Orphan drugs: the challenges of availability and sustainability

Prevalence data from the monitoring system established in Veneto Region, Italy, since 2002 allow a rough estimation of the costs related to orphan medical products for rare disorders patients. The impact on the regional and national system is not negligible due to the high costs of these treat- ments and to the increasing number of patients eligible to therapy. A chal- lenge concerning sustainability of this system in the near future is arisin