Sitbon Olivier, Nikkho Sylvia, Benza Raymond, Cq Deng Chunqin, W Farber Harrison, Gomberg-Maitland Mardi, Hassoun Paul, Meier Christian, Pepke-Zaba Joanna, Prasad Krishna, Seeger Werner, Corris Paul A
Assistance Publique-Hôpitaux de Paris, Service de Pneumologie, Hôpital Bicêtre, Université Paris-Sud, Laboratoire d'Excellence en Recherche sur le Médicament et Innovation Thérapeutique, Le Kremlin-Bicêtre, France.
Bayer AG, Clinical Development, Berlin, Germany.
Pulm Circ. 2020 Nov 18;10(4):2045894020962960. doi: 10.1177/2045894020962960. eCollection 2020 Oct-Dec.
This manuscript on endpoints incorporates the broad experience of members of Pulmonary Vascular Research Institute's Innovative Drug Development Initiative as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in pulmonary hypertension. It reviews our current understanding of endpoints used in phase 2 and 3 trials for pulmonary hypertension and discusses in detail the value of newer approaches. These include the roles of composite endpoints and how these can be developed and validated. The newer concept of risk analysis is also discussed, including how such risk scores might be utilised as endpoints in clinical trials.
这份关于终点指标的手稿融合了肺血管研究所创新药物开发倡议成员的广泛经验，该倡议是一个供学术界、制药行业和监管专家围绕未来肺动脉高压临床试验设计展开公开辩论的平台。它回顾了我们目前对肺动脉高压2期和3期试验中使用的终点指标的理解，并详细讨论了新方法的价值。这些方法包括复合终点指标的作用以及如何开发和验证这些指标。还讨论了风险分析这一新概念，包括此类风险评分如何可被用作临床试验中的终点指标。
