OBJECTIVES: To assess AIFA Technical Scientific Committee (CTS) approach and evaluation outcome of requests for inclusion in Law 648/96 reimbursement list -UPDATE

METHODS: Requests for inclusion of an active substance/therapeutic indication into 648/96 lists were extracted from CTS meetings' agendas as published on AIFA website from January 2017 to June 2021. This analysis is focused on new requests, and was performed on CTS outcome, by year and therapeutic area (rare diseases, rare tumors, other oncology indications).

RESULTS: From January 2017 to June 2021 AIFA evaluated 209 requests. CTS approved 91 requests (43.5%), 111 (53.1%) were rejected and 7 (3.4%) are ongoing. Nineteen percent of requests concerned rare diseases (23% positive), 13% rare tumors (50% positive), 24% oncology (51% positive), and 43% involved other therapeutic areas (47% positive).

In 2020, requests assessed by CTS (72) were more than doubled vs previous years (36, 35 and 30 requests assessed respectively in 2017, 2018 and 2019); this is not related to assessment of COVID-19 treatments, since only 5 requests were assessed in 2020 (and 2 in 2021). Only 26 (36%) of requests assessed in 2020 obtained a positive opinion. The majority (42-58%), was for indications other than rare diseases or oncology, but there was also an increase of requests for rare tumors (from 4 in 2017 to 10 in 2020).

A reduction of positive opinions was observed. After reaching a maximum of 54% in 2018, positive opinions decreased to 47% in 2019, 36% in 2020, and 21% in the first half of 2021. The decrease was more marked in rare diseases, with 0% positive opinions in 2021.

CONCLUSIONS: Across the analyzed period, a clear trend was observed toward reduction of positive opinions, more marked for rare disease. Also, there was a higher percentage of approvals for rare tumors and in general for oncology.