Brem Anna-Katharine, Kuruppu Sajini, de Boer Casper, Muurling Marijn, Diaz-Ponce Ana, Gove Dianne, Curcic Jelena, Pilotto Andrea, Ng Wan-Fai, Cummins Nicholas, Malzbender Kristina, Nies Vera J M, Erdemli Gul, Graeber Johanna, Narayan Vaibhav A, Rochester Lynn, Maetzler Walter, Aarsland Dag
Department of Old Age Psychiatry, King's College London, Institute of Psychiatry, Psychology and Neuroscience, London, United Kingdom.
University Hospital of Old Age Psychiatry, University of Bern, Bern, Switzerland.
Front Neurol. 2023 Jun 15;14:1210974. doi: 10.3389/fneur.2023.1210974. eCollection 2023.
Alzheimer's disease (AD) and other neurodegenerative diseases such as Parkinson's disease (PD) and Huntington's disease (HD) are associated with progressive cognitive, motor, affective and consequently functional decline considerably affecting Activities of Daily Living (ADL) and quality of life. Standard assessments, such as questionnaires and interviews, cognitive testing, and mobility assessments, lack sensitivity, especially in early stages of neurodegenerative diseases and in the disease progression, and have therefore a limited utility as outcome measurements in clinical trials. Major advances in the last decade in digital technologies have opened a window of opportunity to introduce digital endpoints into clinical trials that can reform the assessment and tracking of neurodegenerative symptoms. The Innovative Health Initiative (IMI)-funded projects RADAR-AD (), IDEA-FAST () and Mobilise-D () aim to identify digital endpoints relevant for neurodegenerative diseases that provide reliable, objective, and sensitive evaluation of disability and health-related quality of life. In this article, we will draw from the findings and experiences of the different IMI projects in discussing (1) the value of remote technologies to assess neurodegenerative diseases; (2) feasibility, acceptability and usability of digital assessments; (3) challenges related to the use of digital tools; (4) public involvement and the implementation of patient advisory boards; (5) regulatory learnings; and (6) the significance of inter-project exchange and data- and algorithm-sharing.
阿尔茨海默病（AD）以及其他神经退行性疾病，如帕金森病（PD）和亨廷顿病（HD），与认知、运动、情感功能的进行性衰退相关，进而严重影响日常生活活动（ADL）和生活质量。标准评估，如问卷调查、访谈、认知测试和运动能力评估，缺乏敏感性，尤其是在神经退行性疾病的早期阶段和疾病进展过程中，因此在临床试验中作为疗效测量指标的效用有限。过去十年数字技术的重大进展为将数字终点引入临床试验打开了一扇机会之窗，这可以改革神经退行性症状的评估和跟踪。由创新健康倡议（IMI）资助的项目RADAR-AD（）、IDEA-FAST（）和Mobilise-D（）旨在确定与神经退行性疾病相关的数字终点，这些终点能够对残疾和健康相关生活质量提供可靠、客观和敏感的评估。在本文中，我们将借鉴不同IMI项目的研究结果和经验，讨论（1）远程技术在评估神经退行性疾病方面的价值；（2）数字评估的可行性、可接受性和可用性；（3）与使用数字工具相关的挑战；（4）公众参与和患者咨询委员会的实施；（5）监管经验；以及（6）项目间交流以及数据和算法共享的重要性。
