来潜在发展方向。本综述将通过阐述背后的逻辑，帮助试验相关研究人员增强临床试验设计的创新性和可行性，这对于加速精准医疗的发展至关重要。