Un rapido accesso ai nuovi trattamenti efficaci rimane una priorità dell'Agenzia Italiana del Farmaco (AIFA) e del Servizio Sanitario Nazionale (SSN) e un importante traguardo per le aziende farmaceutiche. 1 Poiché molti dei nuovi farmaci sono approvati a livello centralizzato dalla European Medicines Agency (EMA), l'accesso ai nuovi trattamenti riveste anche un carattere di equità comunitario. Proprio per questo, la Legge 189/2012 ha istituito la classe C NN , in cui è previsto che i farmaci approvati con procedura centralizzata sono automaticamente classificati entro 60 giorni dall'ottenimento dell'AIC europea. 2 Questo ne consente la commercializzazione, nonostante non abbiano concluso la procedura negoziale con AIFA (" NN " sta per

Objective. To compare the time-to-reimbursement of the last two committees of the Italian Medicines Agency (AIFA), respectively appointed in 2015 and in 2018. Methods. The analysis was run through a specific internal database created by MA-Provider. The database was populated with information regarding European Medicines Agency (EMA) approved new drugs, including each step of the Italian Price and Reimbursement (P&R) process reported in the monthly outcomes of Technical Scientific Committee (CTS) and Price and Reimbursement Committee (CPR) meetings from September 2015 to April 2020. Results. The 2015 and the 2018 committees have reimbursed respectively 39 and 28 drugs by comparing their initial 19 months of activity. Significant differences have been observed in negotiated economic conditions, in particular an increase in the number of drugs with confidential discount (2018-committee: 96.4% vs 2015-committee: 64.1%; p = 0.003) and a reduction in the application of Managed Entry Agreements (MEAs) (2018-committee: 10.7% vs 2015-committee: 33.3%; p = 0.036). The average duration of the P&R procedure managed by the 2018-committee has increased by 45 days compared to the 2015-committee (287 days vs 242 days; p = 0.071) and this trend of delay is associated to the active scientific/economic assessment phase by CTS and CPR (particularly by the latter) and not to administrative phases (e.g. Official Journal publications). Conclusions. The observed differences between committees may be explained by the higher number of oncological and/or innovative drugs assessed by the 2018-committee (regarding the time delay, probably linked to greater difficulties in finding a win-win agreement able to satisfy both AIFA and Pharmaceutical Company).

, were collected and categorized according to: i) kind of innovativeness: full, conditional or no innovativeness; ii) orphan designation; iii) therapeutic area: oncological vs non-oncological drugs. For each single indication assessed for innovativeness, the ranking of the three criteria (therapeutic unmet need maximum to absent, added therapeutic value maximum to absent, and quality of evidences high to very low) was analyzed. Sub-analyses according to the three categories were also run. Results: Out of forty-one indications assessed by AIFA for innovativeness (and corresponding to 30 drugs), 12 (29%) were granted full innovative status, 14 (34%) obtained the conditional innovativeness and the remaining 15 (37%) were assessed as not innovative (7 orphan and 8 oncological indications). 50% of full innovative indications referred to orphan drugs (6/12) and 42% to oncological diseases (5/12); regarding conditionally innovative indications, 29% were for orphan drugs (4/14) and 79% (11/14) for oncological ones. Analysing innovativeness criteria application, AIFA have never recognized the maximum grading for added therapeutic value; moreover, focusing on fully innovative indications, 9 out of 12 (75%) did not meet all the three criteria thresholds stated by AIFA decree. Conclusions: This analysis highlighted that there is no strict matching between AIFA criteria (as reported by AIFA decree) and the granting of full innovative status. In fact, most of the analysed cases are borderline situations (full vs. conditional innovativeness) and have been assessed case-by-case, leaving a shade of subjectivity that gives innovativeness decision-making process a margin of flexibility. The limited sample does not let us make conclusions on the impact of orphan designation or oncological setting.

A659that contain both pharmaceutical and non-pharmaceutical schemes was calculated, and non-pharmaceutical expenditure items were identified and subtracted to produce a total pharmaceutical and non-pharmaceutical expenditure estimates and calculate their relative proportions. Results: For non-high-tech medicine, total expenditure decreased from € 1.66bn in 2012 to € 1.4bn in 2015 (15.7% decreases). For non-pharmaceutical items reimbursed under PCRS, the decrease in expenditure during this period was 0.68m (< 0.5% decrease). In 2015, total expenditure on nonpharmaceutical products reimbursed under PCRS was € 173m. ConClusions: Since the impact of the economic recession, the total cost of medicines reimbursed in Ireland has decreased. The decrease in expenditure, at a time of increasing demand, was supported by the introduction of mandatory rebates for innovative pharmaceutical medicines. This analysis shows that for non-pharmaceutical items, expenditure reductions over this period were not proportional.

OBJECTIVES: To track and analyse the economic negotiated conditions (Managed Entry Agreements [MEAs], monitoring registries, discounts) of novel drugs reimbursed in Italy through Official Journal publications. METHODS: Eighty-eight drugs which were granted EU approval between May 2015 and May 2018 and completed the P&R process in Italy were tracked and categorized by type and therapeutic area in a monthly updated database. Negotiated MEAs, applied confidential discounts and monitoring registries were evaluated through Official Journal publications. RESULTS: Categorization by type yielded 26.1% (23) orphan drugs (EU), 19.3% ( 17) innovative (AIFA), 5.7% (5) classified as orphan and innovative drugs and 48.9% (43) others; 14.8% (13) oncological drugs, 11.3% (10) oncohaematological and 73.9% (65) others by therapeutic area. Data analysis showed that 33% (29/88) of drugs had MEAs as approval condition; 26 (29.5%) drugs had one single MEA, 3 drugs (3.5%) had two MEAs (payment-by-results + budget cap or price-volume + capping) and the remaining 59 had none. Further analysis of the total 32 (31 and 1 undisclosed) MEAs showed that 31.3% (10) had price-volume agreements, 25% (8) budget cap, 15.6% (5) cost-sharing agreements, 12.5% (4) payment-by-results, 9.3% (3) had capping. Furthermore, AIFA set a market access agreement, a flat fee per patient, for Zalmoxis (3.1%). Twenty-two (75.9%) of the screened drugs with MEAs had monitoring registries and 16 (55.2%) had confidential discounts. CONCLUSIONS: Data evaluation showed an unbalance between outcome based and non-outcome based (financial) MEAs suggesting that regulatory attention is predominantly on the economic impact of new drugs; although AIFA recognised the added value of a new patient specific cell therapy by applying flat fee per patient. The low number of outcome based MEAs, though, are counterbalanced by the large number of monitoring registries requested. Budget cap and price-volume agreements are the most commonly negotiated MEAs.