Pmu54 a 10-Year Review (2009-2018) of Market Access of Pharmaceuticals in the Eu5 and Us

Abstract

71.9%) orphan drugs had a monitoring registry, and 23 (71.9%) had confidential discounts. Categorization by therapeutic area yielded 5/16 (31.3%) orphan-oncological drugs with MEAs and 6/16 (37.5%) orphan-non oncological drugs with MEAs. Conclusions: Our analysis showed a majority of orphan drugs without MEAs. Therapeutic area did not impact on MEAs assignment, given the similar percentage of orphan-oncological and orphan-non oncological drugs with MEAs. There is a preponderance of financial MEAs compared to outcome based MEAs. Although orphan drugs are a peculiar category of medicinal products, often conditionally approved or under exceptional circumstances or with lack of solid data, Italian authority keeps focusing on budget management rather than possible clinical outcomes uncertainties, maybe due to difficulties in applying outcome-based MEAs.