Statistical design and application of clinical trials with small sample sizes for rare diseases
10.3760/cma.j.cn112338-20240705-00401
- VernacularTitle:罕见病小样本临床试验的统计设计及其应用
- Author:
Yuan GAO
1
;
Zihang ZHONG
;
Mengyi LU
;
Feng CHEN
Author Information
1. 南京医科大学公共卫生学院生物统计学系，南京 211166
- Keywords:
Rare diseases;
Small sample;
Statistical design;
Clinical trial
- From:
Chinese Journal of Epidemiology
2024;45(12):1700-1704
- CountryChina
- Language:Chinese
-
Abstract:
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.
