Mission Possible for Rare Disease

The conference brings together patient advocates, policymakers, researchers, clinicians, health care professionals, industry representatives, regulators and member state representatives to explore rare disease policy issues, both national and international.

This year’s conference focused specifically on recommendations stemming from Rare 2030, a two-year study that gathered input from rare disease stakeholders across Europe. Findings led to eight recommendations:

1. Develop a European policy framework.
2. Develop earlier, faster, and more accurate systems of diagnosis for rare diseases.
3. Implement the use of a highly specialised health care ecosystem.
4. Guarantee the integration of people living with a rare disease in societies and economies.
5. Encourage meaningful participation and engagement of people living with a rare disease
6. Maintain rare disease research as a priority.
7. Use data to its maximum potential.
8. Improve the availability, accessibility, and affordability of rare disease treatments.

It is this first recommendation, the need for a European policy framework, that served as the focus of this year’s conference. Attendees made clear that it is not enough for member states to continue to take action on their own, or to look for legislative changes in specific areas. Helping the rare disease population in the region requires a collective strategy, bringing together the goals of different member states into one collective, cohesive policy approach.

Policy formulation sessions offered opportunities to propose concrete recommendations for improving access. They also allowed participants to explore ways to better utilise new and innovative technologies and methodologies for Europe’s rare disease population.

With the assistance of these eight recommendations and continued engagement and input from the rare disease community, the European region is working to create a roadmap to guide and bolster rare disease policy in the years ahead.