2nd International Conference on Rare Diseases

Different infrastructures and organisational arrangements are required in each country to address the healthcare needs of its rare disease population in an effective way. Whether a single rare disease affects thousands or only a handful of individuals, they all share similar challenges to get a timely diagnosis, face the same barriers to access highly specialized care and treatment options, and need to deal with a substantial psychosocial burden.

The health of people living with a rare disease should not be left to luck or chance especially amidst the COVID-19 pandemic. Planning ahead requires all stakeholders to acknowledge their individual responsibility to build a successful pathway from diagnosis to access and at the same time, commit to working together in a much more agile and coordinated way than what we have seen so far. If we fail to do so, we will have failed the 30 million people living with a rare disease in Europe.