Giunta Regionale Direzione Generale Sanità e Politiche Sociali TREATMENT OF PATIENTS WITH RARE DISEASES: INDIVIDUAL TREATMENTS OR PROTOCOLS? M. Volta, M. Vizioli, E. Calzolari, E. Rozzi, E. Di Ruscio Servizio Presidi Ospedalieri, Direzione Generale Sanità e Politiche Sociali, Regione Emilia-Romagna INTRODUCTION Several Italian regions (Veneto, Emilia-Romagna, P.A. Trento, P.A. Bolzano, Liguria, Campania, Puglia and Umbria) have shared the same database for the recording of diagnoses and treatment plans of patients with rare diseases. This database provides real-time interaction between all the people involved in the care pathway of patients with rare diseases. Concerning the care management of these patients, it is necessary identifying tools and objectives. Among the tools, it is important identifying working groups aiming at developing shared protocols for each specific class of diseases, in order to define pathways as well as needful and effective pharmacological approaches for the treatment of the disease. METHODS PROTOCOLS Systematic review of scientific literature EBM The working groups examined the pharmacological treatments for 1954 rare diseases by reviewing 1682 articles. 354 drugs have been surveyed, of which 144 were approved and included in acts as regional extensions of Essentials Levels of Care. Agreement among the centers of the network for specific diseases Approved Regional Acts leading to enlargement of Essentials Levels of Care (LEA) 144 Refused 215 Drugs approved (144/359) included in protocols allowing their rapid prescription by recording of treatment plans in the database Data courtesy of Regional Register for Rare Diseases of Veneto Region RESULTS Fo reigner co untries Galenic Law 648/96 Orphan drugs Other treatments The database nowdays contains 6522 treatment plans for a total of 16.885 prescriptions. Only Veneto, Emilia-Romagna and P.A. Bolzano register treatment plans. Of all the prescriptions recorded: -425 prescriptions of Orphan Drugs; -85 requests of drugs which can be prescribed according to the Law 648/96; -558 prescriptions of Galenic Magisterial; -919 prescriptions of drugs currently approved only in foreigner countries. 11.71% of all prescriptions are referred to drugs included in protocols. CONCLUSIONS The development of shared protocols (based on EBM) for each specific class of diseases defines pathways as well as needful and effective pharmacological approaches for the treatment of rare diseases. It has been recognized that whether care prescriptions follow the proper established protocols, there would be a cost of 1.75 million € per year/per million of citizens, but it has also been acknowledged a saving of 6.51 million € per year/ per million of citizens, due to the lack of treatments’ prescriptions not included in the aforementioned protocols.