2025-10-23
Gianni Ghetti, Eleonora Spiombi, Lucrezia Amoroso, Raffaella Viti, Marco Spada
BACKGROUND AND AIM: Fabry disease is a rare X-linked lysosomal hereditary disease caused by pathogenic variants in the GLA gene that results in deficient α-galactosidase A enzyme activity. Enzyme replacement therapy (ERT) remains a primary approach to address the enzymatic defect and its pathophysiological impacts. This study aimed to evaluate the annual treatment costs associated with agalsidase alfa, agalsidase beta and pegunigalsidase alfa within the context of the Italian National Healthcare Service (NHS). METHODS: A cost comparison model was developed to estimate the treatment-related expenditure for agalsidase alfa, agalsidase beta, and pegunigalsidase alfa over 1-year time horizon from the hospital perspective. The analysis accounted for drug acquisition and administration costs across hospital-based, home-based, and self-administration settings. Infusion durations were estimated based on product specifications and patient characteristics from published literature. Costs were calculated using adjusted ex-factory list prices and literature-sourced hourly rates for healthcare professionals. Expert validation ensured model accuracy, and sensitivity analyses examined the impact of varying infusion scenarios. RESULTS: In the base case, agalsidase alfa demonstrated the lowest annual treatment cost (€172,395), followed by pegunigalsidase alfa (€173,744), while agalsidase beta incurred the highest cost (€191,143). Scenario analysis confirmed that agalsidase alfa offers a more sustainable alternative compared to agalsidase beta, while demonstrating comparable costs to pegunigalsidase alfa. Furthermore, the analysis indicated that variations in infusion settings had impact on overall outcomes, depending on the home or hospital preference. CONCLUSION: This economic evaluation suggests that agalsidase alfa may offer cost advantages relative to other ERTs, particularly in comparison to agalsidase beta. These advantages are primarily driven by lower annual treatment costs. In addition, agalsidase alfa’s unique approval for self-administration in Italy, has the potential to reduce healthcare expenditures, optimizing hospital resource allocation, and enhancing the efficiency of healthcare delivery for patients with Fabry Disease.
2025-10-10
Ippazio Cosimo Antonazzo, Paolo Angelo Cortesi, Antonio Silvani, Pietro Ferrara, Giorgia Gribaudo, Lorenzo Losa, Lorenzo Giovanni Mantovani
INTRODUCTION: Grade 4 glioma is among the most aggressive forms of central nervous system tumors, posing a substantial clinical, humanistic, and economic burden on patients, caregivers, and society. Recent clinical and observational studies have demonstrated the efficacy and safety of Tumor Treating Fields (TTFields), in combination with temozolomide (TMZ), as a treatment option for patients with grade 4 glioma (TTFields-TMZ). However, this therapeutic strategy is not currently reimbursed in Italy. This treatment is currently reimbursed by the Regional Health Services/Health local authorities on a case-by-case basis, leading to inconsistent access nationwide. Therefore, the objective of this study was to assess the cost-effectiveness of TTFields-TMZ compared to TMZ monotherapy. METHODS: A partitioned survival model was developed from the perspective of the Italian National Healthcare Service (NHS), adopting a lifetime time horizon. The model estimated total costs, life years (LYs), quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER). Demographic and clinical characteristics of the simulated cohort were primarily derived from the pivotal EF-14 trial. Other model parameters (e.g., adverse event rates, survival probabilities, utility values) were obtained from the EF-14 trial and supplemented by literature review. Treatment costs, as well as those related to adverse event management and disease progression, were calculated based on Italian national and regional reimbursement tariffs. One-way sensitivity analysis and probabilistic sensitivity analysis (PSA) were performed to evaluate the impact of parameter uncertainty. RESULTS: TTFields-TMZ was associated with an overall cost of €186,386, compared to €40,743 for TMZ monotherapy. TTFields-TMZ yielded 3.82 LYs and 2.99 QALYs, whereas TMZ monotherapy yielded 2.12 LYs and 1.65 QALYs. This resulted in an ICER of €83,221 per LY gained and €109,026 per QALY gained. Sensitivity analyses confirmed the robustness of the base-case findings. CONCLUSIONS: The findings align with the QALY threshold typically applied by international Health Technology Assessment (HTA) bodies for rare disease treatment and offer robust evidence to inform healthcare decision-makers regarding the adoption of TTFields-TMZ for patients with grade 4 glioma, thereby supporting the efficient allocation of healthcare resources.
2025-07-29
Giorgio Lorenzo Colombo, Giuseppe Novelli
OBJECTIVE: To assess the economic impact of two preventive strategies against respiratory syncytial virus (RSV) infection in Italian newborns: universal administration of nirsevimab versus a complementary approach combining maternal RSVpreF vaccination and selective use of nirsevimab. METHODS: A cost-minimization analysis was conducted over a 3-year time horizon, based on the assumption of equal clinical efficacy between the two strategies, as supported by published evidence. The analysis compared: (1) the exclusive use of nirsevimab in all newborns, and (2) a complementary strategy with RSVpreF vaccination during pregnancy for infants born in the RSV season (October–March), combined with targeted use of nirsevimab for extremely and very preterm infants and for those born outside the protective window of maternal immunization. Only drug acquisition costs were included, assuming equivalent healthcare resource use in both scenarios. Epidemiological and cost data were derived from national birth statistics, literature, and public pricing sources. RESULTS: Assuming a birth cohort of 379,890 infants per year and 70% coverage, the annual cost of universal nirsevimab administration (Scenario 1) was €61,162,290, amounting to €183,486,870 over three years. In the complementary strategy (Scenario 2), annual costs were €55,062,016, totaling €165,186,049. This represents a saving of €6.1 million per year and €18.3 million over three years, equivalent to a 10% reduction in expenditure. CONCLUSIONS: In the Italian context, a complementary RSV prevention strategy combining maternal vaccination with targeted neonatal immunization may offer substantial cost savings for the national health system, without compromising clinical efficacy. These findings support the implementation of integrated and flexible immunization programs and highlight the potential role of maternal RSV vaccination in enhancing both sustainability and effectiveness of public health strategies.
2025-06-13
Lorenzo Giovanni Mantovani, Ippazio Cosimo Antonazzo, Paolo Angelo Cortesi
BACKGROUND: Venous thromboembolism (VTE), which includes deep vein thrombosis (DVT) and pulmonary embolism (PE), is a significant health problem with especially increased prevalence, morbidity and mortality in patients with cancer. This study aimed at assessing the economic impact of tinzaparin in patients with cancer associated thrombosis (CAT). METHODS: A budget impact model (BIM) was developed to assess the economic impact of tinzaparin as treatment for patients with CAT. The analysis was conducted over a 3-year time horizon and by adopting the Italian Healthcare system perspective. The model estimated and compared direct medical costs associated with tinzaparin (scenario with tinzaparin) to the ones associated without tinzaparin (scenario where only enoxaparin is available). Epidemiological data as well as VTE events’ rates were retrieved from literature, while costs data were retrieved from the Italian rate tables. The model estimated the economic impact as well as the economic variation associated with drug wastage and VTE management. RESULTS: The model estimated 2,090, 4,202 and 5,429 patients potentially eligible to the treatment during the first, second and third year, respectively. The use of tinzaparin resulted in a cost saving of about €3 millions over 3 years (−€446,378 during the first, −€1,025,848 during the second, and −€1,657,508 during the third year). In the same timeframe, the use of tinzaparin also resulted in decreased costs associated with drug wastage (−€738,604) and recurrent VTE management (−€404,470). CONCLUSIONS: Tinzaparin for the management of CAT patients has the potential for substantial savings, compared to treatments currently available. Stakeholders may consider these data to improve healthcare resource allocation in the Italian setting
2025-02-28
Linet Odonde, Lorenzo Pradelli, Claudio Micheletto
BACKGROUND: The benefits of triple therapy for Chronic Obstructive Pulmonary Disease (COPD) have been established in two 52-week phase-3 randomized controlled trials. The current systematic literature review (SLR) appraised available evidence on the real-world outcomes of single-inhaler triple therapy (SITT) when prescribed by general practitioners (GPs). METHODS: Using the PICOS (Population, Intervention, Comparator, Study design) framework, a literature search was conducted to identify suitable studies for the review. PubMed and Embase databases were searched, and Rayyan was used to screen articles for inclusion. The study selection followed the Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines. RESULTS: 1,379 non-randomized clinical studies published between 2020 and 2024 were identified from the literature search. 1,367 articles were later excluded from the review (duplicates: n = 119; not eligible: n = 1,248), leaving 12 studies for inclusion. 10 studies were single-armed, while 2 were comparative. 7 studies were from Germany, 3 from the United Kingdom, and 1 each from France, Greece, and Belgium. The SITT combinations studied were beclomethasone dipropionate/formoterol fumarate/glycopyrronium bromide (n = 9) and fluticasone furoate/umeclidinium bromide/vilanterol (n = 3). One study did not specify this. One study reported on inhaler use, 8 on adherence, 2 on drug persistence, 5 on lung function, 3 on exacerbations, and 6 on health-related quality of life. CONCLUSIONS: We found evidence of possible benefits for SITT prescribed by GPs on real-world outcomes. Nevertheless, the evidence is limited in quantity and quality, and future real-world studies need to confirm our findings.
2025-01-31
Valentina Perrone, Stefania Mazzoni, Maria Cappuccilli, Margherita Andretta, Marcello Bacca, Antonietta Barbieri, Fausto Bartolini, Alessandro Chinellato, Andrea Ciaccia, Alberto Costantini, Francesco De Vita, Stefania Dell'Orco, Fulvio Ferrante, Simona Gentile, Stefano Grego, Daniela Mancini, Marzia Mensurati, Rossella Moscogiuri, Romina Pagliaro, Nicola Petragnani, Cataldo Procacci, Davide Re, Fiorenzo Santoleri, Michele Giuseppe Tari, Loredana Ubertazzo, Carlo Giuseppe Antozzi, Luca Degli Esposti
AIM: This analysis investigated the population with Myasthenia Gravis (MG) in Italy, to describe epidemiology, mortality, patients’ characteristics, comorbidity profile, therapeutic management, and healthcare consumption and related costs. METHODS: From 2012 to 2021, MG patients were identified in administrative flows of healthcare entities through hospitalization discharge diagnosis or exemption code for MG or a pyridostigmine prescription. Medications and comorbidities were searched before inclusion and healthcare costs were analysed at 1-year follow-up. Epidemiology estimates were reported as cases/100,000 people, and mortality rates, stratified by age classes and gender, were assessed during 2019. MG patients were compared with age- and gender-matched subjects without MG. RESULTS: At the end of 2021, MG prevalence was 35.1/100,000 and incidence 4.7/100,000 people. Mortality in 2019 was 3.2% in overall MG sample, and tended to rise in males and elderly patients. About 90% received MG-related treatments, namely pyridostigmine, corticosteroids and immunosuppressants (81.3%, 76.9% and 26.1% of patients respectively). Unsurprisingly, the yearly healthcare resource consumption/patient was higher in MG patients than in non-MG subjects (p<0.001), resulting in increased annual direct costs for MG patients (€5,495 vs €823, p<0.001), related to expenses for hospitalizations (mostly related to nervous system and respiratory system), drugs and outpatient services. CONCLUSIONS: In this study, incidence and prevalence of MG estimated in Italy were similar to other European countries and mortality rates were from 2 to 3-times higher than general population. Despite the current treatment options and adherence to guidelines for MG management, the clinical and economic burden of the disease remains high.
2024-12-17
Lorenzo Mantovani, Paolo Angelo Cortesi, Michele Molina, Laura Santoni, Luca Prosperini
2024-11-19
Alfredo Del Gaudio, Assunta Caiazza, Luana Bortone, Sonia Tomasso, Ilaria Bozzari, Laura Vincenzi, Gianluca Furneri, Luigi Tritapepe
INTRODUCTION: Prolonged administration of norepinephrine to critically ill patients can lead to serious adverse events. In this context, the concept of “decatecholaminization” has emerged over the past decade, involving the association of vasopressin with norepinephrine to reduce catecholamines need. Additionally, beta-blockers can help prevent increased heart rate resulting from sepsis treatments. This study presents an economic analysis evaluating the economic implications of the decatecholaminization use in patients with septic shock treated in intensive care unit (ICU) from the Italian National Health Service (NHS) perspective. METHODS: Two analyses were conducted: (1) a patient-level comparison of costs between two real-world cases, one treated with decatecholaminization and one without this approach, and (2) a cohort-level analysis using a pharmacoeconomic model to project cost differences for the Italian National Health Service (NHS) before and after implementing decatecholaminization. RESULTS: In the patient-level analysis, the use of decatecholaminization results in increased pharmacological expenses (+€210), and cost reduction in resource utilization (-€30,412). Similarly, the cohort-level shows higher pharmacological costs (+€192 per patient) and lower cost for other resources (-€1,264 per patient) in the future vs current scenario, resulting in a cumulative cost reduction of -€1,072 per patient. Considering an eligible population of 39,207 patients, decatecholaminization results in a total cost reduction of approximately €42.4 million. CONCLUSION: This analysis supports the economic viability of decatecholaminization as an effective treatment for comprehensive management of septic shock. Further evaluation in real-world settings is needed to validate these findings and optimize clinical application.
2024-05-31
Gaetano Senatore, Emilio Di Lorenzo, Orietta Zaniolo, Giovanni Liuzzi
INTRODUCTION: The manuscript describes the long-term cardiac rhythm monitoring system, known as Reveal LINQ, along with an analysis of the evolution in the Italian cardiology practice that its introduction has brought and may still bring given the smaller size and the implantable procedure, performed even by nursing personnel. METHODS: The project was articulated, firstly, to understand how the Reveal LINQ implantation is organized in 4 different Italian hospital facilities and to estimate the associated costs. For each of the involved facilities, data on the operating room type, length of hospital stay, resource consumption, and unit costs associated with the implantation procedure were collected. Direct costs for the hospital were calculated, including personnel work, materials used, and overhead. After that, we identified the ideal pathway (IP) that may enhance and optimize efficiency and resources consumption, without compromising the quality of care. The hypothetical cost savings resulting from the adoption of IP were estimated. RESULTS: According to our analysis, total cost per procedure ranges between 2,500 and 3,100 Euros, depending on the hospital facility. The IP is based on the use of an outpatient setting in which trained nursing personnel performs the implantation procedure. The shift from current clinical practice to ideal setting has the potential to decrease administrative costs, overhead expenses, and working time, leading to an average cost saving of 333 euros per procedure. It varies between 115 and 650 Euros, according to the starting background of each facility. CONCLUSIONS: The article concludes by emphasizing the introduction of a dedicated outpatient tariff as key incentive for the transition toward the ideal pathway and, consequently, for enhancing the overall efficiency of the Health Service.
2024-04-22
Orietta Zaniolo, Silvia Maffezzoli, Massimiliano Povero, Lorenzo Pradelli
INTRODUCTION: Long-acting injectable antipsychotics (LAIs), due to a lower frequency of administration, could address the well-established challenge of non-adherence to oral ones. We conducted a Network Meta-Analysis (NMA) to assess the relationship between administration frequency and effectiveness. The recent introduction of a semi-annual paliperidone palmitate formulation, along with the arrival of monthly paliperidone generics, could emphasize LAI’s advantages. Aim of this paper is to present the results derived from an updated version of our previous NMA. These results are used to fuel a budget impact model built to evaluate the economic implications of optimizing dosing intervals. METHODS: We compare the current distribution of patients among available LAI active substances and dosing frequencies with an optimized scenario. In this scenario, 20% of patients are switched to the next permitted regimen with a longer inter-dosing interval. Drug acquisition costs and relapse management costs are taken into account over a one-year simulation period; these last items are estimated by means of the event rates obtained from the updated meta-analysis. The optimized scenario incorporates the reduced cost resulting from the expiration of patents. RESULTS: Throughout the analysis, a total of 11,600 patients were able to switch from shorter to longer dosing intervals, leading to an overall optimization of quality of care. The greater expenditure incurred by the Italian National Health Service (NHS) in the acquisition of newer and longer-lasting drugs is offset by savings associated with the arrival of generics of monthly paliperidone palmitate and the shift toward less-relapsing regimens. The net impact on the NHS budget is a saving of more than 19 million Euros. CONCLUSION: This economic saving has the potential to initiate a virtuous process: it could be reinvested to fund a further shift from oral daily therapies, which are less expensive but marked by poor compliance, to LAIs. According to our simulation, nearly 40 thousand patients could undergo this transition, without additional expenses for the NHS.
2023-05-15
Francesco Mennini, Daniele Rossi, Andrea Marcellusi
L’incontinenza urinaria (IU) è un sintomo frequente a seguito di prostatectomia radicale per carcinoma della prostata, dal momento che interessa quote elevate di pazienti, che variano a seconda della definizione, delle tempistiche di valutazione, dell’approccio chirurgico e di chi effettua la rilevazione e che possono anche superare l’80% dei pazienti [1]. Nella maggior parte dei casi, il problema si risolve a distanza di un anno. Tuttavia, l’incontinenza permane anche dopo 12 mesi in una quota non trascurabile di pazienti, che varia a seconda delle statistiche e che si attesta intorno a percentuali pari al 10% [1]. L’IU, oltre a rappresentare un problema di tipo igienico e sociale, compromette fortemente la qualità della vita del soggetto che, dopo aver superato il trauma e la paura della patologia oncologica, vive sentimenti di perdita di autostima, associati ad alterazioni nelle relazioni familiari, sociali e nel rapporto col partner, che interessano anche la vita lavorativa [2,3]. Il tavolo tecnico sull’incontinenza – istituito presso il Ministero della Salute – ha definito un percorso basato su evidenze cliniche, che risponde alla necessità di prendersi cura del paziente con IU dopo chirurgia prostatica [4]. Tra le diverse criticità rilevate sul territorio nazionale, emerge il mancato accesso alla terapia chirurgica dell’IU, che non viene proposta perché non effettuata nel centro di cura oncologica, per la limitata proposta di soluzioni terapeutiche offerte al paziente dal chirurgo e per la limitata disponibilità di presidi chirurgici, anche in relazione ai costi. Nel caso dell’IU da sforzo da incompetenza sfinterica dopo prostatectomia radicale, l’opzione dello sfintere urinario artificiale (AUS) rimane il gold standard per l’IU moderata-grave (raccomandazione B ICI, livello di evidenza 2b EAU). Tale giudizio è confermato anche dalla Regione Veneto che, con il Decreto del Direttore generale dell’Area Sanità e Sociale n. 58 del 15.06.2016, ha emanato le “Linee di indirizzo regionali per l’utilizzo di sfinteri urinari artificiali, sling e neurostimolatori sacrali nell’incontinenza urinaria e fecale”, con cui si stabilisce che «Lo sfintere urinario artificiale deve essere offerto agli uomini con incontinenza da sforzo (SUI) post-prostatectomia da moderata a severa, a seguito di fallimento della terapia conservativa» [5]. Il paradosso dell’impianto di AUS è che, pur essendo una soluzione terapeutica eccellente e costo-efficace per i pazienti con incontinenza urinaria da moderata a grave, la maggioranza dei candidati non ne ha accesso. Come evidenziato dalla analisi delle Schede di Dimissione Ospedaliera (SDO) 2016 [6], in Italia vengono effettuati non più di 240 impianti all’anno a fronte di una stima media di circa 1.000 pazienti/anno che vivono con incontinenza non risolta con terapia conservativa. Tali numeri dimostrano un evidente sottoutilizzo della tecnologia di elezione per questi pazienti. Le barriere all’adozione della soluzione chirurgica sono diverse; tra di esse si annoverano la scarsità di informazione adeguata ai pazienti e la mancata allocazione di risorse – economiche e professionali – adeguate a garantire la procedura: spesso gli ospedali hanno pochissime unità/professionalità disponibili, con lunghe liste d’attesa, e un rimborso inadeguato a coprire i costi della procedura da parte del Servizio Sanitario Regionale/Nazionale (SSR/SSN) [7]. Questi fattori disincentivano il ricorso all’impianto dell’AUS anche laddove clinicamente appropriato, determinando un trattamento insufficiente e inadeguato della popolazione eleggibile. Alla luce dell’analisi HTA condotta, sarebbe auspicabile un intervento normativo che riconosca dignità e valore all’impianto dell’AUS post-prostatectomia radicale, laddove la terapia farmacologica si rivelasse insufficiente alla soluzione del problema. L’analisi ha dimostrato come incrementando il numero di soggetti trattati con AUS (+29 pazienti trattati con il gold standard AMS 800TM [8]) rispetto alla terapia conservativa si verifichi un importante incremento della qualità di vita dei pazienti (rapporto di costo-efficacia altamente positivo e in alcuni casi dominante) accompagnato da una consistente riduzione del costo legato alla terapia conservativa (pannoloni). Si tratterebbe di un numero limitato di procedure, che consentirebbe, però, di migliorare notevolmente la qualità della vita dei pazienti interessati da questa condizione.