Farmeconomia. Health Economics and Therapeutic Pathways
eISSN: 2240-256X h5-index: 7 Farmeconomia. Health Economics and Therapeutic Pathways is an international, open access, double-blind peer-reviewed journal. It is indexed in: CrossRef Web of Science (ESCI) Google Scholar OpenAIRE Open policy finder The aim of the journal is to provide content of the highest quality, authority, and accessibility. Particular emphasis is placed on the evaluation of new drugs and pharmacoeconomic models, but the journal also publishes economic analyses of all types of healthcare interventions and health policy initiatives, evaluations of methodologies, and analyses of pricing and reimbursement systems. The readership includes health economists, market access experts, and, more broadly, all those with an interest in pharmacoeconomics, health economics, health policy, and public health. Publication fees: Upon acceptance of an article, authors are required to pay the Article Publication Charge (APC) of €1,485.00. The journal considers fee discounts and waivers for authors from developing countries on a case-by-case basis. If you would like to request a discount or waiver, please do so ahead of submission. Fast-Track Review: A fast-track review option is available, providing peer-review comments within 10 business days of submission for an additional fast-track fee (€835). Please note that this payment is strictly to facilitate the accelerated review process and does not guarantee acceptance or publication of the article in any way. If you wish to use this option, please select the ‘fast track review’ choice when submitting your article. Contact: For more information about the journal, including pre-submission enquiries, please contact Ombretta Bandi at o.bandi@seedstm.com
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Cost Evaluation and Economic Sustainability of Enzyme Replacement Therapies for Fabry Disease in Italy from the Hospital Perspective
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.
Copyright (c) 2025 Gianni Ghetti, Eleonora Spiombi, Lucrezia Amoroso, Raffaella Viti, Marco Spada
[Complementary Strategy of RSVpreF Vaccination/Nirsevimab versus Nirsevimab Alone for the Prevention of RSV Infection in Infants in Italy: A Cost-Minimization Analysis]
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.
Copyright (c) 2025 Giorgio Lorenzo Colombo, Giuseppe Novelli
Tinzaparin for the Management of Cancer Associated Thrombosis in Patients with Cancer: a Budget Impact Analysis from the Italian Healthcare System Perspective
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
Copyright (c) 2025 Lorenzo Giovanni Mantovani
[Cost-Effectiveness Analysis of TTFields with TMZ Versus TMZ in Monotherapy in Patients with Grade 4 Glioma in Italy]
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.
Copyright (c) 2025 Ippazio Cosimo Antonazzo, Paolo Angelo Cortesi, Antonio Silvani, Pietro Ferrara, Giorgia Gribaudo, Lorenzo Losa, Lorenzo Giovanni Mantovani
[Cost Evaluation of First-Year Treatment for Locally Advanced or Metastatic Urothelial Carcinoma with Chemotherapy Plus Avelumab vs Chemotherapy Plus Best Supportive Care: A Cost of Care Model]
BACKGROUND: Avelumab is indicated as monotherapy for the first-line maintenance treatment of adult patients with locally advanced or metastatic urothelial carcinoma (la/mUC) who are progression free following platinum-based chemotherapy. This study aimed to estimate the direct medical costs of treating patients with la/mUC, from Italian National Health Service (NHS) perspective. METHODS: A model was developed to estimate the healthcare costs for the first year of treatment with platinum-based chemotherapy as induction therapy followed by maintenance with avelumab and best supportive care (BSC) (avelumab + BSC) vs platinum-based chemotherapy as induction therapy followed by best supportive care (BSC). At the end of firstline treatment (including maintenance) or at disease progression, patients may receive subsequent active treatment or BSC. Estimates of patient survival is determined by the published overall survival (OS) of the systemic treatment received in the first-line setting. Costs and tariffs valid as of 2024—including drug acquisition and administration, disease management and adverse event (AE) management—were calculated, including subsequent therapy costs, according to treatment duration, AEs risk, disease monitoring, progression-free survival and OS. Unit costs used are ex-factory prices net of mandatory price reductions for drugs and reimbursement rates, and DRGs for healthcare services and admissions. RESULTS: While the introduction of avelumab as first-line maintenance therapy is associated with an increase in annual drug expenditure in this setting (49,710€), the overall impact on healthcare expenditure is relatively modest (21,241€/year). The avelumab + BSC arm demonstrated significant reductions in costs related to subsequent therapies (−22,385€/year) and disease monitoring and management (−6,287€/year). The impact on drug administration costs was minimal (126€/year), as was the incremental cost related to AEs (77€/year), highlighting avelumab’s favorable tolerability profile. CONCLUSIONS: The estimated increase in direct healthcare costs resulting from the use of avelumab as a first-line maintenance treatment, support its use as SoC in patients who receive 1L platinum-based chemotherapy and are progression free. Understanding the economic burden associated with la/mUC treatments may facilitate informed decisions, their selection and optimal sequencing
Copyright (c) 2025 Daniela Paola Roggeri, Alessandro Roggeri, Anna Maria Provenzano, Francesca Ludovica Boffo, Roberto Iacovelli
Retifanlimab Vs Avelumab in Patients with Metastatic Merkel Cell Carcinoma: A Cost Utility Analysis in Italy
OBJECTIVE: Merkel cell carcinoma (MCC) is a very rare and aggressive neuroendocrine skin cancer, characterized by a 5-year survival rate of 13.5% in patients with distant metastases. This study aimed to evaluate the cost-utility of retifanlimab compared to avelumab in the treatment of metastatic MCC patients who had not received prior systemic therapies, from the perspective of the Italian National Health Service (SSN). METHODS: A 7-day cycles partitioned survival model with three mutually exclusive health-states—progression-free, post-progression and death—was developed to compare lifetime clinical outcomes and costs for patients treated with retifanlimab versus avelumab in the Italian context. Progression-free survival and overall survival curves were modelled independently, with POD1UM-201 trial data used for retifanlimab efficacy. In the absence of direct head-to-head clinical trial data, avelumab efficacy was estimated using the hazard ratio obtained from a matching-adjusted indirect comparison. Following a previous National Institute for Health and Care Excellence submission, utility values were derived using a timeto-death approach, with health states defined as “>266 days to death”, “35-266 days to death”, and “<35 days to death”. Direct healthcare costs, including drug acquisition and administration, disease monitoring, adverse event management, post-progression therapy, and end-of-life care, were sourced from Italian data. Costs and health outcomes were discounted at an annual 3% rate. Deterministic and probabilistic sensitivity analyses, along with scenario analysis, were conducted to assess the uncertainty of input parameters. RESULTS: In the base case, retifanlimab demonstrated greater efficacy compared to avelumab, with 6.39 vs 3.42 life-years and 5.11 vs 2.68 quality-adjusted life-years (QALYs), at an additional cost of €12,228. The incremental cost-utility ratio was estimated at €5,037 per QALY gained. Sensitivity analyses confirmed the robustness of the base case results. CONCLUSIONS: Retifanlimab can be considered a cost-effective option for Italian patients with metastatic MCC who have not received prior systemic therapies.
Copyright (c) 2025 Gianni Ghetti, Camilla Porta, Massimiliano Povero
A Real-World Analysis of Patients with Triple Class Exposed Multiple Myeloma in Italy: Epidemiology Estimates, Treatment Pattern and Economic Burden
OBJECTIVES: This research aimed to provide updated epidemiological estimates of multiple myeloma (MM) in Italy and to characterize the clinical journey, treatment patterns, and economic burden focusing specifically on the subset of patients who have been exposed to all three major therapeutic classes: proteasome inhibitors, immunomodulatory drugs, and antiCD38 monoclonal antibodies (triple-class exposed, TCE). METHODS: A retrospective analysis was conducted using Italian healthcare administrative databases covering 12 million individuals. The research included (i) an epidemiological analysis of MM prevalence and incidence, and (ii) a longitudinal analysis of TCE patients identified between January 2017 and June 2023. Inclusion criteria required exposure to the three major MM drug classes. Healthcare resource utilization (HCRU) and direct costs from the Italian National Health Service perspective were assessed at one-year follow-up. RESULTS: As of August 2022, MM incidence was 9/100,000 and prevalence 40.9/100,000; TCE prevalence was estimated at 4.1/100,000, projecting to 2,557 TCE patients in Italy. From 6,102 MM patients, 894 were identified as TCE; 887 had sufficient follow-up for inclusion. TCE patients had a mean age of 67 years and a mild comorbidity burden (Charlson Index = 0.7). Among 309 recent TCE cases (2022–2023), 35.6% became TCE in first-line therapy and 46.5% in second-line. HCRU analysis (n=461) showed high service use, with annual per-patient costs averaging €119,899—88% attributable to MM-related drugs. CONCLUSIONS: This real-world analysis highlights a growing population of TCE MM patients in Italy, with increasing exposure to combination therapies earlier in treatment. The findings underscore the substantial clinical and economic burden posed by this population, reinforcing the need for novel therapeutic options to improve outcomes and manage costs within the healthcare system.
Copyright (c) 2025 Valentina Perrone, Stefania Mazzoni, Maria Cappuccilli, Roberto Di Virgilio, Luca Degli Esposti
Designing a Model for the Management of Modern Health Services of Middle-aged Adults in the Iranian Health System
BACKGROUND: Middle adulthood can represent a peak stage of life if health is addressed comprehensively across all dimensions. This study aimed to develop an appropriate model for managing modern health services for middle-aged adults within the Iranian health system in 2019. METHODS: This mixed-method study was conducted in two phases. In the qualitative phase, the participants were 21 policymakers and experts involved in the middle-aged adults’ program at the Ministry of Health as well as provincial and county health centers, selected through purposive sampling. In the quantitative phase, 431 executives of the middle-aged adults’ program in Ardabil Province were enrolled. Data were analyzed using SPSS version 20 (Chicago, IL, USA) and Amos software. RESULTS: The mean age of participants was 35.41 ± 6.94 years. The Content Validity Ratio (CVR) was 0.79, and Cronbach’s alpha for the questionnaire was 0.95. Six key components of health service management were identified: human resources, financial resources, executive management, health service package, essential medicines and equipment, and community participation. CONCLUSIONS: The proposed model was most strongly influenced by human resources and the health service package, whereas community participation had the weakest impact. This model can serve as a guide for policymakers and health authorities to improve health indicators among middle-aged adults in Iran.
Copyright (c) 2025 akbar shokri, fatemeh dabbaghi, azadeh ahmadidashtian, ghahraman mahmoodi
Real-World Outcomes of Single-Inhaler Triple Therapy Prescribed by General Practitioners in Managing Chronic Obstructive Pulmonary Disease: A Systematic Literature Review
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.
Copyright (c) 2025 Linet Odonde, Lorenzo Pradelli, Claudio Micheletto
[Pharmacoeconomic Comparison Between Dupilumab and Tralokinumab for the Treatment of Atopic Dermatitis in Italy: A Cost-per-Responder Analysis]
OBJECTIVE: A cost-per-responder model was developed to compare the pharmacoeconomic value of dupilumab and tralokinumab, in combination with topical corticosteroids (TCS), for treating atopic dermatitis (AD) in Italy. METHODS: An indirect treatment comparison (ITC) was conducted using placebo arms from the LIBERTY AD CAFÈ, LIBERTY AD CHRONOS, and ECZTRA studies as a common comparator to assess the efficacy of dupilumab versus tralokinumab in adult AD severe patients with inadequate responses to cyclosporine. Response was evaluated at 16 weeks using different clinical outcomes: proportion of patients achieving Eczema Area and Severity Index (EASI) improvement by more than 50/75/90%, ≥4-point improvement in Itch Numeric Rating Scale (I-NRS 4), and Investigator’s Global Assessment of clear/almost clear skin score (IGA 0/1). Number Needed to Treat (NNT) associated with each efficacy outcome was calculated for dupilumab and tralokinumab (versus best supportive care [BSC]) and multiplied by the 16-week cost of treatment in order to estimate the cost per additional responder over this period. Since ex-factory prices of dupilumab and tralokinumab may not reflect the actual transfer prices charged to healthcare facilities, a scenario analysis was conducted assuming hypothetical additional discounts between 0 and 60%. Finally, an additional scenario analysis was implemented by estimating the response rate at 52 weeks. RESULTS: Dupilumab was more effective than tralokinumab across all assessed efficacy indicators. In the base case analysis, using EASI 50 as the efficacy indicator, the NNT for dupilumab was 3 patients compared to 8 patients for tralokinumab. The 16-week cost per responder was €15,595 for dupilumab and €41,587 for tralokinumab, resulting in a saving of €25,992 per patient treated effectively. In all scenario analyses, considering other efficacy indicators, dupilumab was found to be more cost-effective than tralokinumab, with savings ranging from €26,000 to €47,000 per responder, depending on the efficacy indicator considered. Dupilumab confirmed its economic advantage for all possible price reduction combinations with savings ranging from €1,000 to €56,000. The same results were also found in the 52-week endpoint analysis. Finally, assuming a fixed budget of €38 million, 1,725 additional patients can be treated effectively every year with dupilumab instead of tralokinumab. CONCLUSIONS: The findings of this study demonstrate that dupilumab provides significantly greater efficacy improvements and economic benefits compared to tralokinumab in AD patients. These results underscore the pharmacoeconomic advantages of dupilumab for managing severe AD in Italy, emphasizing its potential as a preferred treatment option for healthcare decision-makers.
Copyright (c) 2025 Luca Castello, Massimiliano Povero, Paolo Gisondi, Andrea Chiricozzi, Marcello Pani, Maria Paola Pedone, Kerry Noonan, Zhixiao Wang, Roberta Di Turi
[Horizon Scanning Of Therapeutic Alternatives For Generalized Myasthenia Gravis And Five-Year Healthcare Expenditure Forecast Model]
OBJECTIVE: The objective of the analysis was to assess and quantify the economic impact of generalized Myasthenia Gravis (gMG) in Italy over the next five years (2025—2029) using Horizon Scanning and expenditure forecasting methodologies. METHODS: The analysis is characterized by conducting Horizon Scanning of therapies for gMG that will be reimbursed in the Italian market over the next five years (2025—2029), followed by the development of a disease expenditure model for the same time frame. The Horizon Scanning methodology for gMG included research on ClinicalTrials.gov to identify relevant Phase 2 or 3 studies. A forecasting model was developed to evaluate the impact of new therapies for gMG from the perspective of the Italian National Health Service, considering total patient management costs. The analysis included acquisition and administration costs of treatments, as well as costs for managing exacerbations and myasthenic crises. The assumptions were validated by an expert panel. RESULTS: Horizon Scanning identified 14 new drugs for gMG in clinical development, with 7 of these expected to enter the Italian market between 2025 and 2029. The expenditure forecast analysis results indicate a slight increase in gMG treatment costs, stable administration costs and a slight reduction in costs for managing exacerbations and myasthenic crises. Total expenditure is projected to rise by 10.3%, from approximately €126 million in 2025 to €139 million in 2029. CONCLUSIONS: Through the Horizon Scanning and expenditure forecasting, it was estimated that, with the arrival of new therapeutic alternatives for gMG on the market, despite the high initial cost of these new treatments, their greater effectiveness could reduce the costs for managing exacerbations and myasthenic crises. This analysis provides essential information to improve the clinical management of the disease, optimize the allocation of healthcare resources and ultimately enhance the quality of life of patients with gMG.
Therapeutic Journey and Economic Burden of Patients with Myasthenia Gravis in Italy: Results of a Real-World Analysis
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.
Copyright (c) 2025 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
Cost-Effectiveness of Dimethyl Fumarate Compared to Teriflunomide for Relapsing Remitting Multiple Sclerosis Patients in Italy: Results of an Updated Analysis
Copyright (c) 2024 Lorenzo Mantovani, Paolo Angelo Cortesi, Michele Molina, Laura Santoni, Luca Prosperini
Decatecholaminization of Septic Shock Patients in Intensive Care Unit: an Economic Assessment in the Italian Setting
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.
Copyright (c) 2024 Alfredo Del Gaudio, Assunta Caiazza, Luana Bortone, Sonia Tomasso, Ilaria Bozzari, Laura Vincenzi, Gianluca Furneri, Luigi Tritapepe
[Enhanced Influenza Vaccines Under Review by the ECDC and the Latest Scientific Literature]
[Implantation of Reveal LINQ in Different Italian Scenarios: How to Further Improve?]
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.
Copyright (c) 2024 Gaetano Senatore, Emilio Di Lorenzo, Orietta zaniolo, Giovanni Liuzzi
Optimizing Treatment of Schizophrenia: Clinical and Economical Potential for Patient Switching to Long‑Acting Injectables
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.
Copyright (c) 2024 Orietta Zaniolo, Silvia Maffezzoli, Massimiliano Povero, Lorenzo Pradelli
[Cost per Responder Analysis of Filgotinib in Patients Suffering from Ulcerative Colitis]
OBJECTIVE: To evaluate the costs and benefits associated with the use of biologics and Janus kinase inhibitors (JAKi) for the treatment of bio-experienced patients suffering from ulcerative colitis in Italy. METHODS: This pharmacoeconomic analysis compared JAKi (filgotinib, tofacitinib and upadacitinib) and biologics (vedolizumab and ustekinumab). A decision tree model with a 1-year time horizon was built in MS Excel in order to estimate treatment cost and clinical efficacy expressed in terms of clinical response (CR), clinical remission (CRe) and mucosal healing (MH). Efficacy of each treatment was estimated using relative risks elaborated from a recent network meta-analysis of 17 RCTs. The investigated population concerned patients previously treated with biologic drugs (bio-experienced). Both induction and maintenance phases were considered and dose escalation (when allowed) was planned for patients not reaching an appropriate response after induction. Outcomes included total treatment cost (acquisition and administration), overall response rate, and cost per responder at one year. RESULTS: According to the model results, the treatment associated with the best clinical response at 1 year was vedolizumab (69.6%) followed by filgotinib (60.9%) and upadacitinib (60.1%). Despite differences in efficacy, filgotinib resulted in the lowest cost per responder, in every outcome, compared with all the other alternatives. The same trend applies when observing results at the end of the induction phase only. Results were robust to probabilistic sensitivity analyses, filgotinib has a posterior probability to be better than the other alternatives higher than 96% according to all the three response definitions (CR, CRe, and MH). CONCLUSIONS: Generally, JAKis and vedolizumab were associated with higher treatment response. Among the alternatives included in the analysis, filgotinib has the lowest cost per responder for all the outcomes included. These findings could help deliver more effective and efficient healthcare in the NHS.
Copyright (c) 2024 Massimiliano Povero, Luca Castello, Lorenzo Pradelli, Elena Visentin
[Cost-Effectiveness Analysis of Maribavir in Patients with Post-Transplant Cytomegalovirus Infection or Disease that are Refractory or Resistant to Conventional Therapy]
OBJECTIVES: Cytomegalovirus (CMV) infection represents a serious post-transplantation complication, particularly among transplant recipients with resistant or refractory (R/R) infection. Despite the improvement made in CMV prevention and treatment, remains a need for new therapies that are better tolerated and safer for patients, especially for refractory or resistant (R/R) infections. This analysis investigates the cost-effectiveness of maribavir versus investigator-assigned therapy (IAT) for treatment of post-transplant R/R CMV in the Italian context. METHODS: The analysis was conducted from the National Health Service (NHS) perspective using a Markov model previously developed in Excel® to evaluate the cost-effectiveness of maribavir versus IAT for the treatment of post-transplant R/R CMV. Health event rates and transition probabilities were informed by data from observational studies and from SOLSTICE clinical trial, which compared maribavir to conventional antiviral treatments for transplant patients with R/R CMV infections. Utilities were from SOLSTICE and a utility vignette study. Costs inputs were derived from published literature and Italian national tariffs for outpatient and hospital services. Costs and outcomes were discounted by 3%. RESULTS: Maribavir compared to the IAT used for the treatment of post-transplant R/R CMV achieved an incremental cost of € 11,455 for an incremental quality adjusted life year (QALY) gain of 0.313. This resulted in an incremental cost-effectiveness ratio of € 36,626/QALY, for maribavir compared to IAT, which is below the generally accepted, national willingness-to-pay threshold for orphan drugs (€ 37,000/QALY). CONCLUSIONS: This analysis showed that maribavir, an orphan drug treatment, is cost-effective compared to the current antiviral treatments used for treatment of post-transplant R/R CMV.
The Importance of the Pharmacoeconomic Analyses in Drug Negotiation. A Farewell Editorial
[Burden of Short Bowel Syndrome in Italy: Direct and Indirect Costs and Quality of Life]
OBJECTIVE: The Short Bowel Syndrome (SBS) is the most common cause of chronic intestinal failure (CIF) due to benign disease. The prevalence in Europe was estimated about 1.4 cases per million and few information are available for Italy. Home parenteral nutrition (HPN) is the primary and lifesaving treatment for patients with CIF. The parenteral nutrition (PN) has a great impact on the quality of life (QoL) of patients and the role of the caregiver is crucial for the disease management. The aim of this study was to evaluate the economic burden of SBS in Italy and to assess the impact of the disease and the parenteral nutrition on the quality of life of patients. METHODS: The total burden of SBS in Italy was assessed both in terms of costs and QoL using different tools. A prevalence-based cost of illness (COI) model was developed to estimate direct costs (PN cost, central venous catheter insertion cost, monitoring cost, hospitalization cost) and indirect costs (absenteeism, presenteeism, unemployment, abandonment of work due to the disease and economic benefits granted by the National Social Security Institute) associated with patient with SBS in Italy. The total annual costs were calculated considering a micro-costing approach, thus associating the average costs per patient with the prevalence of the disease. A systematic literature review (SLR) was conducted to collect epidemiological and direct cost data related to the patients with SBS. Hospital costs were estimated using the national Hospital Information System. Indirect costs were estimated using a human capital approach; therefore, the productivity loss was estimated both for patients and caregivers. A survey was conducted to obtain data about productivity loss and quality of life of patients and caregivers. The questionnaires were completed by clinicians, who were asked to indirectly report the experience of patients with SBS in parenteral nutrition currently being treated at their referral center. In a subsequent phase, a focus group was conducted to collect further information on QoL for patients and caregivers based on the experience of the KOLs involved. The QoL was evaluated considering a Likert scale. RESULTS: The prevalence of patients with gastrointestinal disease in HPN was estimated equal to 9.4 and 2.3 patients per million inhabitants for adults (age >18) and pediatric (age 0-18 years) patients, respectively. Knowing that SBS is the main cause of CIF due to benign disease, constituting 75% among adults and 56% among children, the number of adults with SBS in HPN present in Italy were 420, while the number of children with SBS in HPN were 77. Regarding direct costs, the mean total annual cost associated with adult and pediatric patient with SBS in Italy was estimated equal to € 36,434 and € 46,682, respectively. Parenteral nutrition accounted for 91% of the mean total cost estimated for the adult and for 87% of the mean total cost estimated for pediatric patient. Concerning indirect costs, the mean total annual cost per adult patient was estimated equal to € 51,093 (81% related to the productivity loss because of the abandonment of work due to the disease), while the mean total cost per pediatric patient was estimated equal to € 3,201 (60% related to caregiver’s presenteeism and 40% attributable to caregiver’s absenteeism). Finally, the average total annual cost per adult SBS patient in Italy was estimated at € 87,527 (42% of direct costs and 58% of indirect costs), whereas for pediatric patients it was estimated at € 49,882 (94% of direct costs and 6% of indirect costs). Overall, the weighted average cost for an SBS patient in Italy was estimated at € 81,712 (47% of direct costs and 53% of indirect costs). The analysis conducted on the QoL of patients with SBS in PN has shown that QoL was perceived as low (mean value equal to 5). The greatest impact on QoL was due to the disease (mean value equal to 9), while PN appeared to have less impact (mean value equal to 6). CONCLUSIONS: The analysis provides an estimate of the total burden associated with patients with SBS in Italy both in terms of cost and QoL. The cost associated with parenteral nutrition and indirect costs represent the main drivers of the total cost estimated for a patient with SBS in Italy. Based on the experience of the KOL involved in this study it was also found that the disease has a great impact on the QoL of these patients.
Copyright (c) 2023 Francesco Saverio Mennini, Chiara Bini, Martina Paoletti, Andrea Marcellusi, Paolo Sciattella, Raffaella Viti, Lucrezia Amoroso, Laura Fioravanti, Gennaro Di Martino, Angela Ragonese, Riccardo Caccialanza, Lorenzo Norsa, Antonella Diamanti, Loris Pironi
HTA dei dispositivi di controllo urinario artificiale per il trattamento dell’incontinenza urinaria maschile grave post-prostatectomia
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.
[Measurement-Based Procurement Approach for Biosimilars in Italy: A Position Paper]
The full potential of biosimilars has not been achieved yet, and this document has been written with the aim of methodizing an effective and measurable procurement system that ensures wider, simplified, and economically sustainable access of biosimilars to patients. The economic and financial sustainability generated by the increased use of biosimilars for the benefit of patients and society has been estimated and confirmed in several observational studies and probabilistic projections conducted in Europe. Despite differences in procurement policies, significant reductions in spending for some biosimilars have been recorded in several European countries between 2015 and 2018 compared with the period preceding the patent’s expiry. An accurate evaluation of the economic outcomes resulting from an extended use of biosimilars has also been conducted in Italy, with the settled purpose of calculating the combined effects that various biosimilars with patent expired have generated and will generate in terms of overall spending reduction. Even with the limitations typically associated with probabilistic models, it is also evident that an expanded use of biosimilars is an effective tool to govern the expenditure and channel resources to support the intrinsic balance of the health system (i.e., sustainability) or to ensure the introduction of innovative drugs or diagnostic procedures (i.e., accessibility). The economic sustainability of the health system must also be achieved through the maintenance of a competitive procurement system. This requires a serious reconsideration of the purchasing criteria. The principle of competitiveness can contribute to the governance, containment, reduction or streamlining of public expenditure on biosimilars, but it is appropriate to establish which factors can feed and support a lawful competition, actually. Competition merely based on price reduction between biosimilar and biosimilar, excluding, undervaluing, or taking for granted that between originator and biosimilar, leads to a price erosion and this condition could have unpredictable and undesirable consequences. The market must have its own balance to be of interest to manufacturing companies and ensure the competitiveness. In the field of biosimilars, as in other areas, the need to move from purchasing criteria based on cost exclusively to those based on overall economic benefit by combining price with quality is generally shared. This approach allows for a balanced competition on multiple parameters and above all enables the best service to be provided to patients and the National Health Service. Hence, what are the quality criteria to be considered in relation to price? The areas relevant to the procurement code regulations, the determination of needs, the technical-market knowledge, the encoding of quality criteria, and their specific parameterization to the contingent dynamics of the market must certainly be identified and examined: this is what the present document aims to do. The intention is to provide a guide that can facilitate the implementation of tenders awardable by evaluating the economically most advantageous offer and that will enable us to measure the effect of a “new” procurement system, comparing the differences between what is recorded now and what is detected in the phase following the introduction of quality-price ratio-based tenders for biosimilars in Italy. In conclusion, through the joint work of specialists and experts in the sector, this Position Paper identifies the quality criteria for evaluating biosimilars and proposes a technical specification based on identified criteria, so as to provide central purchasing bodies and institutional payers with a strategy for the actual implementation both at regional and national level of a procurement method focusing on the quality-price ratio. The full potential of biosimilars has not been achieved yet, and this document has been written with the aim of methodizing an effective and measurable procurement system that ensures wider, simplified, and economically sustainable access of biosimilars to patients. The economic and financial sustainability generated by the increased use of biosimilars for the benefit of patients and society has been estimated and confirmed in several observational studies and probabilistic projections conducted in Europe. Despite differences in procurement policies, significant reductions in spending for some biosimilars have been recorded in several European countries between 2015 and 2018 compared with the period preceding the patent’s expiry. An accurate evaluation of the economic outcomes resulting from an extended use of biosimilars has also been conducted in Italy, with the settled purpose of calculating the combined effects that various biosimilars with patent expired have generated and will generate in terms of overall spending reduction. Even with the limitations typically associated with probabilistic models, it is also evident that an expanded use of biosimilars is an effective tool to govern the expenditure and channel resources to support the intrinsic balance of the health system (i.e., sustainability) or to ensure the introduction of innovative drugs or diagnostic procedures (i.e., accessibility). The economic sustainability of the health system must also be achieved through the maintenance of a competitive procurement system. This requires a serious reconsideration of the purchasing criteria. The principle of competitiveness can contribute to the governance, containment, reduction or streamlining of public expenditure on biosimilars, but it is appropriate to establish which factors can feed and support a lawful competition, actually. Competition merely based on price reduction between biosimilar and biosimilar, excluding, undervaluing, or taking for granted that between originator and biosimilar, leads to a price erosion and this condition could have unpredictable and undesirable consequences. The market must have its own balance to be of interest to manufacturing companies and ensure the competitiveness. In the field of biosimilars, as in other areas, the need to move from purchasing criteria based on cost exclusively to those based on overall economic benefit by combining price with quality is generally shared. This approach allows for a balanced competition on multiple parameters and above all enables the best service to be provided to patients and the National Health Service. Hence, what are the quality criteria to be considered in relation to price? The areas relevant to the procurement code regulations, the determination of needs, the technical-market knowledge, the encoding of quality criteria, and their specific parameterization to the contingent dynamics of the market must certainly be identified and examined: this is what the present document aims to do. The intention is to provide a guide that can facilitate the implementation of tenders awardable by evaluating the economically most advantageous offer and that will enable us to measure the effect of a “new” procurement system, comparing the differences between what is recorded now and what is detected in the phase following the introduction of quality-price ratio-based tenders for biosimilars in Italy. In conclusion, through the joint work of specialists and experts in the sector, this Position Paper identifies the quality criteria for evaluating biosimilars and proposes a technical specification based on identified criteria, so as to provide central purchasing bodies and institutional payers with a strategy for the actual implementation both at regional and national level of a procurement method focusing on the quality-price ratio.
Copyright (c) 2023 Arturo Cavaliere, Francesco Saverio Mennini, Marcello Pani, Paolo Gennaro Torrico, Salvatore Torrisi
[Budget Impact Analysis of the Adjuvanted Quadrivalent Influenza Vaccine in the Elderly in Italy]
BACKGROUND: Vaccination is the most effective way to prevent influenza and its complications. The MF59-adjuvanted quadrivalent (aQIV) and the high-dose quadrivalent (QIV-HD) influenza vaccines have been specifically developed to protect subjects aged ≥65 years. The aim of this study was to evaluate the economic consequences associated with the use of aQIV in the elderly population in Italy. METHODS: An Excel-based budget impact model was developed to estimate the costs of aQIV for the prevention of influenza and its complications in elderly subjects, from the perspective of the Italian National Health Service. In the base-case analysis a current scenario (with aQIV) was compared with a past scenario (without aQIV), in which only the standard quadrivalent influenza vaccine (QIV-STD) was available. In the scenario analysis, a current scenario was compared with a future (hypothetical) scenario, in which the market share of aQIV grows. Efficacy (or effectiveness) data of vaccines, in terms of reduction of influenza-related deaths and hospitalizations for influenza/pneumonia, respiratory, and cardiac complications, were obtained from the scientific literature. Relative effectiveness of aQIV and QIV-HD vs. QIV-STD in preventing laboratory-confirmed influenza cases came from two meta-analysis of real-world studies. Epidemiological data and unit costs are collected from Italian published sources. RESULTS: In the base-case analysis the introduction of aQIV e QIV-STD was associated with a reduction of influenza and influenza-like illness – ILI (-93,171) cases, hospitalization for influenza, respiratory and cardiovascular complications (-6,823), and deaths (-1,841) and a consequent saving of € 1.9 million and € 24 million related to events and hospital admissions avoided, respectively. In the scenario analysis, the growth of the market shares of aQIV was associated with 13,602 fewer cases of influenza and ILI, 996 fewer hospitalizations, and an overall saving equal to € 3.8 million. CONCLUSIONS: aQIV reduces the number of cases of influenza and ILI, hospitalizations for influenza and respiratory or cardiac complications, and deaths in the Italian population aged ≥65 years.
Adjuvanted quadrivalent influenza vaccine
Copyright (c) 2022 Vincenzo Baldo, Marco Bellone, Ombretta Bandi
Cost-effectiveness of Empagliflozin, in Addition to Metformin, in Patients with Type 2 Diabetes in Italy
BACKGROUND: Cardiovascular diseases represent the main cause of mortality and morbidity in type 2 diabetes mellitus (T2DM) patients. Empagliflozin is used as a treatment for T2DM because of its association with reduced risk of hospitalization for heart failure (hHF). Recently oral semaglutide, in association with metformin, has shown better results. This study analyzes the cost-effectiveness of empagliflozin versus oral semaglutide, in addition to metformin, in patients with T2DM who are inadequately controlled on metformin alone in Italy. METHODS: This analysis was conducted from the Italian National Health Service (SSN) perspective using the IQVIA Core Diabetes Model. For the base case analysis, a 50-year time horizon was chosen to capture the complications, their associated costs, and the final impact on life-years (LYs) and quality-adjusted life-years (QALYs) gained. Cohort baseline characteristics and efficacy data, were mainly sourced from the PIONEER 2 study. Health-state utilities and event disutilities were based on published sources. Drug acquisition and administration costs and patient management inputs were sourced from Italian-specific data. A sensitivity analysis and a range of scenario analyses were carried out. RESULTS: In the base case analysis treatment cost of empagliflozin plus metformin were significantly lower compared to oral semaglutide plus metformin both including and excluding the effect of empagliflozin on hHF (€-13.371/€-13.580; LYs -0.004/0.109 and QALYs -0.037/0.038). The sensitivity analysis confirmed the robustness of the model with empagliflozin plus metformin that was dominant in 63% and in 42% of simulations considering and non-considering the treatment effect on hHF, respectively. CONCLUSIONS: Empagliflozin 25 mg plus metformin is a cost-effective option versus oral semaglutide 14 mg plus metformin for patients with T2DM uncontrolled on metformin alone in Italy
Copyright (c) 2022 Edoardo Mannucci, Gianni Ghetti
The Importance of Real-World Evidence in Understanding Influenza Vaccine Effectiveness
Copyright (c) 2022 Pietro Ferrara, Lorenzo Giovanni Mantovani
[Cost-Utility Analysis of Dupilumab for the Treatment of Severe Atopic Dermatitis in Children and Adolescents in Italy]
BACKGROUND AND OBJECTIVE: Atopic dermatitis (AD) is a chronic, multifactorial, inflammatory condition characterized by a significant impact on patients’ quality of life. Dupilumab is reimbursed by the Italian Medicines Agency (AIFA) for the treatment of adolescent and adult patients with severe AD (according to AIFA registry criteria). Recently, dupilumab has been reimbursed in the treatment of children with severe AD. The objective of this analysis was to estimate the incremental cost-utility ratio (ICUR) of dupilumab compared to current supportive care (SC), for the treatment of severe AD in children (6-11 years) and adolescents (12-17 years) in Italy. MATERIALS AND METHODS: Cost-effectiveness analysis was conducted using a 1-year decision tree followed by a Markov model over a lifetime period. The base case analysis was performed on the overall population of the LIBERTY AD ADOL (NCT03054428) and LIBERTY AD PEDS (NCT03345914) studies, adopting the National Health Service (NHS) perspective. The following costs were considered: acquisition of treatment, management of disease, adverse events and complications. The robustness of the model was tested through sensitivity analysis. In addition, a scenario analysis adopting the social perspective was performed. RESULTS: In the base case, over a lifetime, dupilumab was more effective than SC in both children and adolescents (+2.44 and +1.62 quality-adjusted life years—QALYs, respectively). The introduction of dupilumab generated an increase in treatment costs (+€ 64,800 and +€ 52,853 € for children and adolescents, respectively), partially offset by a decrease in the costs of disease management and complications. Incremental cost-utility ratios (ICURs) were € 21,189 per QALY gained, for children, and € 26,569 per QALY gained, for adolescents. In both cases, the ICUR was lower than the willingness to pay threshold considered in Italy (€ 50,000 per QALY gained). Both the deterministic and probabilistic sensitivity analysis confirmed the robustness of the base case results. Finally, the scenario analysis, adopting the social perspective, showed coherent results compared to the base case. DISCUSSION: Dupilumab is a cost-effective option for the treatment of children and adolescents with severe AD eligible for systemic treatment in Italy compared to SC, from both the NHS and social perspective, confirming the results obtained in the adult population.
Copyright (c) 2022 Elena Galli, Maria Paola Pedone, Miryana Dobreva, Rossella Bitonti, Roberta Di Turi
Economic Consequences of Administering Obinutuzumab as a Short Duration Infusion in Italian Patients with Advanced Follicular Lymphoma: A Cost Analysis
OBJECTIVE: To assess time- and cost-savings in relation to active time of health care professional (HCP) and resource consumption of administering obinutuzumab as a short duration infusion (SDI) in patients in treatment for Follicular Lymphoma (FL). METHODS: A cost-minimization model was developed to compare resource consumption and cost of the obinutuzumab SDI relative to obinutuzumab regular infusion rate (RIR) for the previously untreated and rituximab-refractory FL. Monetary valuation of resource and time allocated to treatment as a whole was carried out from the Italian Hospital and the societal perspective. Direct costs included HCP costs for drug preparation and administration activities, non-drug consumable costs, drug acquisition costs, and formal care costs. Indirect costs included the lost productivity of patients and informal caregivers. All costs (updated to 2021-value) were estimated by multiplying resource use by the unit cost of each resource. Evidence on resource use and unit costs were retrieved from scientific literature and standard Italian tariffs. A deterministic sensitivity analysis was used to test the results. RESULTS: The administration time of obinutuzumab SDI is shorter than with obinutuzumab RIR, with a difference of 102 minutes per patient and for every cycle of administration beyond the first one. On average, the cost of HCP time invested in the preparation and administration of obinutuzumab RIR is € 92 during cycle 2 and from cycle 2 onwards, compared to € 54 per cycle of obinutuzumab SDI. Overall, the cost from the societal perspective is estimated to be € 38,698 for obinutuzumab RIR and € 37,692 for obinutuzumab SDI, resulting in a cost-saving per patient of € 1,007 (2.6%). CONCLUSIONS: The application of obinutuzumab SDI schedule allows substantial reduction of hospital stay, improving quality of life of patient and caregiver and reducing costs and health care system burden. The time-savings with obinutuzumab SDI may improve clinical unit capacity by optimizing chair utilization and/or allowing rearrangements of the nurse residual time into valuable supplementary activities, spanning from more patient-centered clinical support to research and learning activity
Copyright (c) 2022 Marco Bellone, Lorenzo Pradelli, Rita Tavarozzi, Marco Ladetto, Giuliana Nepoti, Emanuele Guardalben, Daniela Ghislieri
Artificial Urinary Sphincters as a Treatment for Post-Prostatectomy Severe Urinary Incontinence in Italy: A Cost-Utility Analysis
Objective: This study aimed at evaluating the cost-utility of artificial urinary sphincter (AUS) in men affected by postprostatectomy severe urinary incontinence and identifying the most cost-effective alternative among the various devices analyzed in Italy. Methods: A 5-year cycles Markov model was developed to simulate the disease evolution. The analysis compared conservative therapy, ZSI 375®, single-cuff (SC) AMS 800TM, and double-cuff (DC) AMS 800TM. A Probabilistic Sensitivity Analysis (PSA) was performed. One thousand Monte Carlo simulations were conducted to generate the Cost-Effectiveness Acceptability Curve for each intervention strategy. A sensitivity analysis on the price of the device was conducted. Results: From the Italian National Health Service perspective, DC AMS 800TM was the most cost-effective alternative in comparison with conservative therapy, with an Incremental Cost-Effectiveness Ratio (ICER) value equal to € 12,893. From the NHS + patient perspective, both the AMS 800TM devices (SC and DC) were dominant in comparison with conservative therapy. From the societal perspective, ICER was dominant for all the alternatives considered in terms of cost-effectiveness. The PSA showed that DC AMS 800TM had a greater probability to be cost-effective with respect to the other strategies considered in the analysis. The sensitivity analysis on the price of the device showed that in all the cases analyzed the incremental cost per QALY gained would be below € 25,000. Conclusions: This cost-utility analysis confirms that AUSs are cost-effective options in the Italian context with respect to conservative therapy. Among AUSs, DC AMS 800TM has the greatest probability to be cost-effective.
Copyright (c) 2022 Francesco Mennini, Daniele Rossi, Andrea Marcellusi
Cost-Effectiveness of Dimethyl Fumarate Compared to Teriflunomide for Relapsing Remitting Multiple Sclerosis Patients in Italy
BACKGROUND: The objective of this economic analysis was to compare the cost-effectiveness of dimethyl fumarate vs teriflunomide for the treatment of adult patients with relapsing-remitting multiple sclerosis (RRMS) in the Italian setting. Additionally, the cost-effectiveness analysis was used to predict some patient-relevant outcomes such as burden of relapses and survival with disability over time. METHODS: A Markov model was used to conduct the cost-effectiveness analysis. The model measured health outcomes and costs of RRMS patients treated with either dimethyl fumarate or teriflunomide. Data from a published mixed treatment comparison were used for efficacy and safety input. Local economic data were used to calculate costs. A supplementary analysis was carried out to assess ICER variability over time from the Italian National Healthcare Service (NHS) and societal perspectives. Further analyses were conducted to compare clinical effectiveness of the alternatives over time, in terms of incidence of relapses, proportion of patients with EDSS (Expanded Disability Status Scale) score ≤3 and EDSS score ≥6. RESULTS: In the base-case analysis (lifetime horizon; societal perspective) dimethyl fumarate was dominant over teriflunomide (6.526 vs 5.953 QALYs – quality-adjusted life-years; € 1.01 M vs € 1.03 M). The most relevant cost savings (per-patient) with dimethyl fumarate were related to relapses (-€ 5,096), inpatient care (-€ 5,767), informal care (-€ 9,603), long-term absence/early retirement (-€ 14,187). The additional analysis of ICER by time horizon shows that dimethyl fumarate is cost-effective vs teriflunomide (i.e., ICER <€ 50,000 per QALY gained) at already 6 years and at 15 years in societal or NHS perspectives, respectively. Results favoured dimethyl fumarate vs teriflunomide also for: cumulative burden of relapses (-0.23 and -1.37 relapses saved per patient already at 1 year and 10 years, respectively), proportion of patients with mild disability (+4.0% at 10 years), proportion of patients with severe disability (-4.0% at 10 years). CONCLUSIONS: Dimethyl fumarate is dominant (societal perspective), or cost-effective (NHS perspective), referring to a threshold of € 50,000 per QALY gained, vs teriflunomide for the first-line treatment of RRMS, in the Italian setting.
Copyright (c) 2022 Lorenzo Giovanni Mantovani, Paolo Angelo Cortesi, Alessandra Cardillo, Laura Santoni, Luca Prosperini