In the real world of Italian diabetes centers, prescriptions of incretins have been made in many cases outside the regulatory limits. Nevertheless, when appropriately utilized, incretins may grant results at least in line with pivotal trials.

Earlier patient access to beneficial therapeutics that addresses unmet need is one of the main requirements of innovation in global healthcare systems already burdened by unsustainable budgets. "Adaptive pathways" encompass earlier cross-stakeholder engagement, regulatory tools, and iterative evidence generation through the life cycle of the medicinal product. A key enabler of earlier patient access is through more flexible and adaptive payer approaches to pricing and reimbursement that reflect the emerging evidence generated.

Background: Available scientific literature about open cholecystectomy (OC) and laparoscopic cholecystectomy (LC) does not show univocal results in terms of postoperative pulmonary function. A meta-analysis was carried out to evaluate the postoperative pulmonary function after OC and LC focusing on the Tiffenau index. Methods: Electronic databases were consulted (Cochrane Library, Embase and Pubmed). Standardized mean difference (SMD) with 95% CI was calculated for the Tiffenau Index. The kappa test was performed to evaluate agreement between the reviewers regarding the quality of the selected studies. A sensitivity analysis was carried out to assess the robustness of our study. Heterogeneity among studies was tested by using a χ² test at 0.05 significance level. A random effects model meta-analysis was performed. Results: Meta-analysis of the 13 articles included, resulted in a SMD of 53% (95% CI 0.04, 1.02) for the Tiffenau Index in favor of laparoscopic cholecystectomy. The χ² test analysis showed the presence of heterogeneity among studies (Tiffenau index χ² = 99.97, p = 0.03). The sensitivity analysis confirms the validity of our results. Conclusions: Postoperative pulmonary function is better preserved after laparoscopic cholecystectomy than open cholecystectomy.

ObjectiveThe 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.

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.