Leyens Lada, Northcott Carrie A, Maloney Lesley, McCarthy Marie, Dokuzova Nona, Pfister Thomas
Product Development Regulatory, Hoffmann-La Roche, Basel, Switzerland.
Digital Sciences and Translational Imaging, Pfizer, Inc., Cambridge, MA, USA.
Digit Biomark. 2024 Jan 11;8(1):1-12. doi: 10.1159/000534954. eCollection 2024 Jan-Dec.
BACKGROUND: Developments in the field of digital measures and digitally derived endpoints demand greater attention on globally aligned approaches to enhance digital measure acceptance by regulatory authorities and health technology assessment (HTA) bodies for decision-making. In order to maximize the value of digital measures in global drug development programs and to ensure study teams and regulators are referring to the same items, greater alignment of concepts, definitions, and terminology is required. This is a fast-moving complex field; every day brings new technologies, algorithms, and possibilities. A common language is particularly important when working in multifunctional teams to ensure that there is a clear understanding of what is meant and understood. SUMMARY: In the paper, the EFPIA digital endpoint joint subgroup reviews the challenges facing teams working to advance digital endpoints, where different terms are used to describe the same things, where common terms such as "monitoring" have significantly different meaning for different regulatory agencies, where the preface "e" to denote electronic is still used in some contexts, but the term "digital" is used in other, and where there is significant confusion as to what is understood by "raw" when it comes to data derived from digital health technologies. KEY MESSAGE: The EFPIA subgroup is calling for an aligned lexicon. Alignment provides a more predictable path for development, validation, and use of the tools and measures used to collect digital endpoints supporting standardization and consistency in this new field of research, with the goal of increasing regulatory and payer harmonization and acceptance.
背景:数字测量和数字衍生终点领域的发展需要更加关注全球统一的方法,以提高监管机构和卫生技术评估(HTA)机构对数字测量用于决策的接受度。为了在全球药物开发项目中最大化数字测量的价值,并确保研究团队和监管机构所指的是相同的项目,需要在概念、定义和术语上实现更大程度的统一。这是一个快速发展的复杂领域;每天都有新技术、算法和可能性出现。在跨职能团队工作时,通用语言尤为重要,以确保对所指和所理解的内容有清晰的认识。 总结:在本文中,欧洲制药工业协会联合会(EFPIA)数字终点联合小组回顾了致力于推进数字终点的团队所面临的挑战,包括不同术语用于描述相同事物的情况;诸如“监测”等常用术语对不同监管机构有显著不同含义的情况;在某些情况下仍使用前缀“e”表示电子,但在其他情况下使用“数字”一词的情况;以及在涉及源自数字健康技术的数据时,对于“原始”数据的理解存在重大混淆的情况。 关键信息:EFPIA小组呼吁制定统一词汇表。统一为用于收集支持这一新兴研究领域标准化和一致性的数字终点的工具和测量方法的开发、验证和使用提供了更可预测的路径,目标是提高监管和支付方的协调性与接受度。
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