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引用次数: 0
摘要
传统的双臂随机试验设计在确定医疗干预措施的疗效方面发挥了关键作用。然而,当面临多种实验治疗或资源有限时,其效率往往会大打折扣。为应对这些挑战,多臂多阶段设计应运而生,可在一次试验中同时评估多种治疗方法。在这种方法中,如果某一治疗臂在中期阶段达到疗效成功标准,整个试验就会停止,并选择该治疗臂进行进一步研究。然而,当多个治疗方案同时进行时,如果在一个治疗方案取得成功时停止试验,就会降低选择最佳治疗方案的概率。为了解决这个问题,我们开发了一种分组顺序多臂多阶段生存试验设计,其中包含针对特定臂的停止规则。所提出的方法能有效控制族式 I 型误差,并能高概率地选择最佳治疗臂。
Multi-arm multi-stage survival trial design with arm-specific stopping rule.
Traditional two-arm randomized trial designs have played a pivotal role in establishing the efficacy of medical interventions. However, their efficiency is often compromised when confronted with multiple experimental treatments or limited resources. In response to these challenges, the multi-arm multi-stage designs have emerged, enabling the simultaneous evaluation of multiple treatments within a single trial. In such an approach, if an arm meets efficacy success criteria at an interim stage, the whole trial stops and the arm is selected for further study. However when multiple treatment arms are active, stopping the trial at the moment one arm achieves success diminishes the probability of selecting the best arm. To address this issue, we have developed a group sequential multi-arm multi-stage survival trial design with an arm-specific stopping rule. The proposed method controls the familywise type I error in a strong sense and selects the best promising treatment arm with a high probability.
期刊介绍:
The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers:
Drug, device, and biological research and development;
Drug screening and drug design;
Assessment of pharmacological activity;
Pharmaceutical formulation and scale-up;
Preclinical safety assessment;
Bioavailability, bioequivalence, and pharmacokinetics;
Phase, I, II, and III clinical development including complex innovative designs;
Premarket approval assessment of clinical safety;
Postmarketing surveillance;
Big data and artificial intelligence and applications.