Gardner Family Center for Parkinson's Disease and Movement Disorders, Department of Neurology, University of Cincinnati, Cincinnati, OH, USA; Department of Neuroscience "Rita Levi Montalcini", University of Turin, via Cherasco 15, 10125, Torino, Italy.
Gardner Family Center for Parkinson's Disease and Movement Disorders, Department of Neurology, University of Cincinnati, Cincinnati, OH, USA.
Parkinsonism Relat Disord. 2018 Jan;46 Suppl 1(Suppl 1):S53-S56. doi: 10.1016/j.parkreldis.2017.07.022. Epub 2017 Jul 26.
We sought to review the landscape of past, present, and future use of technology-based outcome measures (TOMs) in clinical trials of neurodegenerative disorders.
We systematically reviewed PubMed and ClinicalTrials.gov for published and ongoing clinical trials in neurodegenerative disorders employing TOMs. In addition, medical directors of selected pharmaceutical companies were surveyed on their companies' ongoing efforts and future plans to integrate TOMs in clinical trials as primary, secondary, or exploratory endpoints.
We identified 164 published clinical trials indexed in PubMed that used TOMs as outcome measures in Parkinson disease (n = 132) or other neurodegenerative disorders (n = 32). The ClinicalTrials.gov search yielded 42 clinical trials using TOMs, representing 2.7% of ongoing trials. Sensor-based technology accounted for over 75% of TOMs applied. Gait and physical activity were the most common targeted domains. Within the next 5 years, 83% of surveyed pharmaceutical companies engaged in neurodegenerative disorders plan to deploy TOMs in clinical trials.
Although promising, TOMs are underutilized in clinical trials of neurodegenerative disorders. Validating relevant endpoints, standardizing measures and procedures, establishing a single platform for integration of data and algorithms from different devices, and facilitating regulatory approvals should advance TOMs integration into clinical trials.
我们旨在回顾神经退行性疾病临床试验中基于技术的结局测量（TOMs）的过去、现在和未来使用情况。
我们系统地检索了 PubMed 和 ClinicalTrials.gov，以获取发表和正在进行的神经退行性疾病使用 TOMs 的临床试验。此外，还对选定制药公司的医学主任进行了调查，了解他们公司在将 TOMs 作为主要、次要或探索性终点纳入临床试验方面的当前努力和未来计划。
我们在 PubMed 中确定了 164 项索引的已发表临床试验，这些试验将 TOMs 用作帕金森病（n=132）或其他神经退行性疾病（n=32）的结局测量指标。ClinicalTrials.gov 的搜索结果显示，有 42 项临床试验使用了 TOMs，占正在进行的临床试验的 2.7%。基于传感器的技术占应用 TOMs 的 75%以上。步态和身体活动是最常见的目标领域。在未来 5 年内，83%参与神经退行性疾病的调查制药公司计划在临床试验中部署 TOMs。
尽管很有前景，但 TOMs 在神经退行性疾病临床试验中的应用仍不充分。验证相关终点、标准化测量和程序、建立单一平台以整合来自不同设备的数据和算法，以及促进监管批准，应能促进 TOMs 纳入临床试验。
