The conference aims to underscore the need to address the root causes of inequality and discrimination faced by persons living with a rare disease and their families. In this regard, it recognizes that there is a need for policies and programmes aimed at eliminating the root causes of discrimination and stigma in healthcare settings to ensure universal and equitable access to quality health services without financial hardship for all people with RD. In addition, the conference’s scope is to promote the need to foster innovation and the positive contribution that innovation can make in promoting social cohesion, reducing inequalities and expanding opportunities for all, including persons living with a rare disease, along with recognizing the need to support, streamline and increase attention on research in rare diseases.

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 call upon all EU Member States to strengthen health systems, notably in terms of primary health care, in order to provide universal access to a wide range of health-care services that are safe, of quality, accessible, available and affordable, timely, and clinically and financially integrated.

This in turn will help to empower persons living with a rare disease in addressing their physical and mental health needs to realize their human rights, including their right to the highest attainable standard of physical and mental health, to enhance health equity and equality, end discrimination and stigma, eliminate gaps in coverage and create a more inclusive society.

By co-creating policy options today that can lead to a better patient journey in the future and towards engaging all stakeholders to take action, the 2nd International Conference on Rare Diseases aspires to bring together all stakeholders in the rare disease community – patient representatives, policymakers, clinicians, researchers, industry representatives, payers and regulators to exchange invaluable knowledge with the aim of enhancing dialogue and promoting a common action plan that will help balance equity and sustainability across all EU countries so as to unite them on the front of combatting rare diseases.