Cost of illness of spinal muscular atrophy (SMA) in Italy

The objective of this study was to estimate the indirect and direct non-health costs associated with spinal muscular atrophy (SMA), a disease that burdens the daily life of adults, children and their families in Italy. In order to develop the economic model, a multidisciplinary group of researchers was created to prepare and computerize a questionnaire, which was promoted by SMA families in collaboration with the Economic Evaluation and Heath Technology Assessment center at the University of Rome Tor Vergata. The analysis envisaged a first phase for implementing and validating the questionnaire by the multidisciplinary group. Subsequently, the questionnaire was computerized and sent out to be completed through all the association's distribution channels. The social channels and specific mailing lists were limited exclusively to SMA families. To achieve the sample number required by the research protocol, data collection began on January 8, 2018, and closed on April 15, 2018. Finally, all the data were analyzed using the economic model in order to estimate the average costs per patient.The questionnaire was able to identify a sample of 118 families (22.88% SMA I, 48.31% SMA II, 28.81% SMA III). The average age of the patients was 18.49 years (average age at diagnosis 2.88 years) with more females (55,08%) in the total respondents, taking into account a 4.24% rate of non-respondents. The economic model estimated an average annual cost per patient with SMA of €15.371,41 (€17.683,85 for SMA I, €15.974,78 for SMA II and €12.523,52 for SMA III). Of these costs, about 52% were attributable to indirect costs associated with caregivers, 15% for indirect costs associated with the patient and 4% for social security costs. A total of 17% was attributable to the direct costs incurred by the patient and 12% was attributable to the direct costs incurred by the Italian National Health Service (SSN).To our knowledge, this survey represents the first nationwide analysis estimating the costs incurred by families for the management of SMA. This study highlights the need for specific policies to support families who must live with the disease, not only from the standpoint of their compromised quality of life but also due to the significant economic burden imposed by the disease

Economic evaluation of spondyloarthritis : economic impact of diagnostic delay in Italy

Spondyloarthritis (SpA) is a disease that normally affects the axial skeleton. It progressively leads to overall stiffness up to severe postural deformity of rachis and functional impotence. The objective of the study was to quantify, through an economic model, the impact of specialized testing and pharmacological treatments carried out by the National Health Service (NHS) in normal clinical practice, before the patient is diagnosed with SpA in Italy. In line with the analysis objective, the chosen perspective is that of the NHS

Challenges and solutions to embed cancer survivorship research and innovation within the EU Cancer Mission

We have reached a watershed moment in Europe in our efforts to ensure increased survival and better outcomes for cancer patients. The EU Cancer Mission and the European Beating Cancer Plan together provide an unrivalled opportunity to make significant inroads into a disease that kills over 1.7 million European citizens annually. Harnessing these twin pillars of cancer research and cancer control can be transformative for the European cancer community and in particular for the European cancer patient. However, from a research perspective, in order to fully realise these benefits, we need to ensure that all aspects of the cancer continuum are addressed. Previous research efforts have focussed more on the diagnosis and treatment of cancer, whereas cancer survivorship, to date, has been overlooked. Here, we aim to redress this balance, by identifying the key challenges in cancer survivorship research that need to be addressed and proposing a series of recommended solutions, which, if acted upon, would deliver significant benefits for the nearly 20 million cancer survivors in Europe. To achieve this, we propose the development of a clearly articulated and sustainably funded European Cancer Survivorship Research and Innovation Plan. Embedding this plan within the framework of the EU Cancer Mission would be transformative for cancer survivors and society

Budget impact of bi-monthly use of Cetuximab in patients diagnosed with mCRC

Aim: Cetuximab is used for the treatment of RAS wild-type metastatic colorectal cancer patients. Standard administration schedule is once a week; however, the bioequivalence of an every-other-week (EOW) schedule was demonstrated. Methods: We compared a base case scenario of 100% weekly administration to an EOW at 50 or 100%. Medical examinations, patient management and loss of productivity were considered. Results: Base case was estimated at €100.6 million versus €92.8 million and €84.9 million of EOW 50 and 100%, which showed a cost reduction of 8 and 16%, respectively. Indirect costs accounted for 65% in both scenarios. Conclusion: The adoption of an EOW administration schedule of cetuximab reduced direct and indirect costs substantially

The impact of direct acting antivirals on hepatitis C virus disease burden and associated costs in four European countries

Background and Aims: We assessed the clinical and economic impact of direct-acting antiviral (DAA) therapy for hepatitis C virus (HCV) in England, Italy, Romania and Spain. Methods: An HCV progression Markov model was developed considering DAA eligibility and population data during the years 2015-2019. The period of time to recover the investment in DAAs was calculated as the cost saved by avoiding estimated clinical events for 1000 standardized treated patients. A delayed treatment scenario because of coronavirus disease (COVID-19) was also developed. Results: The estimated number of avoided hepatocellular carcinoma, decompensated cirrhosis and liver transplantations over a 20-year time horizon was: 1,057 in England; 1,221 in Italy; 1,211 in Romania; and 1,103 in Spain for patients treated during 2015-2016 and 640 in England; 626 in Italy; 739 in Romania; and 643 in Spain for patients treated during 2017-2019. The cost-savings ranged from \u20ac 45 to \u20ac 275 million. The investment needed to expand access to DAAs in 2015-2019 is estimated to be recovered in 6.5 years in England; 5.4 years in Italy; 6.7 years in Romania; and 4.5 years in Spain. A delay in treatment because of COVID-19 will increase liver mortality in all countries. Conclusion: Direct-acting antivirals have significant clinical benefits and can bring substantial cost-savings over the next 20 years, reaching a Break-even point in a short period of time. When pursuing an exit strategy from strict lockdown measures for COVID-19, providing DAAs should remain high on the list of priorities in order to maintain HCV elimination efforts

Budget impact analysis of Dalbavancin in the treatment of acute bacterial skin and skin structure infections in three European countries

Background and Objective: Acute bacterial skin and skin structure infections (ABSSSIs) have been defined by the US Food and Drug Administration (FDA) in 2013 to include a subset of complicated skin and skin structure infections commonly treated with parenteral antibiotic therapy. Inpatient treatment of ABSSSIs involves a significant economic burden on the healthcare system. This study aimed to evaluate the economic impact on the National Health System associated with the management of non-severe ABSSSIs treated in hospitals with innovative long-acting dalbavancin compared to standard antibiotic therapy in Italy, Spain, and Austria. Methods: A budget impact analysis was developed to evaluate the direct costs associated with the management of ABSSSI from the national public health system perspective. The model considered the possibility of early discharge of patients directly from the Emergency Department (ED), after 1 night in the hospital, or after two or three nights in the hospital. A scenario with Standard of Care was compared with a dalbavancin scenario, where patients had the possibility of being discharged early. The epidemiological and cost parameters were extrapolated from national administrative databases and from a systematic literature review for each country. The analysis was conducted in a 3-year time horizon. A one-way deterministic sensitivity analysis was conducted to examine the robustness of the results. Results: The model estimated an average annual number of patients with non-severe ABSSSI in Italy, Spain, and Austria equal to 5396, 7884, and 1788, respectively. A total annual expenditure of about €9.9 million, €13.5 million, and €3.4 million was estimated for treating the full set of ABSSSI patients in Italy, Spain, and Austria, respectively. Dalbavancin reduced the in-hospital length of stay in each country. In the first year of its introduction, dalbavancin significantly reduced the total economic burden in Italy and Spain (− €352,252 and − €233,991, respectively), while it increased the total economic burden in Austria (€80,769, 0.7% of the total expenditure for these patients); in the third year of its introduction, dalbavancin reduced the total economic burden in each Country (− €1.1 million, − €810,650, and − €70,269, respectively). Conclusions: The introduction of dalbavancin in a new patient pathway to treat non-severe ABSSSI could generate a significant reduction in hospitalized patients and the overall patient length of stay in hospital

The economic impact of biosimilars in Italy : a scenario analysis

Background: the first generation of biotechnology drugs is reaching, or has already reached, the patent expiry and a large number of biosimilars is entering the Italian pharmaceutical market. The objective of the analysis was to evaluate the economic impacts of biosimilars on the national health expenditure in Italy between 2014 and 2020. Methods: Based on the information deriving from consumption per standard unit and equivalent patients, it was estimated monthly expenditure for some of the biological drugs currently available in Italy that have had or will have a patent expiry within the analysis period (infliximab, etanercept, adalimumab, insulin glargine, trastuzumab, rituximab, bevacizumab and insulin aspart). Pharmaceutical expenditure was calculated on hospital sales prices net of transparent discounts required by law and visible from the AIFA database. Three alternative scenarios have been developed based on the perceptions of a board of clinical experts, pharmacologists and pharmacoeconomists involved in the study. The experts involved analyzed the estimates of treated patients between 2014 and 2017 and reports their hypothetical biosimilar penetration during the period 2018-2020. The results were represented as the difference between the estimated expenditure in the absence of biosimilars and the estimated expenditure in the presence of biosimilars with the real or hypothetical biosimilar penetration. Results: considering the standard units dispensed for each year, the economic model estimate an annual expenditure in 2014 equal to € 1.47 billion for the molecules considered in the analysis. These estimates rise to € 1.54, € 1.50 billion and € 1.51 billion during 2015, 2016 and 2017 in the scenario without biosimilar introduction. Biosimilar introduction generates cost savings between € 3.8 million in 2015 and € 32.9 million in 2017 if compared with the scenario without. Assuming an increasing biosimilar penetration between 2018 and 2020, scenario analysis estimates a cumulative cost reduction equal to € 597 million. Conclusions: Overall, biosimilar penetration generates important cost reduction that could be re-invested in the National Health Sistem

Notulae to the Italian native vascular flora: 6

In this contribution, new data concerning the distribution of native vascular flora in Italy are presented. It includes new records, confirmations and status changes to the Italian administrative regions for taxa in the genera Alchemilla, Arundo, Bupleurum, Clematis, Clinopodium, Cota, Crassula, Cytisus, Euphorbia, Hieracium, Isoëtes, Lamium, Leontodon, Linaria, Lychnis, Middendorfia, Ophrys, Philadelphus, Pinus, Sagina, Sedum, Taeniatherum, Tofieldia, Triticum, Veronica, and Vicia. Nomenclature and distribution updates, published elsewhere, and corrigenda are provided as supplementary material

Notulae to the Italian native vascular flora: 6

In this contribution, new data concerning the distribution of native vascular flora in Italy are presented. It includes new records, confirmations and status changes to the Italian administrative regions for taxa in the genera Alchemilla, Arundo, Bupleurum, Clematis, Clinopodium, Cota, Crassula, Cytisus, Euphorbia, Hieracium, Isoëtes, Lamium, Leontodon, Linaria, Lychnis, Middendorfia, Ophrys, Philadelphus, Pinus, Sagina, Sedum, Taeniatherum, Tofieldia, Triticum, Veronica, and Vicia. Nomenclature and distribution updates, published elsewhere, and corrigenda are provided as supplementary material