Eli Lilly and Company, Indianapolis, Indiana, USA.
Cogstate Ltd, London, UK.
Drug Discov Today. 2021 May;26(5):1330-1336. doi: 10.1016/j.drudis.2021.01.012. Epub 2021 Jan 21.
Go/No-Go decision making in early phase clinical trials is challenging for drug developers working in Alzheimer's disease. Recent negative trial results have been attributed to a lack of efficacy and important safety concerns. Furthermore, demonstrated target engagement has rarely translated into demonstrable clinical efficacy. Cognitive data might provide valuable insights at various points during drug development, and a thoughtful and robust set of decision-making criteria, specified a priori, can and should be applied under many circumstances. This review provides insights into how to utilize cognitive data for Go/No-Go decisions, with an emphasis on how these cognitive criteria differ depending on the context (e.g., stage of development, mechanism of action and trial design).
在阿尔茨海默病药物研发领域，对于早期临床试验中的“行/不行”决策制定来说是极具挑战性的。近期的阴性试验结果归因于缺乏疗效和重要的安全性问题。此外，已证明的靶标确证很少转化为可证明的临床疗效。认知数据在药物开发的各个阶段都可能提供有价值的见解，并且可以并应该在许多情况下应用事先指定的深思熟虑且稳健的决策标准集。本综述提供了如何利用认知数据进行“行/不行”决策的见解，重点介绍了这些认知标准如何根据具体情况（例如，开发阶段、作用机制和试验设计）而有所不同。
