[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

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 settin

[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 morecost-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

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

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

[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

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

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