Managing rare diseases: examples of national approaches in Europe, North America and East Asia

Abstract

Around 4% of the global population suffers from a rare disease. Apart from the medical aspect, economic, organizational, and political approaches remain key aspects concerning the evolution of the world of rare diseases. Here we review the principal specific national initiatives and organizations dedicated to rare diseases in Europe, North America, and East Asia. We then present the outline of a possible optimal approach inspired by the successes of individual national organizations. This work should be taken into account in the definition of large-scale multinational rare diseases programs, such as the European Joint Program on Rare Diseases.

Keywords

Rare diseases / national plans / Europe / expertise / reference / networks

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