Novel statistical approaches for basket trials in ageing-related diseases

目的我的研究的目的是探讨新的统计方法来分析篮子试验，重点是慢性衰老相关疾病。例如，需要为免疫介导的炎症性疾病（IMID）开发创新的试验方法。IMID由许多不同的疾病组成，这些疾病具有共同的炎症通路，并且由于许多疾病是在生命后期发生的，因此随着世界人口的老龄化而增加。试验设计和分析的新方法可以节省大量的时间、金钱和资源，因为大部分IMID患者对治疗没有反应或在成功治疗后最终复发（Grayling et al.，2021年）。在篮子试验框架内，有可能设计一项有效的研究，以研究各种IMID的特定治疗。目标1.开发新的贝叶斯方法，在纵向篮子研究中的子试验之间借用信息。在患者符合多个子试验条件或存在潜在的患者错误分类的情况下，评估典型分析对统计特性的影响，并提出更好的分析方法。在子试验之间共享信息时，开发控制错误率的新方法

AimThe aim of my research will be to investigate new statistical methods for the analysis of basket trials, with a focus on chronic ageing related diseases. For example, there is a need for innovative trial methods to be developed for Immune Mediated Inflammatory Diseases (IMIDs). IMIDs consist of many distinct conditions that share common inflammatory 6pathways, and since many develop later in life, are increasing as the world population ages. Novel approaches to trial design and analysis could lead to substantial savings in time, money, and resources, since a large proportion of IMID patients fail to respond to treatment or eventually relapse after successful treatment (Grayling et al., 2021). Within the basket trial framework, it could be possible to design an efficient study to investigate a particular treatment across a wide variety of IMIDs. Objectives1. Develop novel Bayesian methods to borrow information between subtrials within a longitudinal basket study.2. To assess the implications on the statistical properties of typical analyses and propose better analytical methods, in the case where patients are eligible for multiple sub-trials, or there is potential misclassification of patients.3. Develop novel methods for controlling the error rate when sharing information between subtrials