| 高源,仲子航,陆梦依,陈峰.罕见病小样本临床试验的统计设计及其应用[J].中华流行病学杂志,2024,45(12):1700-1704 |
| 罕见病小样本临床试验的统计设计及其应用 |
| Statistical design and application of clinical trials with small sample sizes for rare diseases |
| 收稿日期:2024-07-05 出版日期:2024-12-16 |
| DOI：10.3760/cma.j.cn112338-20240705-00401 |
| 中文关键词: 罕见病 小样本 统计设计 临床试验 |
| 英文关键词: Rare diseases Small sample Statistical design Clinical trial |
| 基金项目: |
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| 中文摘要: |
| 罕见病由于病例少，难以开展大规模临床试验。本文介绍了几种潜在的可用于罕见病的小样本统计设计，包括交叉设计、自身多阶段交叉设计、随机安慰剂阶段设计、随机撤药设计、成组序贯设计、适应性设计等，讨论这些设计的优缺点及应用场景，进一步讨论了Bayes决策在罕见病临床试验中的优势。旨在为罕见病小样本临床试验的设计和实施提供参考。 |
| 英文摘要: |
| 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, n-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. |
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