[Seven years of the National Action League for People with Rare Diseases : NAMSE - a success story?!]

Abstract

It is estimated that four million people in Germany are living with a rare disease; in the EU, approximately 30 million people are affected. All rare diseases are chronic, linked to invalidity and/or restricted life expectancy, and their symptoms are often already manifest in childhood. Some 80% of rare diseases are genetically determined; they can rarely be cured. Furthermore, the pathogenesis of many rare diseases is unknown.In a move to ameliorate the situation of persons with rare diseases, the National Action League for People with Rare Diseases (NAMSE) published the National Plan of Action for People with Rare Diseases containing 52 measures that aim to achieve the desired improvements. More than half of these measures have already been implemented or are currently in the process of implementation. The Federal Ministry of Health is supporting this implementation process with numerous projects.NAMSE has achieved a great deal as a result of the National Plan of Action for People with Rare Diseases, even though not all of the measures included in the National Plan of Action have been implemented. The first results of individual measures are already becoming apparent. In order to achieve long-lasting improvements in the prevention, diagnosis and therapy of rare diseases, initiatives will have to be coordinated in the future as well.The Federal Ministry of Health will be funding the NAMSE Coordinating Office until mid-2018, thus ensuring that NAMSE will remain operational until that time. All of the partners collaborating in the Action League agree on the need for NAMSE, together with its coordinating office, to remain in place. To this end, a structure that keeps the health care situation of persons with rare diseases under observation, that suggests solutions and offers a possibility for the parties involved to network, is necessary. The question of conducting a legal restructuring of NAMSE in the future that will be sustainable in the long term is currently under discussion.

Keywords: Action plan; Centers; NAMSE; Rare diseases; Sustainability.