From left to right: MEP Vytenis Andriukaitis (S&D, Lithuania), Dr Mauro Cancian (Head of allergology unit, Padua University Hospital), Tamsin Rose (moderator), Henrik Balle Boysen (President, HAEi), Sujan Sivasubramiyam (Senior Director, Global Advocacy and Policy, CSL Behring), MEP Stine Bosse (Renew, Denmark)
In November, a multi-disciplinary panel engaged in a debate titled: "Towards Better Diagnosis and Care for HAE Patients in Europe." Hereditary angioedema (HAE) is an unpredictable chronic genetically inherited condition marked by swelling in several body locations that can be life-threatening and exemplifies the struggles of rare disease patients.
Despite advancements in the field, barriers remain in treatment access, with delays from marketing authorizations and reimbursement. The President of HAEi called on the EU to promote national plans for rare diseases and empower patient organizations to make “HAE a beacon of hope for all rare diseases.”
In addition to national plans, the panel strongly recommended that the European Commission adopt an EU Action Plan on Rare Diseases to drive progress under a unified framework, connecting expert knowledge, standardizing data, and fostering innovation. Enrique Terol, Health Counsellor from Spain, suggested reinforcing this effort with a World Health Assembly Resolution on Rare Diseases, potentially adopted as early as 2025.
Approximately 36 million Europeans live with a rare disease, yet access to specialized care varies significantly across the continent, necessitating coordinated EU action.
Speakers highlighted initiatives like the EU Life Science Strategy to improve patient access to next-generation therapies. MEP Bosse stressed that “patients should be at the centre of the Life Sciences Strategy, and collaboration with industry and investors is vital” to secure a competitive, resilient Europe. Sujan Sivasubramaniyam from CSL Behring, continued that, “with advancements in treatment, the goal shifts from merely reducing mortality to achieving complete symptom control and enhancing quality of life,” emphasizing the need for ongoing investment in innovations. MEP Hilde Vautmans added, “We need to improve diagnosis, treatment, and access to new medicines, ensuring no rare disease patient is left behind.”
The panel further aligned on seeking fast and equitable action, as many rare diseases are debilitating conditions which require immediate attention. MEP Andriukaitis urged faster EU action, reflecting on the European Reference Networks (ERNs) established in 2017 and their lengthy national integration challenges. Dr Cancian reiterated the need for multistakeholder collaboration in rare diseases, essential for developing diagnostic tools, conducting clinical trials, and ensuring sustainable access.
Adding to the debate, MEP Kulja stated that, “improving access to care and innovation for HAE patients requires prioritizing timely diagnosis, fostering cross-border research collaboration, and ensuring equitable access to advanced treatments.”
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