Orphan drug policies in different countries

Abstract

Objectives: Due to the low prevalence of rare diseases (RDs) and the small number of individuals affected with a RD, challenges complicate the development of and accessibility to medications and medical devices to prevent, identify, treat or cure these RDs. The aims of this study were to analyse and compare regulations and policies related to accessibility of orphan drugs (ODs) in different countries which have policies or systems in place which are specific for RDs and ODs in Europe and the United States (US). Methods: A comprehensive literature review was carried out. A total of 20 120 articles which contained information about accessibility and legislations, regulations and governmental policies related to RDs and ODs were retrieved. Key findings: Sixty-eight articles were included in this study. Classification of information related to 24 countries was based on six themes: OD policies, marketing authorisations, incentives offered, pricing of ODs, reimbursement and pharmacovigilance. The EU and United States have adopted policies and regulations aimed to improve OD accessibility in the last 20 years. Although all included countries had an OD legislation, only 16 countries had an OD or RD plan in place. Conclusions: Comparison of different legislations and policies in each country can be used to identify and address deficiencies in the field of RDs and improve accessibility of ODs.