Drug Prices and Value of Oncology Drugs in Italy.

OBJECTIVE: The main objective of this study was to evaluate the potential role of efficacy data and other information available at the time of price and reimbursement (P&R) decision-making process within the definition of oncology treatment costs in Italy. METHODS: The study included all P&R dossiers submitted to the Italian Medicines Agency between July 2015 and December 2017. It prospectively collected the data of the P&R process starting from dossier submission up to the Italian Health Service reimbursement decision. The cost of treatment per patient was estimated using both the list price ("gross cost") and the confidential net price ("net cost") of drug packages and applied to the median duration of treatment. A 2-sample stage Heckman decomposition model was used to evaluate the potential role of efficacy data and other information available at the time of P&R decision making on the gross and net cost. RESULTS: A total of 37 oncology drugs related to 58 therapeutic indications were analyzed. The multivariate model showed that the variation of progression-free survival is the only variable predictor statistically associated with treatment cost, but this effect was observed only when confidential net prices were used (P=.026). CONCLUSIONS: Considering the perspective of a developed country having a public healthcare service with a central reimbursement negotiation is determined a relevant reduction in the treatment cost purchased by public payers. This is a useful approach to guarantee the affordability of innovative oncology drugs and to contain public expenditures on healthcare. Furthermore, the negotiation of confidential discounts and agreement clauses in managed entry agreements seemed to reward oncology drugs displaying an added therapeutic benefit.

Quality of health economic evaluations submitted to the Italian Medicines Agency: current state and future actions.

OBJECTIVES: The purpose of this study was to evaluate the current state of health economic evaluations (HEEs) submitted by pharmaceutical companies to the Italian Medicines Agency (AIFA) as part of their pricing and reimbursement (P&R) dossiers, and to explore potential future actions in order to enhance their quality. METHODS: All company dossiers submitted from October 2016 to December 2018 were reviewed to select those containing pharmacoeconomic studies. The general characteristics of HEEs were described and their quality assessed based on a checklist adapted from Philips et al. (Review of guidelines for good practice in decision-analytic modelling in health technology assessment. Health Technol Assess. 2004;8: 1-158). RESULTS: Of the 299 dossiers submitted to AIFA, 105 included one or more pharmacoeconomic studies, of which fifty-three were cost-effectiveness analyses. Overall, the compliance of the HEEs with the quality checklist was highly variable: some studies reached high methodological standards whereas others had serious flaws (mean 59.22 percent, range 19.35-90.32 percent). The main weaknesses were the unjustified exclusion of relevant alternatives, poor description and justification of model data and assumptions, and insufficient exploration of uncertainty and study validity. Non-homogeneity across studies was found in study perspectives, discount rates, methods for costing, estimating quality-adjusted life-years and conducting sensitivity analyses. CONCLUSIONS: Based on the results of this study, the recommended actions for increasing the quality of HEEs within reimbursement submissions in Italy are twofold: first, to set methodological standards for conducting and reporting HEEs; second, to strengthen the internal assessment process, also through the acquisition of companies' models and re-evaluation of results. These actions will hopefully provide greater contribution to the evidence-based P&R decision making.

Role of Economic Evaluations on Pricing of Medicines Reimbursed by the Italian National Health Service.

OBJECTIVE: The main objective of this study was to explore the extent to which the incremental cost-effectiveness ratio (ICER), alongside other factors, predicts the final outcome of medicine price negotiation in Italy. The second objective was to depict the mean ICER of medicines obtained after negotiation. METHODS: Data were extracted from company dossiers submitted to the Italian Medicines Agency (AIFA) from October 2016 to January 2021 and AIFA's internal database. Beta-based regression analyses were used to test the effect of ICER and other variables on the outcome of price negotiation (ΔP), defined as the percentage difference between the list price requested by manufacturers and the final price paid by the Italian National Health Service (INHS). RESULTS: In our dataset of 48 pricing and reimbursement procedures, the ICER before negotiation was one of the variables with a major impact on the outcome of negotiation when ≥ 40,000€/QALY. As resulting from multiple regression analyses, the effect of the ICER on ΔP seemed driven by medicines for non-onco-immunological and non-rare diseases. Overall, the negotiation process granted mean incremental costs of €64,688 and mean incremental QALYs of 1.96, yielding an average ICER of €33,004/QALY. CONCLUSIONS: This study provides support on the influence of cost-effectiveness analysis on price negotiation in the Italian context, providing an estimate of the mean ICER of reimbursed medicines, calculated using net confidential prices charged by the INHS. The role and use of economic evaluations in medicines pricing should be further improved to get the best value for money.

Medicine price transparency and confidential managed-entry agreements in Europe: findings from the EURIPID survey.

Pricing of pharmaceuticals is an all-time challenge for healthcare systems. Often public payers agree with companies on confidential managed-entry agreements (MEAs) that, e.g. foresee discounts under specific circumstances. The EURIPID Executive Committee surveyed 22 European countries, who all reported the use of confidential agreements between pharmaceutical companies and public payers, confirming that the actual prices paid are typically lower than the published list price. In 68% of the countries, the confidentiality of MEAs is required by non-disclosure clauses between companies and public payers. In some countries (27%) this is even backed up by a specific law. Our study identified legal constraints for the sharing of information on actual prices and confidential agreements among European countries and consequently restrictions in transparency. In conclusion, the EURIPID survey findings suggest that the current possibility to improve the medicines' price transparency across countries is limited and the issue probably requires international institutional engagement, at least to coordinate initiatives toward a greater collaboration among member states.