Lanius Vivian, Glocker Barbara, Lösch Christian, Bratton Daniel J, Callegari Francesca, Wright Melanie, Rajamäki Suvi
Statistical Innovation, UCB Biosciences GmbH, Monheim am Rhein, Germany.
Clinical Development, Operations and Medical Writing, Bayer AG, Berlin, Germany.
Trials. 2025 Jul 11;26(1):241. doi: 10.1186/s13063-025-08915-6.
Reports of the results of clinical studies are integral to regulatory decision making. They are used to support marketing authorization, to substantiate labeling information, and to inform academic publications, trial postings, and promotional messages intended to communicate study results to doctors and patients. Therefore, beyond summarizing the study design, methods, and data, study reports should provide clear descriptions of the benefits and risks of an intervention for a given medical condition. The ICH E9(R1) addendum on estimands and sensitivity analysis in clinical trials provides a framework aiming for more clarity about the reported "treatment effects." In practice, stakeholders are still learning how to embrace the estimand framework and how it impacts the reporting of study results in the most value-adding manner. This paper provides recommendations and considerations for implementing the estimand framework in the reporting of results to realize its full potential of increased transparency for interpretation and decision-making. These recommendations are based on practical experiences of working with clinical trial teams through the reporting process following implementation of the estimand framework in the protocol. As illustrated by two case studies, the primary application is in reporting studies for the scientific evaluation of medicines by regulatory agencies, directly impacting clinical study reports and submission documents, and will also extend to publications in scientific journals.
临床研究结果报告是监管决策的重要组成部分。它们用于支持上市许可、证实标签信息,并为学术出版物、试验发布以及旨在向医生和患者传达研究结果的宣传信息提供依据。因此,除了总结研究设计、方法和数据外,研究报告还应清晰描述针对特定医疗状况的干预措施的益处和风险。国际人用药品注册技术协调会(ICH)关于临床试验中估计量和敏感性分析的E9(R1)附录提供了一个框架,旨在使所报告的“治疗效果”更加清晰。在实践中,利益相关者仍在学习如何采用估计量框架,以及如何以最具增值性的方式影响研究结果的报告。本文就如何在结果报告中实施估计量框架提出建议和注意事项,以充分发挥其在提高解释和决策透明度方面的潜力。这些建议基于在方案中实施估计量框架后,在报告过程中与临床试验团队合作的实际经验。如两个案例研究所示,其主要应用在于为监管机构对药品进行科学评估的报告研究,直接影响临床研究报告和提交文件,并且还将扩展到科学期刊上的出版物。
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