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.