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Prioritisation and design of clinical trials
European Journal of Epidemiology ( IF 7.7 ) Pub Date : 2021-06-06 , DOI: 10.1007/s10654-021-00761-5
Anna Heath 1, 2, 3 , M G Myriam Hunink 4, 5, 6, 7 , Eline Krijkamp 4, 6 , Petros Pechlivanoglou 1, 8
Affiliation  

Clinical trials require participation of numerous patients, enormous research resources and substantial public funding. Time-consuming trials lead to delayed implementation of beneficial interventions and to reduced benefit to patients. This manuscript discusses two methods for the allocation of research resources and reviews a framework for prioritisation and design of clinical trials. The traditional error-driven approach of clinical trial design controls for type I and II errors. However, controlling for those statistical errors has limited relevance to policy makers. Therefore, this error-driven approach can be inefficient, waste research resources and lead to research with limited impact on daily practice. The novel value-driven approach assesses the currently available evidence and focuses on designing clinical trials that directly inform policy and treatment decisions. Estimating the net value of collecting further information, prior to undertaking a trial, informs a decision maker whether a clinical or health policy decision can be made with current information or if collection of extra evidence is justified. Additionally, estimating the net value of new information guides study design, data collection choices, and sample size estimation. The value-driven approach ensures the efficient use of research resources, reduces unnecessary burden to trial participants, and accelerates implementation of beneficial healthcare interventions.



中文翻译:

临床试验的优先顺序和设计

临床试验需要大量患者的参与、巨大的研究资源和大量的公共资金。耗时的试验会导致有益干预措施的实施延迟,并降低对患者的益处。本手稿讨论了两种分配研究资源的方法,并回顾了临床试验的优先次序和设计框架。临床试验设计的传统错误驱动方法控制 I 型和 II 型错误。然而,控制这些统计误差与政策制定者的相关性有限。因此,这种错误驱动的方法可能效率低下,浪费研究资源并导致对日常实践影响有限的研究。新颖的价值驱动方法评估当前可用的证据,并专注于设计直接为政策和治疗决策提供信息的临床试验。在进行试验之前估计收集进一步信息的净值,可以告知决策者是否可以利用当前信息做出临床或健康政策决定,或者是否有理由收集额外的证据。此外,估计新信息的净值指导研究设计、数据收集选择和样本量估计。价值驱动的方法确保了研究资源的有效利用,减少了试验参与者的不必要负担,并加速了有益的医疗干预措施的实施。告知决策者是否可以利用当前信息做出临床或健康政策决定,或者是否有理由收集额外证据。此外,估计新信息的净值指导研究设计、数据收集选择和样本量估计。价值驱动的方法确保了研究资源的有效利用,减少了试验参与者的不必要负担,并加速了有益的医疗干预措施的实施。告知决策者是否可以利用当前信息做出临床或健康政策决定,或者是否有理由收集额外证据。此外,估计新信息的净值指导研究设计、数据收集选择和样本量估计。价值驱动的方法确保了研究资源的有效利用,减少了试验参与者的不必要负担,并加速了有益的医疗干预措施的实施。

更新日期:2021-06-07
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