Crisis readiness for rare disease populations: learnings and recommendations by the European Reference Networks

Patients with rare diseases are particularly vulnerable during emergencies due to dependence on specialised care pathways, medicines, and expertise. European Reference Networks (ERNs) for rare and complex diseases have operated since 2017 and were confronted with two major crises: the COVID-19 pandemic and the war in Ukraine. To assess ERN experiences, responses, and preparedness for crisis and disaster situations, we conducted a cross-sectional survey of all 24 ERN coordinators between July and September 2025. Only 2 (8·3%) ERNs reported pre-existing preparedness plans, while 70% lack structured frameworks. Major bottlenecks included patient access to healthcare facilities, drug and device shortages, bed shortage, staff unavailability, and diagnostic service interruption. Our findings highlight the need to formally integrate and mandate ERNs into European health emergency preparedness and response mechanisms. We propose a 10-point global crisis preparedness plan for rare diseases emphasizing cross-border coordination, digital infrastructure, patient education, and integration with national emergency services and non-governmental organizations.