Italian National Healthcare Service Research Articles

OPT-In; Optimized Patient Treatment Outcomes in Plaque Psoriasis: A 3-Year State-Transition Treatment-Sequencing Model in the Italian Setting.

There are several treatment options for plaque psoriasis (PsO), but uncertainty remains around the optimal sequencing of treatments. The aims of this study were to investigate how adopting a best-treatment-first treatment sequence impacts patient outcomes and healthcare systems and to quantify the cost of treatment failure to the healthcare system. A 3-year state-transition treatment-sequencing model which identifies all possible treatment sequences in PsO was adapted to the Italian healthcare setting. Treatments considered in the model are those with European Medicines Agency marketing authorization and reimbursement in Italy as of December 2022. Italian market share data (2019-2021) and list prices (2022) informed the current prescribed sequences; these sequences were compared against all possible sequences to determine opportunities for improvement. Both the national perspective in Italy as well as the local perspective from seven regions were considered. The cost of treatment failure was informed through a questionnaire circulated to Italian dermatologists. Overall, 1284 possible treatment sequences are possible when four lines of treatment are considered for patients with moderate-to-severe PsO in Italy. Within the estimated range of treatment failures across those sequences (0.97-2.56 per patient over 3 years), current prescribing behavior from the national perspective suggests patients will face 1.44 failures on average; this highlights the potential for improvement. For every treatment failure, the cost borne by the Italian National Healthcare Service (NHS) is €676.80. Overall, prescribing more optimized treatment sequences results in a 22.95% reduction in failures with a 2.27% increase in costs. The regional analyses found similar trends. Results suggest that selecting the most effective treatment sequences for incident patients provides the greatest opportunity to reduce treatment failures and maximize patient outcomes with a modest impact on costs. While regional variations exist, there is room for improvement across the board, which could translate to more efficient local healthcare systems.

Framework for evaluating the performance of local healthcare: international experiences and prospects for Italy.

The Italian National healthcare service currently lacks a synthetic framework for measuring the primary care legislative reform established by the National recovery and resilience plan and the legislative reform, Decree number 77/2022. This paper explores the existing international and national literature on primary care' monitoring and evaluation systems with the purpose of drawing guidelines to build up a global and systematic framework. The 2022 World health organization framework is the most advanced reference point as a result of more than twenty years of theoretical and field research. Indeed, it can be regarded as the basic model to be adapted to the specificities of the current Italian legislation and organization.

Redesigning the Drugs Distribution Network: The Case of the Italian National Healthcare Service

Drug distribution performed through hospital pharmacies facilitates public expenditure savings but incurs higher social costs for patients and caregivers. The widespread presence of community pharmacies could support patient access while also improving drug distribution. The implementation of prescriptive data analyses as constrained optimization to achieve specific objectives, could be also applied with good results in the healthcare context. Assuming the perspective of the Italian National Healthcare Service, the present study, built upon existing research in this field, proposes a decision support tool that is able to define which self-administered drugs for chronic diseases should be distributed by community pharmacies, answering to critical challenges in the case of future pandemics and healthcare emergencies, while also providing suggestions for the institutional decision-making process. Moreover, the tool aids in determining the optimal setup of the drug distribution network, comparing centralized (hospital pharmacies) and decentralized (community pharmacies) approaches, as well as their economic and social implications.

Access and use of WHO essential medicines in Italy.

Many countries use the WHO Essential Medicines List (EML) as a guide for health policy choices to promote the efficient use of healthcare resources or adopt the concept of essential medicines (EMs) to develop their own national list of essential medicines. The aim of this study is to analyse the availability and use of medicines included in the 22nd WHO EML in Italy. Using the ATC code (5th level), a comparison was made between the medicines included in the WHO EML and those retrieved from the Italian Medicines Agency (AIFA) database. The availability (regulatory and reimbursement status) of EMs, as well as the market share in expenditure (million euros) and consumption [measured in WHO-defined daily doses (DDDs)], compared to all reimbursed medicines in 2021, were analysed. In 2021, approximately 85.2% (n = 414) of medicines included in the WHO EML were commonly marketed in Italy. Of these, 396 EMs were fully reimbursed by the Italian National Healthcare Service (INHS), corresponding to 81.5% (396/486) of the WHO EML, while the remaining 18.5% (90/486) were neither authorised (n = 72) nor reimbursed (n = 18). The study found a low coverage for anti-parasitic, insecticides, and repellent products (ATC P) in addition to medicines for the genitourinary system and sex hormones (ATC G). Even though medicines on the WHO EML, including therapeutic alternatives, accounted for ~48.5% of the expenditure for medicines reimbursed by INHS, the list covered 74% of all national drug consumed. Novel high-cost therapies indicated in high-prevalence diseases and rare conditions, mostly antineoplastic and immune-modulating agents (ATC L) not included in the WHO EML, were also guaranteed. In Italy, high coverage of EMs was found. It was largely reimbursed by the INHS, even when compared to other European countries. Essential medicines represented a high percentage of the overall expenditure and consumption in Italy. The WHO EML could be an important tool to guide the health policy choices of high-income countries, although a more frequent update and easier access to information on rejected medicines are needed.

Budget impact analysis of anakinra in the treatment of familial Mediterranean fever in Italy

ABSTRACT Introduction: Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disease that significantly reduces occupational productivity and quality-of-life in affected patients. Italy has an estimated FMF prevalence of 1 in 60,000 people. While colchicine is the primary treatment for FMF, biologics are administered to intolerant and non-responder patients. Anakinra and canakinumab are the only biologics approved and reimbursed for FMF in Italy. Both medicines have demonstrated efficacy in FMF patients yet differ in treatment costs. This study aimed to perform a budget impact analysis (BIA) following anakinra’s reimbursement for FMF treatment, considering pharmaceutical costs from the Italian National Healthcare Service (NHS) perspective. Methods: A ‘Reference scenario’ (all patients treated with canakinumab) was compared to an ‘Alternative scenario’, with increased anakinra market shares. The target population was estimated based on the Italian population, epidemiological and market research data. Drugs costs were estimated based on Summary of Product Characteristics and net ex-factory prices. Sensitivity analyses were implemented to test results’ robustness. Results:The base case analysis showed an overall cumulative expenditure of €30,586,628 for ‘Reference scenario’ and € 16,465,548 for ‘Alternative scenario’. A cumulative savings of €14,121,080 (46.2%) was calculated over 3 years as a result of the reimbursement and increasing uptake of anakinra. The sensitivity analyses, even considering a discount of 50% for canakinumab, confirmed the base case results. Conclusions: Anakinra’s introduction, in FMF treatment, provides a financially sustainable option for Italian patients, with savings increasing according to greater use of anakinra.

The Value of Hearing Aids for the Italian NHS: A Cost-utility Analysis.

Hearing loss (HL) prevalence in Italy is expected to increase due to population aging. Hearing aids (HAs) are the main tool for HL rehabilitation; however, cost-utility analyses of HAs are limited. Our objective was to estimate the cost-utility of HAs use. Cost-utility analysis. Italian National Healthcare Service, societal perspective. A multistate Markov model was developed to model a cohort of 55-year-old individuals starting from normal hearing and moving across HL states to compare cost-utility and net monetary benefit of HA use accompanied by post-purchase service, HA use alone, and no treatment. Parameters were estimated using secondary data. Incremental cost-utility ratio (ICUR) and incremental net monetary benefit (INMB) were computed against a €16,625/quality-adjusted life year (QALY) willingness-to-pay (WTP) threshold. Deterministic and probabilistic sensitivity analysis (DSA, PSA) was implemented to assess how uncertainty affected results. Scenario analysis was performed on different assumptions on costs, dropout and compliance rates. The model suggests HAs use is a cost-effective strategy compared to no treatment (in the base case: incremental costs €429-€476, incremental QALY gain 0.18 and 0.19, ICUR €2'404/QALY-€2'450/QALY, INMB €2'476-€2'682 for male and female cohort, respectively). By assuming no dropout, INMBs increase up to €10,643-€10,728. DSA highlights that utility weights contribute the most to model uncertainty, PSA shows that the treatment has 97.8%-97.3% probability of being cost-effective at the WTP threshold considered. We proposed an original model to assess the cost-utility of HAs use; the application to the Italian setting suggests the treatment is cost-effective, reinforcing the importance of early uptake.

Savings associated with therapeutic appropriateness for patients with relapsed refractory multiple myeloma and high cytogenetic risk diagnosed by FISH test

Background: Approximately 23% of myeloma patients have cytogenetic abnormalities which confer a significantly poorer prognosis, and therefore they are defined as “high-risk myeloma” (HRC+) patients. Objective: To estimate the economic impact of a large-scale use of the fluorescence in situ hybridization (FISH) test for the identification of HRC+ patients. Methods: The comparison between the total costs of two scenarios, the first one where patients are not tested for cytogenetic abnormalities, the second one where FISH test is used to identify HRC+ patients and to treat them with the most appropriate therapies, was conducted. The Italian National Healthcare Service (NHS) perspective was adopted. The cost of each treatment was estimated by multiplying Italian unit ex-factory prices by median progression-free survival (PFS). For each scenario, the cost per month of PFS gained was calculated. Sensitivity analysis was conducted to evaluate the impact on the results. Results: N = 777 patients are expected to be HRC+ in Italy. If they were all identified through FISH test and treated with the most appropriate therapies, this would result into savings of € 552,000 and a PFS gain of 419 months, compared with a scenario with no identification. The cost per patient per month of PFS gained is lower in the scenario where FISH test was available (€ 9,921 vs € 10,229 in the scenario without testing). Sensitivity analyses confirmed the robustness of the base case results. Conclusions: A larger adoption of FISH test to identify HRC+ patients is expected to improve clinical outcomes in relapsed refractory multiple myeloma.

Cost-Utility Analysis of Dupilumab for the Treatment of Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) in Italy.

The objective of this analysis was to estimate the incremental cost–utility ratio (ICUR) of dupilumab as an add-on treatment to best supportive care (BSC), versus BSC alone, in Italy for severe uncontrolled chronic rhinosinusitis with nasal polyps (CRSwNP). A simulation of outcomes and costs was undertaken using a 1-year decision tree, followed by a lifetime horizon Markov model. Clinical data were derived from a pooled analysis of two studies (SINUS-24 NCT02912468 and SINUS-52 NCT02898454). The Italian National Healthcare Service (NHS) perspective was considered. Model robustness was tested through sensitivity analyses. In the base-case analysis, treatment with dupilumab + BSC resulted in an increase in quality of life-adjusted survival (+1.02 quality-adjusted life years (QALY-gained)), compared to the BSC alone. The resulted ICUR was €21,817 per QALY-gained and it is below the acceptability threshold commonly used in Italy. Both one-way deterministic and probabilistic sensitivity analyses confirmed the robustness of base-case results. The cost–utility analysis showed that dupilumab, as an add-on treatment to BSC, is a cost-effective therapeutic alternative to BSC in the treatment of patients with severe uncontrolled chronic rhinosinusitis with nasal polyps, confirming that it is economically sustainable.

Cost-effectiveness analysis of brolucizumab versus aflibercept for the treatment of neovascular age-related macular degeneration (nAMD) in Italy

BackgroundAge-related macular degeneration (AMD) is a common and chronic eye condition characterized by the presence of progressive degenerative abnormalities in the central retina (macula). Notably, neovascular, or wet, AMD (nAMD) occurs when new, abnormal blood vessels grow under the macula causing scarring of the macula itself and resulting in a loss of central vision, visual distortion, and an impaired capacity of perceiving colour contrast and intensity. Brolucizumab, a new generation anti-vascular endothelial growth factor (anti-VEGF) monoclonal antibody, was approved by the European Medicines Agency for the treatment of nAMD. The aim of this analysis is to evaluate the cost-effectiveness profile of brolucizumab, compared to the main therapeutic alternative available (aflibercept), for the treatment of nAMD.MethodsThe simulation of costs and outcomes was carried out using a Markov model over a time horizon of 15 years. In base-case, treatment effectiveness inputs for brolucizumab and aflibercept were extracted from the HAWK and HARRIER studies and from a network meta-analysis. The Italian National Healthcare Service (NHS) perspective was considered, therefore only healthcare direct costs (treatment acquisition, administration, adverse events, disease monitoring) were analysed. In the alternative scenarios, the societal perspective and a prolonged time horizon were considered. Model robustness was tested through sensitivity analyses.ResultsIn the base-case analysis, brolucizumab was dominant over aflibercept (+ 0.11 years QALY gained and -€15,679 costs). Both one-way deterministic and probabilistic sensitivity analyses confirmed the robustness and reliability of base-case results. The results of the probabilistic sensitivity analysis showed that when the willingness to pay is equal to €50,000 per QALY gained, brolucizumab would be dominant in 84% of simulations and in the remaining simulations brolucizumab would be cost-effective compared to aflibercept. Results of the alternative scenarios and sensitivity analyses confirmed the results of base-case.ConclusionThe cost-utility analysis shows that brolucizumab is dominant over aflibercept. Treatment with brolucizumab reduces the economic impact of nAMD and determined a slight increase of quality-adjusted survival. This analysis gives a high level of confidence that the treatment with brolucizumab would reduce the burden of intravitreal injections, compared to aflibercept, a relevant therapeutic alternative in Italy.

Comparing risk adjustment estimation methods under data availability constraints

The Italian National Healthcare Service relies on per capita allocation for healthcare funds, despite having a highly detailed and wide range of data to potentially build a complex risk‐adjustment formula. However, heterogeneity in data availability limits the development of a national model. This paper implements and ealuates machine learning (ML) and standard risk‐adjustment models on different data scenarios that a Region or Country may face, to optimize information with the most predictive model. We show that ML achieves a small but generally statistically insignificant improvement of adjusted R 2 and mean squared error with fine data granularity compared to linear regression, while in coarse granularity and poor range of variables scenario no differences were observed. The advantage of ML algorithms is greater in the coarse granularity and fair/rich range of variables set and limited with fine granularity scenarios. The inclusion of detailed morbidity‐ and pharmacy‐based adjustors generally increases fit, although the trade‐off of creating adverse economic incentives must be considered.

Innovative and conventional \u201cconservative\u201d technologies for the treatment of uterine fibroids in Italy: a multidimensional assessment

BackgroundTo evaluate the potential benefits of the Magnetic Resonance-guided high intensity Focused Ultrasound (MRgFUS) introduction in the clinical practice, for the treatment of uterine fibroids, in comparison with the standard “conservative” procedures, devoted to women who wish to preserve their uterus or enhance fertility: myomectomy and uterine artery embolization (UAE).MethodsA Health Technology Assessment was conducted, assuming the payer’s perspective (Italian National Healthcare Service). The nine EUnetHTA Core Model dimensions were deeply investigated, by means of i) a literature review; ii) the implementation of health economics tools (useful for uterine fibroids patients’ clinical pathway economic evaluation, and budget impact analysis), to define MRgFUS economic and organizational sustainability, and iii) administration of specific questionnaires filled by uterine fibroids’ experts, to gather their perceptions on the three possible conservative approaches (MRgFUS, UAE and myomectomy).ResultsLiterature revealed that MRgFUS would generate several benefits, from a safety and an efficacy profile, with significant improvement in symptoms relief. Advantages emerged concerning the patients’ perspective, thus leading to a decrease both in the length of hospital stay (p-value< 0.001), and in patients’ productivity loss (p-value = 0.024). From an economic point of view, the Italian NHS would present an economic saving of − 6.42%. A positive organizational and equity impact emerged regarding the capability to treat a larger number of women, thus performing, on average, 131.852 additional DRGs.ConclusionsResults suggest that MRgFUS could be considered an advantageous technological alternative to adopt within the target population affected by uterine fibroids, demonstrating its economic and organisational feasibility and sustainability, with consequent social benefits.

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 &lt;€ 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.

Budget Saving Potential of Pegfilgrastim Biosimilar for the Treatment of Chemotherapy-Induced Febrile Neutropenia, in Italy

INTRODUCTION: Current Italian guidelines recommend prophylaxis with granulocyte colony-stimulating factors (G-CSFs) to reduce the risk of chemotherapy-induced febrile neutropenia (FN). The availability of G-CSF biosimilars represents an opportunity for savings in the Italian National Healthcare Service (NHS) delivery of care.OBJECTIVE: To assess the cost saving potential associated with the introduction of pegfilgrastim biosimilars to local formularies, compared to the current G-CSF standard practice in Italy.METHODS: A budget impact model was developed to compare the current standard practice of long-acting (LA) and short-acting (SA) G-CSFs use, with a future scenario in which the market share of LA G-CSFs grows due to the more advantageous administration schedule and price of pegfilgrastim biosimilar. The analysis included G-CSF treatment schedules, drug acquisition costs and costs of patient management including hospitalization and ambulatory care.RESULTS: The introduction of pegfilgrastim biosimilar resulted in cumulative 3-year cost savings of € 59,650 and € 41,539 for FN prophylaxis in a potential cohort of 1000 patients with solid tumors and lymphomas, respectively.CONCLUSIONS: The results indicate that the introduction of pegfilgrastim biosimilar is potentially associated with substantial cost savings for the Italian healthcare system.

Chronic use of the unfixed combination of ACE-inhibitors and beta-blockers in Italy from 2013 to 2019 through the healthcare administrative data.

The association between an ACE-inhibitor and a beta-blocker is recommended in case of complicated arterial hypertension and heart failure with reduced ejection fraction. This retrospective drug-utilization study has described its chronic use from the Italian National Healthcare Service perspective. From the ReS (Ricerca e Salute) database, collecting Italian healthcare administrative data, patients receiving an ACE-inhibitor or a beta-blocker from 2013 to 2019 were selected. The prevalence of use (patients treated/1000 inhabitants) and of continuous treatment (patients treated for ≥80% period from the supply to the end of the same year - by means of dosage units - per 1000 inhabitants) of each single active substance were assessed. Among patients continuously treated, those supplied with an ACE-inhibitor and a beta-blocker (unfixed and fixed combinations) were analyzed in terms of prevalence of continuous treatment. Subjects treated with the unfixed combination ramipril-bisoprolol in 2019 were characterized by gender and age. The prevalence of ACE-inhibitors' use increased from 49.7/1000 inhab. in 2013 to 50.2 in 2019: ramipril was the most supplied each year (28.9 to 31.6/1000 inhab.). The prevalence of continuous treatment increased from 27.4 to 28.3/1000 inhab.: ramipril the most continuously dispensed (16 to 18/1000 inhab.). The prevalence of beta-blockers' use increased from 61.0 to 90.4/1000 inhab.: bisoprolol the most supplied and with the highest increase (27.1 to 52.2/1000 inhab.). The prevalence of continuous treatment increased from 33.1 to 55.8/1000 inhab.: bisoprolol the most continuously dispensed (18 to 37/1000 inhab.). Among patients with continuous supplies, from 49,843 a 51,496 were treated with associations between an ACE-inhibitor and a beta-blocker on an ongoing basis. Half of them were continuously treated with the unfixed combination ramipril-bisoprolol (4.3 to 4.8/1000 inhab.). In 2019, subjects with continuous supplies of ramipril-bisoprolol were mainly males (63.4%) and elderly (mean age 71±12), and the prevalence of use increased with age. These findings, together with recommendations from the main international guidelines encourage to make available also ramipril and bisoprolol as fixed dose combination, in order to simplify the therapy administration and improve the adherence, especially among elderly and patients with multimorbidity.

Outcome After Acute Ischemic Stroke Treatment During Covid-19 Outbreak in South-East Tuscany.

During Covid-19 pandemic, the Italian National Healthcare Service has faced increasing pressure, especially in Northern Italy. Even in less-affected regions, such as Tuscany, the changes in the healthcare system to prevent Covid-19 spread resulted in difficulty in treating time-dependent disorders like ischemic stroke rapidly. The aim of our study was to assess the outcome after acute ischemic stroke treatments during the Covid-19 spread in comparison with a similar period of the previous year in Siena-Hospital (Hub center in the South-East Tuscany). We enrolled all patients admitted to Siena-Hospital for ischemic stroke and submitted them to acute treatments (intravenous and/or mechanical thrombolysis) between February 21st and May 18th, 2020 (study group, n:38) and compared the results with ischemic strokes acutely treated in a similar period in 2019 (control group, n:39). The modified Rankin scale score was assessed at 90 days to evaluate a 3-month clinical outcome. In the study group, the time from symptoms onset to hospital arrival and the door-to-groin puncture time were significantly more prolonged than in the control group. In moderate-severe strokes, the 3-month mortality was significantly higher in the study group (31% vs. 6%; p=0.01), and the number of patients with poor functional outcomes was significantly higher in the study group (73% vs. 44%; p=0.03). During the lockdown period due to Covid-19 pandemic, patients with acute ischemic stroke had a worse prognosis. These findings suggest the need to improve the health system organization to guarantee an appropriate treatment during the pandemic, including the patients that are not affected by Covid-19.

Cost-utility analysis of trabecular micro-bypass stents (TBS) in patients with mild-to-moderate open-angle Glaucoma in Italy

BackgroundGlaucoma is a disease characterized by progressive damage of the optic nerve. Several therapeutic options are available to lower intraocular pressure (IOP). In primary open-angle glaucoma (POAG) patients with inadequate IOP control (or controlled with multiple medical therapies or for whom medical therapy is contraindicated), the implantation of micro-invasive glaucoma surgery devices (MIGS) and concomitant cataract surgery has proved to be more effective in reducing intraocular pressure (IOP), as compared to cataract surgery alone. The objective of this study was to assess the cost-effectiveness of iStent inject® device with concurrent cataract surgery vs. cataract surgery alone, in patients with mild-to-moderate POAG, adopting the Italian National Health Service (NHS) perspective.MethodsSimulation of outcomes and costs was undertaken using a Markov model with 4 health states and one-month cycles, that is used to simulate the prognosis of these patients. Efficacy data were obtained from the randomized clinical trial (RCT). A lifetime horizon was adopted in the analysis. A discount rate of 3.5% was applied to both costs and effects. The Italian National Healthcare Service (NHS) perspective was considered, therefore only healthcare direct costs (acquisition of main interventions and subsequent procedures; medications; monitoring and follow-up; adverse events). Model robustness was tested through sensitivity analyses.ResultsResults of the base-case analysis showed that the total lifetime costs were higher in the iStent inject® + concurrent cataract surgery, compared with the cataract surgery alone group (€8368.51 vs. €7134.71 respectively). iStent inject® + concurrent cataract surgery was cost-effective vs. cataract surgery alone, with an incremental cost-effectiveness ratio of €13,037.01 per quality-adjusted life year (QALY) gained. Both one-way deterministic and probabilistic sensitivity analyses confirmed robustness of base-case results. The cost-effectiveness accessibility curve (CEAC) showed that iStent inject® + cataract surgery would have a 98% probability of being cost-effective, compared to cataract surgery alone, when the willingness to pay (WTP) is equal to €50,000 per QALY gained.ConclusionsThe results of the cost-utility analysis confirm that iStent inject® + cataract surgery is a cost-effective option for the treatment of patients affected by mild-to-moderate POAG, compared with cataract surgery alone, when evaluated from the Italian NHS perspective.

PBI11 Cost-Effectiveness Analysis of Dupilumab for the Treatment of Atopic Dermatitis in Adolescent Patients in Italy

Atopic dermatitis (AD) is a chronic, multifactorial, inflammatory skin disease. Dupilumab, a fully human monoclonal antibody, has been recently approved for the treatment of adolescents (aged 12-17) with uncontrolled moderate to severe AD. The objective of this analysis was to estimate the incremental cost-effectiveness ratio (ICER) of dupilumab versus current supportive care (SC) used to treat AD in Italy. Simulation of outcomes and costs was undertaken using a 1-year decision tree, followed by a lifetime horizon Markov model. The base-case analysis was conducted on the overall population of the study LIBERTY AD ADOL 1526 (full analysis set). A secondary analysis was conducted on a subgroup of patients treated with prior use of topical corticosteroids or calcineurin inhibitors. The Italian National Healthcare Service (NHS) perspective was considered, therefore only healthcare direct costs (treatment acquisition, disease management, adverse events, management of complication – i.e. flares-) were analysed. Model robustness was tested through sensitivity analyses. In the base-case, dupilumab was more effective than SC (+1.53 quality adjusted life years, QALYs). The introduction of dupilumab led to an increase in treatment costs (+ €61,121.17), which are partially offset by a decrease in the costs of disease management and the management of complications of the disease (respectively - €8,349.80 and - €907.84). The incremental cost-effectiveness ratio (ICER) was €33,918.29 per QALY gained, below the directional willingness-to-pay threshold considered in Italy (€ 50,000 per QALY gained).The results of the alternative analysis confirmed the base-case results with a modest reduction of the ICER vs. base-case (€33,648 vs €33,918, respectively). Both one-way deterministic and probabilistic sensitivity analyses confirmed robustness of base-case results. Dupilumab is a cost-effective option for the treatment of adolescent patients with uncontrolled moderate to severe AD in Italy, compared with SC, from the NHS perspective.

PRS32 Cost-Effectiveness Analysis of Dupilumab for the Treatment of Type 2 Severe Asthma in Italy

A new biological therapy, dupilumab, has been recently approved for the treatment of severe asthma with type 2 inflammation, characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO). The objective of this analysis was to estimate the incremental cost-effectiveness ratio (ICER) of dupilumab, as an add-on treatment to background therapy (BT), versus the main therapeutic alternatives approved in Italy for severe asthma. Simulation of outcomes and costs was conducted using a lifetime horizon Markov model. In the base-case analysis dupilumab + BT, was compared to BT alone for the treatment of type 2 asthma patients, with raised blood eosinophils (EOS- ≥150 cells/μL) and/or raised fractional exhaled nitric oxide (FeNO≥25 ppb) in the previous 12 months, with experiencing at least 2 acute asthma exacerbations despite maximum inhalation therapy (treated with systemic steroid for at least 3 days and/or who have requested access to the emergency room and/or hospitalization and/or intubation. Data source were retrieved from QUEST trial. An alternative analysis was conducted to compare dupilumab + BT to BT in the treatment of severe asthma (any EOS and FeNO levels; all patients treated with oral corticosteroids); data input were obtained from VENTURE trial. The Italian National Healthcare Service (NHS) perspective was adopted. In the base-case analysis, treatment with Dupilumab + BT reduced the burden of severe exacerbations, and prolonged both survival and quality-adjusted survival compared to BT alone. The resulting incremental cost-effectiveness ratio (ICER) was € 36,829 per QALY gained, below the directional willingness-to-pay threshold considered in Italy (€ 50,000 per QALY gained). The results of the alternative analysis confirmed the base case results with an ICER of € 40,047 per QALY gained. Cost-effectiveness analysis shows that dupilumab + background therapy is a cost-effective and economically sustainable treatment option for the Italian NHS.

HTA and HIV: The Case of Dual NRTI Backbones in the Italian Setting.

The aim of this study is to analyze the potential advantages of emtricitabine/tenofovir alafenamide (FTC/TAF) introduction, creating evidence-based information to orient strategies to reduce costs, thus preserving effectiveness and appropriateness. An Health Technology Assessment (HTA) was implemented in the years 2017–2018 comparing the dual backbones available in the Italian market: FTC/TAF, FTC/TDF (tenofovir disoproxil fumarate/emtricitabine) and ABC/3TC (abacavir/lamivudine). From an efficacy point of view, FTC/TAF ensured a higher percentage of virologic control and a better safety impact than FTC/TDF (improving the renal and bone safety profile, as well as the lipid picture). From an economic point of view, the results revealed a 4% cost saving for the Italian National Healthcare Service NHS with FTC/TAF introduction compared with the baseline scenario. Qualitative perceptions’ results showed that FTC/TAF would decrease the burden of adverse events management, increasing the accessibility of patients to healthcare providers (FTC/TAF: 0.95, FTC/TDF: 0.10, ABC/3TC: 0.28; p-value: 0.016) and social costs (FTC/TDF: −0.23, FTC/TAF: 1.04, ABC/3TC: 0.23; p-value < 0.001), improving patient quality of life (FTC/TDF: 0.31, FTC/TAF: 1.85, ABC/3TC: 0.38; p-value < 0.001). Healthcare services may consider the evidence provided by the present study as an opportunity to include HIV patients in a more adequate antiretroviral treatment arm, guaranteeing a personalized clinical pathway, thus becoming more efficient and effective over time.

Appropriate Timing of Gestational Diabetes Mellitus Diagnosis in Medium- and Low-Risk Women: Effectiveness of the Italian NHS Recommendations in Preventing Fetal Macrosomia

Background Screening strategies for gestational diabetes mellitus (GDM) earlier than 24-28 weeks of gestation should be considered to prevent adverse pregnancy outcomes. Nonetheless, there is uncertainty about which women would benefit most from early screening and which screening strategies should be offered to women with GDM. The Italian National Healthcare Service (NHS) recommendations on selective screening for GDM at 16-18 weeks of gestation are effective in preventing fetal macrosomia in high-risk (HR) women, but the appropriateness of timing and effectiveness of these recommendations in medium- (MR) and low-risk (LR) women are still controversial. Patients and Methods. We retrospectively enrolled 769 consecutive singleton pregnant women who underwent both anomaly scan at 19-21 weeks of gestation and screening for GDM at 16-18 and/or 24-28 weeks of gestation, in agreement with the NHS recommendations and risk stratification criteria. Comparison of maternal characteristics, fetal biometric parameters at anomaly scan (head circumference (HC), biparietal diameter (BPD), abdominal circumference (AC), femur length (FL), estimated fetal weight (EFW)), and neonatal birth weight (BW) percentile among risk groups was examined. Results 219 (28.5%) women were diagnosed with GDM, while 550 (71.5%) were normal glucose-tolerant women. Out of 164 HR women, only 62 (37.8%) underwent the recommended early screening for GDM at 16-18 weeks of gestation. AC and EFW percentiles, as well as neonates' BW percentiles, were significantly higher in HR women diagnosed with GDM at 24-28 weeks of gestation with respect to normal glucose-tolerant women, as well as MR and LR women who tested positive for GDM. Comparative analysis between MR and LR women with GDM and women with normal glucose tolerance revealed significant differences in both AC and EFW percentiles (P < 0.05), while there was no significant difference in neonatal BW percentiles. Conclusion In MR and LR women with GDM, a mild acceleration of fetal growth can be detected at the time of anomaly scan. However, in these at-risk categories, the NHS recommendations for screening and treatment of GDM at 24-28 weeks of gestation are still effective in normalizing BW and preventing fetal macrosomia, thus supporting a risk factor-based selective screening program for GDM.