The 1^st International Conference on Rare Diseases “Building a Pathway from Diagnosis to Access” recognises that the next decade holds great potential for improvement and that while we cannot predict the future, we all have a role in preparing for it. As the EU shapes its future regulations, strategies and access policies, this conference will serve as an opportunity to press the ‘pause button’ and take the time to co-create policy options today that can lead to a better patient journey in the future.

Engaging all stakeholders to take action, the 1^st International Conference on Rare Diseases aspires to bring together all stakeholders in the rare disease community – patient representatives, policy makers, clinicians, researchers, industry representatives, payers and regulators to exchange invaluable knowledge with the aim to enhance dialogue and promote policy changes to help build a better pathway from Diagnosis to Access for 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 work 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.

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