{"title":"[Statistical design and application of clinical trials with small sample sizes for rare diseases].","authors":"Y Gao, Z H Zhong, M Y Lu, F Chen","doi":"10.3760/cma.j.cn112338-20240705-00401","DOIUrl":null,"url":null,"abstract":"<p><p>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, <i>n</i>-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.</p>","PeriodicalId":23968,"journal":{"name":"中华流行病学杂志","volume":"45 12","pages":"1700-1704"},"PeriodicalIF":0.0000,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"中华流行病学杂志","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3760/cma.j.cn112338-20240705-00401","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0
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.
期刊介绍：
Chinese Journal of Epidemiology, established in 1981, is an advanced academic periodical in epidemiology and related disciplines in China, which, according to the principle of integrating theory with practice, mainly reports the major progress in epidemiological research. The columns of the journal include commentary, expert forum, original article, field investigation, disease surveillance, laboratory research, clinical epidemiology, basic theory or method and review, etc.
The journal is included by more than ten major biomedical databases and index systems worldwide, such as been indexed in Scopus, PubMed/MEDLINE, PubMed Central (PMC), Europe PubMed Central, Embase, Chemical Abstract, Chinese Science and Technology Paper and Citation Database (CSTPCD), Chinese core journal essentials overview, Chinese Science Citation Database (CSCD) core database, Chinese Biological Medical Disc (CBMdisc), and Chinese Medical Citation Index (CMCI), etc. It is one of the core academic journals and carefully selected core journals in preventive and basic medicine in China.
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