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Objective: This paper aims to investigate the duration of the pricing & reimbursement (P&R) procedures submitted in Italy by pharmaceutical marketing authorization holders (MAH) for drugs indicated for rare diseases. Methods: All the data used in this analysis were publicly available on different sources of the Italian Ministry of Health, the Italian Medicines Agency (AIFA) and other official websites. The information was systematically collected to investigate the timeline (days) needed to complete the P&R process. The process was divided into 6 simplified steps and the median and range of days needed for each phase were estimated based on data reported in official/published documents. The analysis was stratified considering every single step of the assessment phase and included segmentation of drugs into indications for rare diseases, Orphan designation, Innovation assessment and Managed entry agreements (MEAs). Results: Overall, 181 first indication procedures were submitted to AIFA in the period considered and, of these, 167 (92.3%) were completed and 129 procedures were considered for the final analysis and the median duration of the entire process (MAH submission to final Gazette publication) was 434 days (range 176.0-918.0). The duration of procedures for rare diseases (n = 53) was longer than those for non-rare-disease procedures (n = 76) (463.0 days vs 407.5 days respectively). Among rare disease procedures, orphan designation and MEAs represent predictors for time prolongation while innovation is associated with a shorter assessment time. Conclusion: The study describes the time spent in each phase of the assessment and the appraisal process and demonstrates that uncertainty represents the main driver for the increment in the overall time.

This document illustrates the results of a discussion of two multi-disciplinary expert panels on pricing and reimbursement of medicines. Experts work in different organizations. The discussion focused on comparator(s), endpoint(s), negotiation of prices of new medicines and/or indications to include in the List 648, as well as the role of cost-effectiveness in the price and reimbursement negotiation. The debate took place during the fourth edition of the Seminari di Mogliano, organized on the 30th of September/1st of October, 2021. The two panels agreed on a general need to enhance interaction among the different stakeholders, in the early assessment and negotiation phases, and to increase the transparency/reproducibility of the decisions taken. The experts have also emphasized the need (i) to improve clarity in the evaluation of additional therapeutic value and the place in therapy with respect to comparators and how comparators are identified; (ii) to create work groups to identify the most appropriate endpoint(s), for each therapeutic area and level of unmet needs; (iii) to provide for a systematic use of cost-effectiveness when an added therapeutic value is delivered by a new medicine. With regard to the 648 List, the experts advocated for an overall reorganization of the current rules governing the special uses of drugs.

Inflammation represents the manifestation of the host's vascular and cellular response to tissue damage, hypersensitivity of the immune system or autoimmunity. As soon as the damage is detected, the mechanisms of localization and removal of foreign substances and damaged tissues are triggered. This response is amplified by the activation of inflammatory cells and by the production of chemical mediators derived from arachidonic acid, vasoactive substances and cytokines. At the ocular level, among the different responses implemented by various inflammation mediators, the action of prostaglandins is mainly expressed in three ways: i) change of intraocular pressure; ii) induction of miosis; iii) increase of protein concentration in the aqueous humor. Ocular inflammation may continue long after the cessation of the initial cause and may also lead to serious consequences, including permanent vision reduction. For this reason, it is essential to establish a correct and effective therapeutic strategy, which allows to control inflammation, the symptoms and the clinical signs associated with it. This article therefore deals with therapeutic strategies to control inflammation, with a particular focus on the role of nonsteroidal anti-inflammatory drugs (NSAIDs) in cataract surgery.

Objectives: To describe the length of time taken by the Italian Medicines Agency (AIFA) to formulate pricing and reimbursement decisions for drugs approved via centralized procedures and to evaluate possible differences between categories of drugs (innovative drugs, oncological and rare disease drugs, products negotiated with Managed Entry Agreements and/or managed via registry). Methods: Monthly meeting reports of the Technical Scientific Committee (CTS) and the Price and Reimbursement Committee (CPR) as well European and Italian Official Journals were scrutinized from January 2015 to January 2018. Results: In the scrutinized period, 85 out of 190 drugs obtained reimbursement and were included in the analysis. Overall, time-to-market (CTS assessment opening to Official Journal) was 258 days. About one third of the whole procedure was dedicated to administrative steps, without significant differences between drug categories. Conclusions: Our analysis shows that transparency makes it possible to determine the length of time taken by AIFA to conclude the Price and Reimbursement (P&R) process. It also suggests that administrative procedures may still be optimized to allow faster access to treatments for patients and provides a starting point to determine future policies in this area.