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

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 2020. 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 2020 AIFA evaluated overall 159 requests. CTS approved 66 requests (41.5%), 79 requests (49.7%) were rejected and 14 (8.8%) are ongoing. The results showed that 19% of requests concerned rare diseases (29% positive), 13% rare tumors (60% positive), 27% oncology (49% positive), and 41% involved other therapeutic areas (37% positive). The only 2 requests presented for ATMPs have both been refused. Assessment procedures performed by CTS in 2017, 2018 and 2019 were 36, 35 and 30 respectively. A reduction of requests on rare diseases was observed (12, 6, 5 respectively), while an increase in oncology occurred across this period (9, 12, 14 respectively). In the first half of 2020, CTS assessed a higher number of requests (58), of which only 16 (28%) obtained a positive opinion. Only 5 requests referred to COVID-19 treatment. Differences in duration of the evaluation process between groups were also observed.

CONCLUSIONS : Across the analyzed period, negative CTS opinions are prevalent. The analysis shows a higher percentage of approvals for rare tumors (in 2019 CTS outcomes were all positive), and in general for oncology, although a progressive increase of negative opinions in other oncology indications was observed. In the first half of 2020, an upward trend of inclusion requests was observed, which led to an increase in the percentage of negative opinions. No ATMPs obtained reimbursement through this scheme.