The Article “Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen” has been published on-line in Orphanet Journal of Rare Diseases 2017.

The publication reports the results obtained by EuOrphan database analysis aimed to evaluate the status of orphan drugs designated for rare diseases in Europe (EU) and United States (U.S.A) up to December 2015.

EuOrphan is a database that collects information on medicines for rare diseases designated and/or approved in Europe (EMA) and in the U.S.A (FDA), that has proven to be a useful tool to increase knowledge on rare diseases and facilitate research. It was created by Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) in the framework of a funded European IT-Technology project (eTen 510774 2003/C 118/19) and regularly updated by Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (FGB).

In order to increase the availability of treatment for rare diseases, EU and US health authorities have adopted specific pieces of legislation including incentives to companies developing treatments or diagnostics for rare diseases

Published data show that notwithstanding these incentives, the number of medicines for rare diseases is still limited, and this is more evident in several therapeutic areas.

With reference to paediatrics, published data demonstrated that despite of the interest and the need for drugs approved for children, about half of drugs approved in the EU and US for a rare disease affecting children lack of a paediatric indication and this situation is most serious for younger children, especially neonates.

Authors conclude that joint efforts and cooperation between EU and US seem to be the key to speed up the development and marketing of drugs for rare diseases.

The publication is available at this link.