Farmeconomia. Health economics and therapeutic pathways
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Retifanlimab Vs Avelumab in Patients with Metastatic Merkel Cell Carcinoma: A Cost Utility Analysis in Italy
Full text linkOBJECTIVE: 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
[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]
No full textBACKGROUND: 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 sequencin
[Complementary Strategy of RSVpreF Vaccination/Nirsevimab versus Nirsevimab Alone for the Prevention of RSV Infection in Infants in Italy: A Cost-Minimization Analysis]
No full textOBJECTIVE: 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
[Pharmacoeconomic Comparison Between Dupilumab and Tralokinumab for the Treatment of Atopic Dermatitis in Italy: A Cost-per-Responder Analysis]
No full textOBJECTIVE: 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
[Cost-Effectiveness Analysis of TTFields with TMZ Versus TMZ in Monotherapy in Patients with Grade 4 Glioma in Italy]
No full textINTRODUCTION: 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
A Real-World Analysis of Patients with Triple Class Exposed Multiple Myeloma in Italy: Epidemiology Estimates, Treatment Pattern and Economic Burden
No full textOBJECTIVES: 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
Tinzaparin for the Management of Cancer Associated Thrombosis in Patients with Cancer: a Budget Impact Analysis from the Italian Healthcare System Perspective
Full text linkBACKGROUND: 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
Designing a Model for the Management of Modern Health Services of Middle-aged Adults in the Iranian Health System
No full textBACKGROUND: 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
Real-World Outcomes of Single-Inhaler Triple Therapy Prescribed by General Practitioners in Managing Chronic Obstructive Pulmonary Disease: A Systematic Literature Review
Full text linkBACKGROUND: 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 andMeta-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
Cost Evaluation and Economic Sustainability of Enzyme Replacement Therapies for Fabry Disease in Italy from the Hospital Perspective
No full textBACKGROUND 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