Patients suffering from rare diseases are calling for the immediate implementation of Poland’s Rare Disease Plan, urging authorities to move beyond policy frameworks to deliver coordinated care and faster diagnosis.
Bridging the Gap Between Policy and Reality
Rare disease patients remain in limbo, waiting for systemic changes that shorten diagnostic timelines and provide access to coordinated expert care. Participants at a workshop organized by the National Forum for Rare Disease Therapy ORPHAN highlighted the urgent need to accelerate the implementation of the Rare Disease Plan.
The group’s specific expectations have been formalized into a direct appeal, which will be submitted to the Minister of Health. These stakeholders emphasized that the plan, while well-structured, must shift from a theoretical document to tangible clinical practice to improve lives.
Launching Rare Disease Expert Centers
A primary objective is the activation of Rare Disease Expert Centers (OECR), which are intended to serve as the foundation of the patient care system. These centers are meant to provide comprehensive diagnostics and treatment coordination.
Dominika Gradek, a representative from the Ministry of Health, confirmed that legislative work regarding the OECR system is ongoing. Implementation is expected to occur in stages over the coming months.
Digitalization of the Diagnostic Pathway
Patients often face a multi-year diagnostic odyssey involving numerous specialists before receiving a proper diagnosis. Digital transformation is viewed as a critical solution to streamline the flow of information and improve system efficiency.
Key initiatives include the development of e-Consilia, the implementation of a National Rare Disease Registry, and the introduction of a Rare Disease Patient Card. These tools are intended to assist physicians and improve regional healthcare planning.
Institutional Collaboration and Legal Frameworks
The dialogue between public administration, medical experts, and patient organizations remains vital. Participants stressed that engaging stakeholders early in the legislative process leads to more effective and realistic healthcare outcomes.
There is also an urgent call for a dedicated Rare Disease Act. Stakeholders argue that a permanent legal foundation is required to ensure the long-term sustainability of funding and to clarify the responsibilities of various institutions within the healthcare system.
A Call for Immediate Action
Stanisław Maćkowiak, president of the ORPHAN Forum, stated that the Rare Disease Plan is a solid foundation, but its provisions currently exist primarily on paper. He noted that the patient community is ready to support the government, but emphasized that the time for planning has passed in favor of immediate action.
