Introduction. The national network for rare diseases allows patients suffering from a rare disease to receive medical assistance through the National Health System. Methods. In Emilia Romagna, the Regional Health System may also reimburse drugs not covered by the National Health System, after a positive opinion expressed by a technical committee, which evaluates the application through a standardized procedure, based on published evidence and on the discussion of individual single clinical case. Results. During the first three years (october 2004 - September 2007), the committee examined 331 applications concerning 300 patients, for a total of 832 drugs. An analysis of the decisions of the Committee shows that approximately 70% of the applications are approved, mostly on the basis of the needs and characteristics of the individual cases. Conclusions. This approach to rare diseases responds to a significant need of patients who are in special clinical circumstances that must be evaluated on a case-by-case basis and cannot be defined a priori.
|Translated title of the contribution||The network for rare diseases as managed by the technical committee of Emilia Romagna Region|
|Number of pages||9|
|Journal||Giornale Italiano di Farmacia Clinica|
|Publication status||Published - Apr 2008|
ASJC Scopus subject areas
- Pharmacology (medical)