引入贝叶斯方法的篮式试验设计探讨及在中医药领域的应用价值  

Exploration of basket trial design with Bayesian method and its application value in traditional Chinese medicine

作　　者：王思村 李牧之 党海霞[2] 顾浩 刘骏[1] 王忠[1] 于亚南[1] WANG Si-cun;LI Mu-zhi;DANG Hai-xia;GU Hao;LIU Jun;WANG Zhong;YU Ya-nan(Institute of Basic Research in Clinical Medicine,China Academy of Chinese Medical Sciences,Beijing 100700,China;Experimental Research Center,China Academy of Chinese Medical Sciences,Bejing 100700,China;National Data Center,China Academy of Chinese Medical Sciences,Beijing 100700,China)

机构地区：[1]中国中医科学院中医临床基础医学研究所,北京100700 [2]中国中医科学院中医药发展研究中心,北京100700 [3]中国中医科学院中医药数据中心,北京100700

出　　处：《中国中药杂志》2025年第3期846-852,共7页China Journal of Chinese Materia Medica

基　　金：中国中医科学院科技创新工程项目(CI2021A04707);中国中医科学院基本科研业务费优秀青年科技人才(创新类)培养专项(ZZ13-YQ-076);国家自然科学基金项目(82105052)。

摘　　要：篮式试验作为一种创新性临床试验设计理念,标志着医学研究从传统的大规模、单一疾病治疗模式向精准、个体化治疗模式的转变。这一设计在发展过程中逐步融合了贝叶斯理念,使得试验设计更加科学、合理,且提高了临床试验效率。贝叶斯方法的核心在于利用先验知识和新的观测数据,动态地更新后验概率。这种灵活性使得篮式试验能够更有效地适应研究过程中的变化,不仅能够帮助研究者根据累积的数据动态调整研究策略,还能提高对治疗反应的预测能力。此外,篮式试验的设计理念与中医“异病同治”的理念存在异曲同工之妙。“异病同治”强调的是在特定条件下,不同的疾病可能具有相同的治疗方法。类似地,篮式试验允许对多种疾病使用相同的试验设计,这在中医药领域,尤其是以证候为统领的以证统病研究模式更具可操作性和实践价值。通过引入篮式试验,中医药临床研究设计将更加科学化,从而提供高质量的中医药临床证据。该研究系统梳理了篮式试验中不同贝叶斯方法及模型,分析其优缺点及应用场景等,以期为中医药领域篮式研究提供可行性参考。Basket trial,as an innovative clinical trial design concept,marks the transformation of medical research from the traditional large-scale and single-disease treatment to the precise and individualized treatment.By gradually incorporating the Bayesian method during development,the trial design becomes more scientific and reasonable and increases its efficiency.The fundamental principle of the Bayesian method is the utilization of prior knowledge in conjunction with new observational data to dynamically update the posterior probability.This flexibility enhances the basket trial′s capacity to effectively adapt to variations during the research process.Consequently,it enables researchers to dynamically adjust research strategies based on accumulated data and improve the predictive accuracy regarding treatment responses.In addition,the design concept of the basket trial aligns with the traditional Chinese medicine(TCM)principle of"homotherapy for heteropathy".The principle of"homotherapy for heteropathy"emphasizes that under certain conditions,different diseases may have the same treatment.Similarly,basket trials allow using a uniform trial design across multiple diseases,offering enhanced operational and significant practical value in the realm of TCM,particularly within the context of syndrome-based disease research.By introducing basket trials,the design of TCM clinical studies will be more scientific and yield higher-quality evidence.This study systematically categorized various Bayesian methods and models utilized in basket trials,evaluated their strengths and weaknesses,and identified their appropriate application contexts,so as to offer a practical guide for designing basket trials in the realm of TCM.

关 键 词：贝叶斯方法 篮式试验 中医药 

分 类 号：R2-03[医药卫生—中医学]