An optimal approach to rare disease management based on national experiences in Europe, North America and East Asia

A new study has been published in the Rare Diseases and Orphan Drugs Journal presenting transdisciplinary approaches to managing rare diseases in Europe, North America and East Asia coordinated by the national governments. The study compares the national rare disease plans in Germany, Spain, the UK, France, Japan, Canada and the US.  The aim of the study is to find an optimal approach in rare disease management that can be considered in multi-national rare diseases programmes such as the EJP RD. The study identifies the functioning of each national plan, including the source of funding, the national register and the centres of expertise while highlighting the strengths and limitations of each approach. Based on this comparison, the study reveals an outline for an optimal approach in terms of regulations, funding, centres of expertise, gene identification, registries, and public-private partnerships.