Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science.
Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science.
复制标题
DOI：
10.1159/000512500
复制
发表时间：
2020-01-01
影响因子：
--
通讯作者：
Zach, Neta
中科院分区：
文献类型：
--
作者：
Stephenson, Diane;Alexander, Robert;Zach, Neta
迫切需要创新的工具来加速新疗法的评估和随后的批准，这些新疗法可能会减缓、停止或逆转帕金森病（PD）的无情进展。早期干预疾病连续体的治疗是药物开发管道中许多候选药物的优先事项。缺乏敏感和客观的，但临床上可解释的措施，可以捕捉疾病的有意义的方面。这对新疗法的开发提出了重大挑战，并且由于患者之间临床表现的相当大的异质性以及PD的许多体征和症状的波动性而变得更加复杂。数字健康技术（DHT），如智能手机应用程序，可穿戴传感器和数字日记，有可能通过在自然生活环境中客观，远程和频繁地测量PD体征和症状来解决许多这些差距。当前COVID-19大流行的气候为有效实施此类战略带来了更大的紧迫感。为了在药物开发研究中采用这些技术，需要就实施适当技术的最佳实践达成与监管一致的共识，包括收集、处理和解释数字传感器数据。越来越多的合作计划正在启动，以确定有效的方法，以促进在PD临床试验中使用DHT。关键路径研究所的帕金森氏症联盟的关键路径被强调为一个案例，利益相关者共同参与监管机构在PD临床试验中有效使用DHT。包括美国食品药品监督管理局和欧洲药品管理局在内的全球监管机构正在鼓励通过多利益相关者联盟提高数据驱动参与的效率。为此，我们回顾了如何通过调整知识，专业知识和数据共享的方式，最大限度地提高效率，可以最有效地实现DHT的进步。
Innovative tools are urgently needed to accelerate the evaluation and subsequent approval of novel treatments that may slow, halt, or reverse the relentless progression of Parkinson disease (PD). Therapies that intervene early in the disease continuum are a priority for the many candidates in the drug development pipeline. There is a paucity of sensitive and objective, yet clinically interpretable, measures that can capture meaningful aspects of the disease. This poses a major challenge for the development of new therapies and is compounded by the considerable heterogeneity in clinical manifestations across patients and the fluctuating nature of many signs and symptoms of PD. Digital health technologies (DHT), such as smartphone applications, wearable sensors, and digital diaries, have the potential to address many of these gaps by enabling the objective, remote, and frequent measurement of PD signs and symptoms in natural living environments. The current climate of the COVID-19 pandemic creates a heightened sense of urgency for effective implementation of such strategies. In order for these technologies to be adopted in drug development studies, a regulatory-aligned consensus on best practices in implementing appropriate technologies, including the collection, processing, and interpretation of digital sensor data, is required. A growing number of collaborative initiatives are being launched to identify effective ways to advance the use of DHT in PD clinical trials. The Critical Path for Parkinson's Consortium of the Critical Path Institute is highlighted as a case example where stakeholders collectively engaged regulatory agencies on the effective use of DHT in PD clinical trials. Global regulatory agencies, including the US Food and Drug Administration and the European Medicines Agency, are encouraging the efficiencies of data-driven engagements through multistakeholder consortia. To this end, we review how the advancement of DHT can be most effectively achieved by aligning knowledge, expertise, and data sharing in ways that maximize efficiencies.