Evolution of the Utilization of the Aifa 5% Fund for Orphan Drugs and Rare Diseases

OBJECTIVES: Since 2003 law 326 established the creation of a National Fund at AIFA to be used for the acquisition of orphan drugs for treatment of rare diseases and drugs that represent a therapeutic hope for specific and serious conditions.

The analysis aims to understand the evolution of the utilization and allocation of economic resources for the so-called AIFA 5% from 2015 to 2021.

METHODS: The information available in the 'transparent administration' section of the AIFA website was examined to understand the allocation of resources for the AIFA 5% Fund. Yearly economic parameters such as initial budget, allocation, total expenditure, and final balance were considered. Through OSMED reports, the 10 drugs with the greatest impact and the number of patients treated were also identified. This analysis provides a partial overview of the use of financial resources according to Law 326/2003.

RESULTS: From 2015 to 2019, the Fund maintained a budget surplus, particularly considering the significant allocations: the highest expenditure was € 8.318.000 in 2019, while allocations remained almost stable at around € 17.000.000 per year. From 2019 onwards, higher annual expenditures were observed up to € 40.441.000 in 2021, with a reversal in allocations that decreased to approximately € 10.000.000. In fact, in 2021, AIFA decided to temporarily suspend the activities related to the fund to initiate a simplification and optimization of the system.

CONCLUSIONS: Over recent years, the AIFA 5% Fund has seen an exponential increase in its utilization. Comparing these data with OSMED reports, we also found some discrepancies, as the expenditure in the OSMED reports is higher than that in the AIFA balance sheets, highlighting the need for a more transparent and univocal reporting.

We look forward to 2023 data to understand if the trend of expenditure compared to the allocation is confirmed.