Presentation (CTI)

OBJECTIVES: In Italy, access to rare disease drugs (RDDs) is shaped by a multilevel governance system involving national pricing and reimbursement decisions and regional implementation processes. Time to RDD access show important difference across regions. The identification of prescribing centers (PCs) is a key operational step to enable patient access and appropriate use. This study aimed to (i) map regional processes, criteria, and stakeholders involved in PC identification; (ii) evaluate the perceived impact on regional access timelines and (iii) explore potential improvement actions to optimize regional access pathways.
METHODS: A mixed-methods approach was adopted, including: (i) literature review; (ii) analysis of the regional regulation; and (iii) semi-structured interviews with regional officers from five regions (Lombardia, Veneto, Emilia-Romagna, Campania, Puglia), selected to reflect different governance models. Data were analyzed descriptively and thematically.
RESULTS: The literature review identified a limited number of studies on PC identification. Only 11/21 regional authorities reported formalized guidance. Two main procedural models emerged: reliance on pre-existing rare disease networks versus de novo selection. Interviewees confirmed a top-down and more selective approach for RDDs compared to other drugs, generally aligned with accredited reference centers. Selection criteria mainly refer to clinical expertise, patient volume, and organizational capacity, with a heterogeneous level of formalization and specification. PCs identification is not perceived by interviewees as a major driver of delays in regional access timelines. Pre-existing networks, a horizon scanning activity that includes a pre-evaluation of PCs, early alignment between governance levels and interaction with stakeholders, and effective information exchange were associated with increased regional readiness.
CONCLUSIONS: Strengthening anticipatory planning, improving information sharing across governance levels, and promoting coordination mechanisms may support timely and equitable access to RDDs in decentralized healthcare systems.