Patient access to orphan drugs in France

摘要

BackgroundSince incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. However, patient access on a national level remains very heterogeneous across Europe. This can be explained by healthcare organization and drug reimbursement, which are within the purview of each Member State. We studied access to orphan drugs in France from the patients’ point of view, including marketing but also ease of supply from patients’ perspective, financial and time-based dimensions.