Max-Delbrueck Center for Translational Medicine in the Helmholtz Association, Berlin, Germany; European Medicines Agency, London, UK.
Committee of Orphan Medicinal Products, European Medicines Agency, London, UK; College ter Beoordeling van Geneesmiddelen, Utrecht, The Netherlands.
Drug Discov Today. 2018 Jan;23(1):26-48. doi: 10.1016/j.drudis.2017.09.015. Epub 2017 Oct 4.
Here, we provide an in-depth literature and experience-based review of nonclinical models and data used to support orphan medicinal product designations (OMPDs) in rare neurodegenerative conditions. The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency updates its assessment processes based on scientific progress and aims to provide transparent criteria required in support of OMPDs. Thus, we also provide an updated analysis of existing nonclinical models in selected conditions and identify key features of nonclinical studies that are crucial for the support of OMPDs. This could not only inform future drug development in rare neurological conditions, but also indicate areas where the use of nonclinical models can be made more efficient.
在这里，我们提供了一份深入的文献和经验综述，介绍了用于支持罕见神经退行性疾病孤儿药设计（OMPD）的非临床模型和数据。欧洲药品管理局孤儿药委员会（COMP）根据科学进展更新其评估流程，并旨在提供支持 OMPD 所需的透明标准。因此，我们还对选定条件下现有的非临床模型进行了更新分析，并确定了支持 OMPD 的非临床研究的关键特征。这不仅可以为罕见神经疾病的未来药物开发提供信息，还可以指出可以提高非临床模型使用效率的领域。
