Call to Action

Call to action

On 1 March 2023, Partnering4PNH and Sobi, in collaboration with the Parliament Magazine and EUCOPE, organised an event hosted by and in partnership with MEP István Ujhelyi (S&D, Hungary), MEP Stelios Kympouropoulos (EPP, Greece) and MEP Maria Garça Carvalho at the European Parliament.

During the event participants were invited to sign a call to action to pledge their commitment to continue the search for much needed solutions to address the many unmet medical needs of the rare disease patient community.

The call to action asks decision makers to consider the below points as guiding actions when revising and implementing the upcoming Orphan Medical Products (OMP) and General Pharmaceutical legislation:

• Reduce extended diagnosis journeys, address misdiagnosis and “undiagnosis”
• Allocate funds from EU and national budgets to strengthen the capacity of the Centres of Expertise and enhance the competencies of the European Reference Networks (ERN)
• Use disease-specific instruments in health technology assessments (HTA)
• Empower patients and caregivers in decision-making
• Strengthen the training of healthcare professionals on rare diseases
• Reduce inequalities and expedite access to treatment
• Build a more resilient ecosystem for research and innovation for all rare diseases