Novel statistical approaches for basket trials in ageing-related diseases

目的：我研究的目的将是探索分析篮子试验的新的统计方法，重点是与慢性衰老相关的疾病。例如，需要为免疫介导性炎症性疾病(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途径，而且由于许多疾病是在晚年发展起来的，随着世界人口的老龄化，IMID的数量正在增加。试验设计和分析的新方法可以大大节省时间、金钱和资源，因为很大一部分IMID患者对治疗无效或在成功治疗后最终复发(Grayling等人，2021年)。在篮子试验框架内，有可能设计一项有效的研究，以调查各种IMID的特定治疗方法。目标1.开发新的贝叶斯方法，以便在纵向篮子研究中的子试验之间借用信息。评估对典型分析的统计特性的影响，并提出更好的分析方法，在患者有资格参加多个子试验的情况下，或者存在潜在的患者错误分类的情况。开发在子试验之间共享信息时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