PMU49 NEGOTIATION OUTCOMES IN ITALY: WHAT’S THE IMPACT OF ORPHAN DRUG DESIGNATION?

Abstract

To analyse the negotiated conditions (Managed Entry Agreement, discount and monitoring registry) of orphan drugs reimbursed in Italy between January 2015 and May 2019. Starting from reports of AIFA committees meeting, P&R process of new medicinal products was tracked in internal database; also subanalysis by therapeutic area was run. confidential discounts application and negotiated MEAs were tracked through published Official Gazette. Data analysis showed that 32 out of 121 reimbursed drugs had orphan designation; 21 out of 32 (65.6%) drugs had no MEAs and 11 out of 32 (34.4%) had one single MEA. Analysing the 11 MEAs applied, we tracked: 3 (9.4%) budget caps, 2 (6.3%) cost-sharing agreements, 2 (6.3%) payment by results, 1 (3.1%) capping and 2 (6.3%) undisclosed MEAs; additionally a flat fee per patient was applied to 1 drug (3.1%). 23 out of 32 (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. 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.