Pro71 What Was the Actual Budget Impact of Orphan Medicinal Products Between 2000-2017 in Europe?

Abstract

survival of ovarian cancer (OC) in the EU. The inputs were fed into the DICE model and prevalence over time was evaluated and compared with EMA submissions. Results: A stable OC prevalence of 4.1/10,000 was estimated for the EU and compared with the varying rates submitted to EMA (2.3-4.9/10,000). Multiple scenarios were tested. For example, if incidence was increasing by 2% per year post-2002, prevalence would reach the 5/10,000 cutoff in 2012. Conclusions: The DICE epidemiological model can produce a robust prevalence estimate by synthesizing available data from different sources and allowing for multiple scenarios to be tested. This model can assist sponsors and the EMA in their review by introducing a transparent and flexible tool which can be used as a standard for orphan designation submissions across multiple diseases.