Center for Medical Data Science, Medical University of Vienna, Vienna, Austria.
Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ, United States of America.
PLoS One. 2023 Mar 9;18(3):e0281674. doi: 10.1371/journal.pone.0281674. eCollection 2023.
Non-alcoholic steatohepatitis (NASH) is the progressive form of nonalcoholic fatty liver disease (NAFLD) and a disease with high unmet medical need. Platform trials provide great benefits for sponsors and trial participants in terms of accelerating drug development programs. In this article, we describe some of the activities of the EU-PEARL consortium (EU Patient-cEntric clinicAl tRial pLatforms) regarding the use of platform trials in NASH, in particular the proposed trial design, decision rules and simulation results. For a set of assumptions, we present the results of a simulation study recently discussed with two health authorities and the learnings from these meetings from a trial design perspective. Since the proposed design uses co-primary binary endpoints, we furthermore discuss the different options and practical considerations for simulating correlated binary endpoints.
非酒精性脂肪性肝炎（NASH）是一种进行性非酒精性脂肪性肝病（NAFLD），且存在高度未满足的医学需求。平台试验在加速药物开发计划方面为赞助商和试验参与者带来了巨大的好处。在本文中，我们描述了欧盟-PEARL 联盟（EU Patient-cEntric clinicAl tRial pLatforms）在 NASH 中使用平台试验的一些活动，特别是拟议的试验设计、决策规则和模拟结果。针对一组假设，我们展示了最近与两个卫生当局讨论的模拟研究结果，以及从试验设计角度出发从这些会议中获得的经验教训。由于拟议的设计使用了共同主要的二分类终点，我们进一步讨论了模拟相关二分类终点的不同选项和实际考虑因素。
