Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions

摘要

Highlights ? Non-clinical models for 10 neurological rare conditions are reviewed. ? Relevant study endpoints that can help support orphan applications are suggested. ? COMP considerations when assessing medical plausibility are detailed. ? Functional outcomes are highlighted as critical for showing medical plausibility. 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. Teaser This review provides an updated list of animal models that can be used to support orphan designations in rare neurological diseases, and outlines essential features of nonclinical studies that can demonstrate the medical plausibility of a drug.