OBJECTIVES: To assess the approach and outcome of AIFA Scientific Technical Committee (CTS) on evaluation of applications for inclusion in the Law 648/96 list from January 2017 to May 2022.

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

RESULTS: From January 2017 to May 2022 AIFA evaluated 243 new applications.

In the last two years there was an increase in the number of new applications (64 in 2020 and 51 in 2021 vs. an average of 37/year in the period 2017-2019); the increase was driven by requests in other indications, which represent the majority of requests both in 2020 (60.9%) and 2021 (51%).

In the full period analysed, CTS approved 112 requests (46.1%), 119 (49.0%) were rejected, 1 (0.4%) was not assessable and 11 (4.5%) are ongoing. Rare diseases accounted for 18.1% of requests, 12.3% were for rare tumors, 25.5% other oncology indications and 44.0% involved other indications.

From 2017 to 2019, more requests were approved than rejected. In 2020 rejected requests (61%) exceeded approvals (34%); while in 2021, of 48 requests assessed, 50% were approved and 50% rejected. An important decrease in positive opinions was observed for rare diseases (from 46.7% in 2017 to 0% in 2021).

CONCLUSIONS: Across the period analysed, there was a clear upward trend in number of requests submitted and evaluated by the CTS, particularly for other indications, for which an increase in positive opinions was observed. In contrast, new applications for rare diseases did not increase, and a clear downward trend in positive opinions has been observed.