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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.

Objectives Anti-TNFα agents have been a staple of Crohn's disease treatment for 20 years, but they have weaknesses. New treatments have more recently become available. The aim of this paper is to examine the Crohn's disease patient population for whom anti-TNF treatments are not preferred and where new mechanisms of action should be considered. Methods A representative sample of 100 Italian physicians with documented expertise with biological treatment of moderate-to-severe Crohn's disease were interviewed. A literature review on Crohn's disease treatment was also conducted to identify patient populations for whom anti-TNFs are unsuitable. Results On the basis of the interviewed physicians, about 9% of moderate-to-severe Crohn's disease patients were noneligible to anti-TNFα due to contraindication or possible risk of intolerance, while 11% had discontinued anti-TNFα treatment due to complications or intolerance/hypersensitivity. Patients with severe heart disease and at high risk of infections were more frequently considered unsuitable. The proportion of patients considered unsuitable among elderly patients and in those with recurrent infections, cancer, and other comorbidities ranged between 40 and 60%. Conclusions We provided additional quantitative and qualitative information to help identify patients who are less suitable to anti-TNF agents, who could benefit from newer biologic agents with different mechanisms of action.

Bladder cancer is one of the most frequent cancers in high-income countries. Information on bladder cancer in Italy is scattered across scientific literature and institutional and educational resources and no attempt has been made yet to organize and summarize this information across various sources of available data. We, therefore, present herein a critical literature review of recent epidemiological and healthcare data, including patients' unmet needs. We undertook a critical review of the scientific and grey literature by exploring several different databases and search browsers. Available official statistics indicate a high burden of bladder cancer in Italy, where this neoplasm has one of the highest incidences worldwide and, in consideration of its relatively high survival, it ranks 4th in cancer prevalence. The limited therapeutic options for muscle-invasive and advanced/metastatic urothelial cancer are one of the major unmet needs for patients with this neoplasm, in Italy and worldwide. Advances in cancer immunotherapy and in understanding molecular biology of bladder cancer are, however, rapidly altering the therapeutic landscape for targeted subgroups of patients with advanced/metastatic disease. Other unmet needs include the low quality of life after radical cystectomy, the lack of widespread clinical pathway schemes to improve and standardize the quality of care and low Italian patients empowerment. Bladder cancer represents a health burden in Italy, with high incidence and prevalence rates, and important unmet needs for patients, including the limited therapeutic options for advanced/metastatic cancers, the low quality of life after radical cystectomy, the lack of widespread clinical pathway schemes, and the low patients empowerment.

Simple Summary The burden of prostate cancer is particularly high in Italy, where the disease ranks 4th among incident cancers overall and is 1st in men. In order to adequately plan health services and targeted interventions for the management of this neoplasm in Italy, it is of utmost importance to have a clear picture of the current number of patients surviving prostate cancer and of their needs in terms of cancer care. In our research, we aim therefore to quantify alive prostate cancer patients in Italy, and to provide relevant information on the number of survivors at different clinical stages of disease. Abstract Understanding the distribution of prostate cancer (PC) at various clinical stages of disease is of utmost importance to quantify the cancer care needs of patients and to adequately plan health services. The aim of this analysis is thus to provide a model-based estimation of the number of prevalent PC patients at different clinical stages in the Italian setting. A simulation model of patient transitions was constructed on a yearly basis using data obtained through a literature review on the incidence, prevalence, progression and mortality of PC, with specific focus on disease stage. A total of 462,570 prevalent PC patients were estimated at 1 January 2019. According to the model, 94.8% of them had non-metastatic PC and 5.2% had metastatic disease. Among the non-metastatic patients, most had T1/T2 PC (85.6%), followed by T3/T4 (10.9%) and T0/Tx PC (3.6%). About 20% of the T3/T4 patients had biochemically recurrent PC. Among the metastatic PC patients, 66.1% had castration-resistant PC and 33.9% had hormone-sensitive PC. This study provided original information on the distribution of PC according to different clinical stages that may be useful to define strategies, understand the PC disease pathway, estimate treatment-related needs and, possibly, plan targeted interventions for public health management of prostate cancer in Italy.

IntroductionDifferent value frameworks (VFs) have been proposed in order to translate available evidence on risk-benefit profiles of new treatments into Pricing & Reimbursement (P&R) decisions. However limited evidence is available on the impact of their implementation. It's relevant to distinguish among VFs proposed by scientific societies and providers, which usually are applicable to all treatments, and VFs elaborated by regulatory agencies and health technology assessment (HTA), which focused on specific therapeutic areas. Such heterogeneity in VFs has significant implications in terms of value dimension considered and criteria adopted to define or support a price decision.MethodsA literature research was conducted to identify already proposed or adopted VF for onco-hematology treatments. Both scientific and grey literature were investigated. Then, an ad hoc data collection was conducted for multiple myeloma; breast, prostate and urothelial cancer; and Non Small Cell Lung Cancer (NSCLC) therapies. Pharmaceutical products authorized by European Medicines Agency from January 2014 till December 2019 were identified. Primary sources of data were European Public Assessment Reports and P&R decision taken by the Italian Medicines Agency (AIFA) till September 2019.ResultsThe analysis allowed to define a taxonomy to distinguish categories of VF relevant to onco-hematological treatments. We identified the “real-world” VF that emerged given past P&R decisions taken at the Italian level. Data was collected both for clinical and economical outcomes/indicators, as well as decisions taken on innovativeness of therapies. Relevant differences emerge between the real world value framework and the one that should be applied given the normative framework of the Italian Health System.ConclusionsThe value framework that emerged from the analysis addressed issues of specific aspects of onco-hematological treatments which emerged during an ad hoc analysis conducted on treatment authorized in the last 5 years. The perspective adopted to elaborate the VF was the one of an HTA agency responsible for P&R decisions at a national level. Furthermore, comparing a real-world value framework with the one based on the general criteria defined by the national legislation, our analysis allowed identification of the most critical point of the current national P&R process in terms ofsustainability of current and future therapies as advance therapies and agnostic-tumor therapies.