The pharmacist, rare diseases and orphan medicines

Treatment of rare diseases is often complicated due to the difficulties posed by the research and development of medicines by the pharmacological industry. One of the greatest concerns of this group of patients, which is both scarce and diverse, is access to available medicines. The European Medicine Agency establishes the term orphan medicines and the incentives and bases for their commercialisation. Due to their special characteristics, the majority of orphan medicines require suitable vigilance and control. The hospital pharmacy services are actively involved in their management and supply, as well as in a correct pharmacotherapeutic monitoring of the patients.