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From diagnostic odysseys to treatment breakthroughs: Today’s discussion on rare diseases, organized in the framework of the Healthcare Policy Summit led by the IRSS wasn’t just another conference—it was a catalyst for change.

This policy-shaping event, hosted by #CentrumMedyczne at the Medical University of Warsaw, brought Poland’s rare disease landscape into sharp focus with the participation of a range of well-known experts in the field. With over 3 million Poles affected, we can no longer afford to treat these conditions as ‘rare’ in our policy considerations. Their impact extends beyond individuals, calling for collective action.

Our debate touched upon diagnostic complications, treatment disparities between cities, and the financial challenges of rare disease drug pricing.

We were inspired by Prof. Urszula Demkow’s (Undersecretary of State, Ministry of Health) vision for integrating rare disease care into primary health services, Prof. Alicja Chybicka’s (Chair, Parliamentary Team for Rare Diseases) advocacy for expanded newborn screening, and Prof. Anna Kostera-Pruszczyk’s (Head, ERN Euro-NMD Center, Warsaw Medical University) insights on creating a national rare disease registry, as well as the key role of #ERNs in patient care.

Participants agreed that further efforts are essential to improve the patient’s journey. An appeal was made to allocate additional resources to research projects fostering international cooperation.

The proposed ‘Rare Disease Coordinator’ role within hospitals emerged as a potential game-changer for patient navigation.

As we analyze the National Plan for Rare Diseases, we’re cautiously optimistic about its potential as a comprehensive framework.

To all contributors – from policymakers to patient advocates – thank you. Your expertise drives us toward a Poland where ‘rare’ doesn’t mean ‘overlooked’.

This summit is a launchpad for future action. We will compile a report with recommendations to send to the Ministry of Health.

#RareDiseaseAction #HealthcareRevolution #PolicyInAction #IRSS #HPS