La rete di assistenza per le malattie rare: Esperienza del gruppo tecnico della Regione Emilia Romagna

Translated title of the contribution: The network for rare diseases as managed by the technical committee of Emilia Romagna Region

Stefania Proni, Luca Favero, Fabrizio Salvi, Elisa Calzolari, Alessandro Cicognani, Mauro Mazzolani, Elisa Rozzi, Matteo Volta, Fabrizio De Ponti

Research output: Contribution to journalArticlepeer-review

Abstract

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 contributionThe network for rare diseases as managed by the technical committee of Emilia Romagna Region
Original languageItalian
Pages (from-to)65-73
Number of pages9
JournalGiornale Italiano di Farmacia Clinica
Volume22
Issue number2
Publication statusPublished - Apr 2008

ASJC Scopus subject areas

  • Pharmacology (medical)

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