Department of Neurology, University of Missouri, Columbia, Missouri.
Department of Neurology and Neurology Clinical Trials Unit, Massachusetts General Hospital, Boston, Massachusetts.
Muscle Nerve. 2020 Sep;62(3):321-326. doi: 10.1002/mus.26921. Epub 2020 Jun 8.
Amyotrophic lateral sclerosis (ALS) has the largest drug pipeline among neuromuscular diseases, with over 160 companies actively involved in ALS research. There is a growing need to recruit trial participants, but ALS patients often have limited mobility and most ALS trials are conducted in a small number of major centers. These factors effectively limit patient participation, particularly for those in rural areas. The current coronavirus disease 2019 (COVID-19) pandemic has necessitated the more widespread use of telemedicine technology for clinical care, and has prompted consideration of its increased use for clinical trials. In this opinion piece, we describe the current state of telemedicine for recruitment, consenting, and screening of participants for clinical trials. We also summarize the available data on remote administration of outcome measures. Current challenges include validation of outcome measures for remote assessment, as well as technological, regulatory, and licensure barriers.
肌萎缩侧索硬化症(ALS)是神经肌肉疾病中药物研发管线最丰富的疾病,有超过 160 家公司积极从事 ALS 研究。招募试验参与者的需求日益增长,但 ALS 患者通常行动不便,而且大多数 ALS 试验都在少数几个主要中心进行。这些因素实际上限制了患者的参与,尤其是那些农村地区的患者。当前的 2019 冠状病毒病(COVID-19)大流行需要更广泛地使用远程医疗技术进行临床护理,并促使人们考虑增加其在临床试验中的使用。在这篇观点文章中,我们描述了远程医疗在临床试验参与者招募、知情同意和筛选方面的现状。我们还总结了远程管理结果测量的现有数据。目前的挑战包括远程评估的结果测量的验证,以及技术、监管和许可方面的障碍。