ABSTRACT
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.
Subject(s)
Nervous System Diseases , Orphan Drug Production , Rare Diseases , Animals , Disease Models, Animal , Drug Evaluation, Preclinical , HumansABSTRACT
Every day occur about 4 strokes in the Grand-Duchy of Luxemburg. Stroke is the third cause of death and the first cause of acquired handicap. Stroke constitutes a major individual and social problem. Stroke and TIA are neurological emergency conditions that warrant immediate hospitalisation on a stroke unit, where a dedicated equipe of neurologists, nurses, speech and occupational therapists, physiotherapists and social workers in collaboration with other medical and surgical specialties provide up-to-date diagnostic and therapeutic measures. These include the diagnostic work-up of stroke aetiology and the vascular status, monitoring to ensure the maintenance of homeostasis, thrombolysis and revascularisation, antithrombotic drug treatment relying on individual stroke pathophysiology, treatment of risk factors and early rehabilitation. By treatment on a stroke unit, death and disability can be reduced by 27%, in the case of intravenous thrombolysis even by 45%.
