Children's Hospital of Eastern Ontario Research Institute, Ottawa, Canada.
Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada.
Orphanet J Rare Dis. 2021 Mar 20;16(1):141. doi: 10.1186/s13023-021-01687-7.
It has recently been suggested that registries for rare neuromuscular diseases should be formed and governed exclusively by physicians and patients in an effort to limit conflicts of interest. Enacting such an approach would not only be challenging logistically and financially, but it would also exclude the involvement of sponsors, who are an integral component of drug development within the current compliance framework. Therefore, as an alternative to traditional registries, we propose the use of a better collaborative model for post-marketing follow-up that includes all stakeholders. We developed the concept of Disease Monitoring Programs (DMPs), which are designed to monitor disease manifestations over a 10-year period whether on a sponsored drug or not, and ensure consistent collection, ownership sharing and governance of data.
最近有人建议，应该成立专门由医生和患者管理的罕见神经肌肉疾病登记处，以努力限制利益冲突。这样做不仅在后勤和财务上具有挑战性，而且还会排除赞助商的参与，而赞助商是当前合规框架内药物开发的一个组成部分。因此，作为传统登记处的替代方案，我们建议使用更好的、包括所有利益相关者的上市后随访协作模式。我们提出了疾病监测计划（Disease Monitoring Programs，DMPs）的概念，旨在监测 10 年内的疾病表现，无论是否在受赞助的药物上，以确保数据的一致收集、所有权共享和治理。
