Valutazione dei tempi di accesso per le nuove entità chimiche in Italia: un’analisi critica del periodo 2018-2024

Abstract

Objective:: This study aims to provide an up-to-date analysis, for new chemical entities on the market, on the timing of pricing and reimbursement (P&R) in Italy, covering the entire period of activity (from September 2018 to January 2024) of the last evaluation Commission (Technical Scientific Committee, CTS, and Price and Reimbursement Committee, CPR).

Methods:: The data used in this analysis were obtained from public official websites. The information was systematically collected to investigate the days required to complete the P&R process. The analysis was stratified into indications for rare diseases, orphan designation, innovation assessment, and anatomical therapeutic chemical (ATC) class L. Mann-Whitney U test was used to study the significance of the difference. A p-value < 0.05 was considered significant.

Results:: Overall, 180 procedures were included in the final analysis. The median duration of the entire process, from MAH submission to final Gazette publication, was of 439.5 days. The timelines varied among the analyzed drug classes. Rare disease drugs (n = 78) had a longer timeline than non-rare-disease drugs (n = 102) (462.0 days vs 419.5 days, respectively). Among rare disease procedures, orphan designation was a predictor for time prolongation (orphan drugs, n = 66, 462.0 days vs 443.0 days non-orphan drugs, n = 12). Innovativeness status was associated with a shorter timeframe (-14 days), as was the ATC L classification (-68 days).

Conclusion:: The results indicate a methodological consistency with previous studies, suggesting a continuity of analysis, despite the increase in the complexity of evaluations and the number of drugs treated.

FIGURA 1 -

Flow-chart delle procedure incluse nell’analisi. *Esclusi vista l'assenza della data di sottomissione del P&R dossier in GU.

TABELLA 2 -

Densità delle distribuzioni dei giorni totali del processo di valutazione e negoziazione delle 180 nuove molecole incluse nelle analisi e nei sottogruppi di interesse (periodo di riferimento: settembre 2018 – gennaio 2024)

FIGURA 2 -

Valori mediani dei giorni per step di analisi per tutti i farmaci (n = 180).

FIGURA 3 -

Valori mediani dei giorni per step di analisi per: a) malattie rare (n = 53) e sottogruppo farmaci per malattie rare orfani (FOMR, n = 66) e non orfani (FnOMR, n = 12); b) farmaci innovativi (FI, n = 46) e non innovativi (FnI, n = 134); c) farmaci con ATC L (FATCL, n = 72) e altri farmaci (FnATCL, n = 108). CdA = Consiglio di Amministrazione; FATCL = farmaci ATCL (antineoplastic and immunomodulating agents); FI = farmaci innovativi; FMNR = farmaci per malattie non rare; FMR = farmaci per malattie rare; FNI = farmaci non innovativi; FNATCL = farmaci non ATCL; FNOMR = farmaci non orfani per malattie rare; FOMR = farmaci orfani per malattie rare.