INTRODUCTION: The Italian Law 326/2003, in the art. 48, requires all drugs companies operating in Italy to pay 5% of their promotional expenses to a fund (“Fondo AIFA 5%”), which allows the supply medicines awaiting market entry to rare diseases’ patients.

OBJECTIVES: The study aims to assess AIFA approach on 326/list inclusion requests: number and characteristic of applications submitted and their evaluation. Moreover, it was evaluated the economic impact of inclusion in 326 list on Fondo AIFA 5% during the years.

METHODS: The drugs’ panel was built by systematically reviewing the Reports of the AIFA CTS’ meetings from January 2013 to May 2019, checking the number and characteristics of drugs under evaluation, and analysing each single decision taken by CTS. We have also checked the last available reports drafted by the AIFA’s Italian National Observatory on Drugs Utilization (OSMED) 2013-2017, to check the economic impact of 326 Law on Fondo 5% during the years.

RESULTS: AIFA’s CTS approved 29/69 (42%) requests for access to Fondo AIFA 5%. 8/11 drugs approved (73%) have an Orphan Designation. The ATC mainly represented is the L (antineoplastic and immunomodulants agents) (50%) followed by the J (antinfectives for systemic use) (20%). The reason of negative (40/69) is available only for 28/69 (41%): for 43% of them (12/28) AIFA suggested to access the DM 8 May 2003/compassionate use. The economic impact of 326 list inclusion on Fondo 5% has increased considerably during the years: 183.382,00 € in 2013 (1%), 239.895,00 € in 2014 (2%), 1.108.530,00 € in 2015 (6%) and 13.465.742,00 € in 2017 (76%).

CONCLUSIONS: Our data showed as 326/2003 Law is a powerful early access scheme and that its use is growing significantly during the years, showing AIFA’s commitment to allow early access to drugs not still available for Italian patients.