The rare diseases agenda of the European Parliament

Abstract

During the past years, rare diseases have been one of the main themes on the European Union’s agenda. The reason for this theme being highlighted is the excellent exchange of knowledge between EU policy makers and the relevant patient communities. The former negligence has been caused by the apparent low patient numbers. The definition of a rare disease in Europe contains, that there are less than five patients out of a total population of 10000 persons suffering from this disease. Taking the total of all patients, suffering from rare diseases, it becomes a different number. An estimated 30 million EU citizens are suffering from a rare disease. This tells us that rare diseases are actually not exactly rare. Due to the great part of the population,which is affected, we have a social obligation to prioritise on this matter. Furthermore, the type of health conditions deserves a strong focus. The European Union, and notably the European Parliament, has made significant progress towards the provision of a political framework, which would define rare diseases and provide incentives for the research and development of medicines. The EU Orphan Medicines Regulation, adopted in 1999, was a political milestone in the prioritisation of rare diseases. It has provided the European Union with a clear competence in refining this topic. It was this legislation, which created incentives for medical manufacturers to develop products, furthermore, they generated a market exclusivity going beyond patent protection and a fee waiver for the evaluation and registration process of specialized products. The establishment of the European Medicines Evaluation Agency (EMEA) in 1995, meant the foundation of a centralised body, which can appropriately deal with the necessary central registration of available and registered medicines. This has been a great success story. Since then, 639 products have been registered with the European Medicines Agency. More importantly, the Agency transformed Europe to a leading region for research and development in this field.