BARDS, MSD, P.R.C, Beijing, China.
BARDS, MSD, Zurich, Switzerland.
J Biopharm Stat. 2023 Jul 4;33(4):452-465. doi: 10.1080/10543406.2023.2170396. Epub 2023 Feb 8.
ICH E9(R1) introduces the estimand framework to strengthen dialogues between sponsors and regulators during drug development. A well-structured benefit-risk assessment (BRA) framework also intends to facilitate communication among stakeholders. However, the estimand in ICH E9(R1) is written mainly from the perspective of a single measure of treatment effect in clinical trials. There is lack of systematic discussion on estimand in the context of BRA. This paper initiates the BRA discussion under the estimand framework. By identifying two types of BRA approaches, we summarize and discuss completed clinical trials, using the estimand language for BRA. Benefits and challenges of using estimand for BRA are also discussed.
ICH E9(R1) 引入了评估目标框架，以加强药物开发过程中申办方和监管机构之间的对话。一个结构良好的获益-风险评估（BRA）框架也旨在促进利益相关者之间的沟通。然而，ICH E9(R1) 中的评估目标主要是从临床试验中单一治疗效果衡量指标的角度来撰写的。在 BRA 背景下，对于评估目标缺乏系统的讨论。本文在评估目标框架下启动了 BRA 讨论。通过确定两种 BRA 方法类型，我们使用 BRA 的评估目标语言对已完成的临床试验进行了总结和讨论。还讨论了使用评估目标进行 BRA 的益处和挑战。
