Assessment of the Impact of Orphan Medicinal Products (OMPs) on the European Economy and Society


Orphan Medicinal Products are intended for the diagnosis, prevention or treatment of lifethreatening or serious conditions that are rare. Following the political impetus provided by the OMP regulation EC 141/2000 entering into force in April 2000, which in general terms provides incentives for the research, development and marketing of OMPs, there has been an important increase in the number of OMPs potentially available for patients in Europe compared to the situation before the regulation. Moreover, following this regulation and specifically also the implementation of the National Plan for Rare Diseases in France in 2005, a number of countries have decided to create their own national path. At  the same time, further scientific and policy research in the area is needed to better understand the epidemiological, clinical and personal impact of each rare disease in the EU as well as to support sound policy decision-making and enhance several aspects of caring for patients including access to diagnosis, treatment and care.