Ginevra Mastroianni, Sara De Franceschi, Valeria Viola (Pharma Value)

Law 648/1996 allows a drug that has not yet been authorised or is not available in Italy to be dispensed at the expense of the SSN subject to the opinion of AIFA's Technical-Scientific Commission (CTS) in the absence of a valid therapeutic alternative. In case this was present, Law 79/2014 extended the application where the parameters of cost-effectiveness and appropriateness were met.

The purpose of the analysis was to analyse the number of applications for the inclusion of drugs on the 648/96 list and the positive and negative evaluations of the CTS during the period from February 2018 to December 2021.

The analysis showed a difference in the number of requests between the two two two-year periods: 133 in 2020-2021 and 86 in 2018-2019. This increase was not caused by COVID-19, despite the fact that there were different requests for COVID-19 treatment drugs. From the sub-analysis of the therapeutic areas, it can be seen that the majority of requests in all the years taken into account concern antineoplastic and immunomodulatory drugs

We highlighted a low number of 648 applications for orphan drugs: only 10 out of 133 in 2020-2021 and only 6 out of 86 in 2018-2019. In the future, we intend to further investigate 648/96 listing requests for orphan drugs and understand the low use of 648/96 as an early access tool for these diseases with a high unmet need.

In the immediate future, we will try to highlight the applications for early access drugs (in the EMA application procedure or already authorised but not yet reimbursed in Italy either for new active ingredient or for extension of indication) versus off-label and established use (based on data from the scientific literature).

The poster is attached here. We look forward to your comments.