Gao Y, Zhong Z H, Lu M Y, Chen F
Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing211166, China.
Zhonghua Liu Xing Bing Xue Za Zhi. 2024 Dec 10;45(12):1700-1704. doi: 10.3760/cma.j.cn112338-20240705-00401.
Due to the limited number of cases, conducting large-scale clinical trials for rare diseases is challenging. This review introduces several small sample statistical designs tailored for rare diseases, including crossover design, -of-1 design, randomized placebo-phase design, randomized withdrawal design, group sequential design, and adaptive design. It discusses the advantages, disadvantages, and application scenarios of these designs. Additionally, it explores the benefits of Bayes decision-making in clinical trials for rare diseases. The aim is to provide a reference for designing and implementing small sample clinical trials for rare diseases.
由于病例数量有限，针对罕见病开展大规模临床试验具有挑战性。本综述介绍了几种为罕见病量身定制的小样本统计设计，包括交叉设计、单病例设计、随机安慰剂阶段设计、随机撤药设计、成组序贯设计和适应性设计。它讨论了这些设计的优缺点和应用场景。此外，还探讨了贝叶斯决策在罕见病临床试验中的益处。目的是为设计和实施罕见病小样本临床试验提供参考。
