Cost-Utility Analysis Comparing Pegcetacoplan to Anti-C5 Monoclonal Antibodies in the Treatment of Paroxysmal Nocturnal Hemoglobinuria.

Background: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major breakthrough in PNH treatment, effectively reducing intravascular hemolysis (IVH) but showing limited impact on extravascular hemolysis (EVH). In 2021, the C3 inhibitor pegcetacoplan was approved by EMA and recently reimbursed in Italy, which also has the advantages in the reduction of both IVH and EVH, increasing hemoglobin values and simultaneously improving the quality of life and fatigue of patients. A cost-utility analysis was developed to compare pegcetacoplan to C5i (eculizumab and ravulizumab) in the PNH population who remain anemic after treatment with C5i for at least 3 months. Materials and Methods: The analysis employed a Markov model with a 5-year time horizon whereby patients can transition among 3 PNH health states, adopting the perspective of the Italian NHS. Efficacy data were sourced from the PEGASUS study, with drug prices reflecting ex-factory costs. Additionally, costs associated with resource utilization, adverse events, and complications were estimated based on outpatient and hospital care rates, excluding indirect expenses. Utility and disutility values related to transfusions were also considered, with pegcetacoplan allowing for dose escalation. Results: The cumulative cost of treatment per individual patient at 5 years was estimated to be €1,483,454 for pegcetacoplan, €1,585,763 for eculizumab, and €1,574,826 for ravulizumab. Pegcetacoplan demonstrated a superior increase in quality-adjusted life years (QALYs) compared to both eculizumab (0.51 increase) and ravulizumab (0.27 increase). Furthermore, pegcetacoplan showed a reduction in complication management costs (€22,891 less compared to eculizumab and €22,611 less compared to ravulizumab) and lower transfusion-related expenses (€14,147 less than both C5i treatments). Conclusion: Pegcetacoplan emerged as the dominant strategy in this analysis, being more effective, less expensive and improves quality of life in the analyzed population affected by PNH.

U-CHANGE Project: a multidimensional consensus on how clinicians, patients and caregivers may approach together the new urothelial cancer scenario.

Introduction: Advanced urothelial carcinoma remains aggressive and very hard to cure, while new treatments will pose a challenge for clinicians and healthcare funding policymakers alike. The U-CHANGE Project aimed to redesign the current model of care for advanced urothelial carcinoma patients to identify limitations ("as is" scenario) and recommend future actions ("to be" scenario). Methods: Twenty-three subject-matter experts, divided into three groups, analyzed the two scenarios as part of a multidimensional consensus process, developing statements for specific domains of the disease, and a simplified Delphi methodology was used to establish consensus among the experts. Results: Recommended actions included increasing awareness of the disease, increased training of healthcare professionals, improvement of screening strategies and care pathways, increased support for patients and caregivers and relevant recommendations from molecular tumor boards when comprehensive genomic profiling has to be provided for appropriate patient selection to ad hoc targeted therapies. Discussion: While the innovative new targeted agents have the potential to significantly alter the clinical approach to this highly aggressive disease, the U-CHANGE Project experience shows that the use of these new agents will require a radical shift in the entire model of care, implementing sustainable changes which anticipate the benefits of future treatments, capable of targeting the right patient with the right agent at different stages of the disease.

The Cost-Effectiveness of Anti-IL17 Biologic Therapies for Moderate-to-Severe Plaque Psoriasis Treatment in Italy and Germany: A Sequential Treatment Analysis.

Objective: The objective of this study was to optimise the cost-effectiveness of different anti-IL17 treatment sequences used in the treatment of moderate-to-severe plaque psoriasis in Italy and Germany over a five-year time horizon. Methods: We adjusted a previously published treatment sequence model for biologic drugs used in psoriasis treatment to an Italian and German setting, respectively. The model included all anti-IL17 biologics currently available in the treatment of moderate-to-severe plaque psoriasis in the markets of scope (secukinumab, ixekizumab, brodalumab and bimekizumab). Real-world discontinuation rates were used to model switches between the four anti-IL17 biologics included in the study. The treatment costs were based on label dosing recommendations for each drug, including induction and maintenance therapy, and the manufacturer prices of each drug in Italy and Germany, respectively. We used long-term Psoriasis Area and Severity Index 100 (PASI100) measures to inform the model on the efficacy for each treatment. The cost-effectiveness in the analysis was evaluated based on the cost per PASI100-responder. Results: We found that the most cost-effective treatment sequence was achieved by using brodalumab as first-line treatment, bimekizumab as second-line treatment, ixekizumab as third-line treatment and secukinumab as fourth-line treatment in both Italy and Germany, which resulted in a total cost per responder of €128,200 and €138,212, respectively, over a five-year period. Several scenario analyses were also conducted and ensured that the results were robust to changes in key input parameters. Conclusion: Our study showed that using brodalumab as a first-line therapy to treat moderate-to-severe psoriasis in both Italy and Germany leads to the most cost-effective treatment sequence, when compared to all possible combinations of anti-IL17s over a five-year time horizon. In addition, we found that treatment discontinuation and switching are important factors when assessing the cost-effectiveness of biologic therapies.

Economic Evaluation for Palbociclib Plus Fulvestrant vs Ribociclib Plus Fulvestrant and Abemaciclib Plus Fulvestrant in Endocrine-Resistant Advanced or Metastatic Breast Cancer in Italy.

Background: To date, no study evaluated the cost-effectiveness of palbociclib (PAL) plus fulvestrant (FUL) vs ribociclib (RIB) plus FUL and abemaciclib (ABM) plus FUL in Italy. Cost-effectiveness analysis comparing the three cyclin-dependent 4/6 kinase inhibitors in combination with endocrine therapies for the management of postmenopausal women with HR+, HER2- advanced or metastatic breast cancer in Italy was developed. Material and Methods: To assess the cost-effectiveness of PAL plus FUL vs RIB plus FUL and ABM plus FUL, a cost-minimization has been carried out with a conservative scenario considering three CDK4/6 inhibitors with equal effectiveness in terms of overall survival (OS) (MAIC, Rugo et al 2021). Adverse events (AEs) associated with all therapies were obtained from clinical trials. Ad-hoc analysis was performed to estimate the cost-effectiveness considering the quality-of-life (QoL) data (Lloyd et al 2006). Results: Cost-minimization inputs were drugs, visits and exams, AE monitoring and best supportive care (BSC) before the progression state, active and BSC in the progression and terminal phase of the last two weeks of life. Given the comparability of PAL, RIB and ABM in terms of efficacy, this analysis demonstrated slight economic savings over a lifetime for PAL. Results showed saving per patient of €305 (lifetime) when PAL is compared with RIB; for PAL vs ABM a saving of €243 (lifetime) in a conservative scenario. Results of a budget impact analysis showed a potential savings of €319,563 for PAL vs RIB and €297,544 for PAL vs ABM. When QoL data were considered, results may favor PAL due to the lower impact of AE with savings and improvement in the QoL related to fewer AE. Conclusion: From the Italian perspective, a cost-saving profile associated with the use of PAL+FUL for the management of advanced/metastatic HR+/HER2- breast cancer compared to RIB+FUL and ABM+FUL emerged.

Economic Burden of Non-Alcoholic Steatohepatitis (NASH) Among Diabetic Population in Italy: Analysis and Perspectives.

Background: Aim of our study is to evaluate the economic impact of NASH among diabetic population in Italy and potential benefits of treatments that can slow the disease progression. Methods: A Markov model was conducted from the Italian National Healthcare System perspective reporting results at 3, 5, 10 and 15 years. The model included NASH and T2DM patients with all stages of fibrosis (F0-F3), compensated cirrhosis (CC), decompensated cirrhosis (DCC), hepatocellular carcinoma (HCC), liver transplant (LT), post-LT and death. A 1-year model cycle length was considered, with each patient passing through the stages and exiting the model when reached one of mortality states. Transition probabilities and annual cost related to health states were derived from published literature. Moreover, the model made it possible to develop a scenario analysis to simulate the impact of treatments capable of slowing the disease progression in phases F0-F4 (CC). Results: The results highlighted an economic burden of NASH in T2DM patients of approximately € 1.4 billion, € 3.1 billion, and € 9.4 billion, respectively, after 3, 5 and 10 years, reaching about € 17.3 billion after 15 years. The slowing down of the progression in the early stages of the disease (fibrosis F0-CC) has led to significant savings corresponding to € 2.3 billion at 15 years. These savings were generated by the reduction of the patients in the advanced stages of the disease, which is linked to a reduction in deaths, equal to 92,208 deaths avoided over a 15-year time horizon. Conclusion: Patients with NASH and T2DM reported an important burden in Italy. It is important to investigate the potential clinical and economic benefits of antidiabetic drugs that have been shown to be effective in preventing the transition to advanced disease, simultaneously acting on the therapeutic goals of diabetic disease.

Effect of Reflectance Confocal Microscopy for Suspect Lesions on Diagnostic Accuracy in Melanoma: A Randomized Clinical Trial.

Importance: Previous systematic reviews and meta-analyses have concluded that given data paucity, a comparison of reflectance confocal microscopy (RCM) with dermoscopy is complex. They recommend comparative prospective studies in a real-world setting of suspect lesions. Objective: To test the hypothesis that RCM reduces unnecessary lesion excision by more than 30% and identifies all melanoma lesions thicker than 0.5 mm at baseline. Design, Setting, and Participants: This randomized clinical trial included 3165 patients enrolled from 3 dermatology referral centers in Italy between January 2017 and December 2019, with a mean (SD) follow-up of 9.6 (6.9) months (range, 1.9-37.0 months). The consecutive sample of 3165 suspect lesions determined through dermoscopy were eligible for inclusion (10 patients refused). Diagnostic analysis included 3078 patients (48 lost, 39 refused excision). Data were analyzed between April and September 2021. Interventions: Patients were randomly assigned 1:1 to standard therapeutic care (clinical and dermoscopy evaluation) with or without adjunctive RCM. Information available guided prospective clinical decision-making (excision or follow-up). Main Outcomes and Measures: Hypotheses were defined prior to study initiation. All lesions excised (baseline and follow-up) were registered, including histopathological diagnoses/no change at dermoscopy follow-up (with or without adjunctive RCM). Number needed to excise (total number of excised lesions/number of melanomas) and Breslow thickness of delayed diagnosed melanomas were calculated based on real-life, prospective, clinical decision-making. Results: Among the 3165 participants, 1608 (50.8%) were male, and mean (SD) age was 49.3 (14.9) years. When compared with standard therapeutic care only, adjunctive RCM was associated with a higher positive predictive value (18.9 vs 33.3), lower benign to malignant ratio (3.7:1.0 vs 1.8:1.0), and a number needed to excise reduction of 43.4% (5.3 vs 3.0). All lesions (n = 15) with delayed melanoma diagnoses were thinner than 0.5 mm. Conclusions and Relevance: This randomized clinical trial shows that adjunctive use of RCM for suspect lesions reduces unnecessary excisions and assures the removal of aggressive melanomas at baseline in a real-life, clinical decision-making application for referral centers with RCM. Trial Registration: ClinicalTrials.gov Identifier: NCT04789421.

Economic Impact of COVID-19 Lockdown on Italian NHS: Focus on Diabetes Mellitus.

BACKGROUND: In Italy, the adoption of a total lockdown has generated almost total suspension of outpatient visits except for emergencies. Even after lockdown, the pandemic fear created additional barriers to access the health services. The aim of our study is to evaluate the economic impact of the lockdown for COVID-19 on public health in Italy, focusing on its effects on diabetic population. MATERIALS AND METHODS: We analyzed the impact of the lockdown on excess mortality and morbidity in the Italian diabetic population during 2020. The analysis was divided into several steps: a quantification of specialist visit reduction, the calculation of excess mortality in the diabetic population, the economic evaluation of the slowdown in the use of innovative diabetic therapies. Furthermore, the impact of the lockdown on the reduction of procedures and follow-up visits in diabetic population was evaluated. The overall impact of the pandemic and lockdown effects on costs and quality of life was then calculated. RESULTS: During 2020, a drop of 28% in patient access has been observed. Diabetic patients recorded a twice higher mortality value compared to general population (20.4% vs 10.2%). The analysis of market data revealed a slowdown in consumption of new antidiabetic therapies (-14%, 27% vs 41%). We estimated an expense of €26.6 million for NHS and a loss of 257 utilities in diabetic population due to the missed benefits related to slowdown in innovative antidiabetic drugs use and non-optimal follow-up and control of diabetes complications. In simulation scenarios, we also estimated an overall expenditure ranging from €38.7 to 94.0 million and a loss of 294-836 utilities. CONCLUSION: Diabetic population paid a high tribute to pandemic and lockdown, both in terms of number of deaths and burden of diabetic complications, together with an overall deterioration of quality of life.

Utilization of Healthcare Resources in Osteoarthritis: A Cost of Illness Analysis Based on Real-World Data in Italy.

PURPOSE: Understand the demographics and clinical features of patients with osteoarthritis (OA), quantify healthcare resource utilization by OA patients, and estimate the annual direct medical costs per OA patient from a National Health Service (NHS) perspective in Italy. PATIENTS AND METHODS: Retrospective observational cohort analysis using data from electronic medical records captured by the Italian IQVIA Longitudinal Patient Database (LPD). Only direct medical costs reimbursed by the NHS were considered. Patients were included if they received at least one diagnosis of OA during the period from January 1 to December 31, 2018. Each patient was observed for 3 years: a 24-month baseline period preceding the index date, and a 12-month follow-up period starting at the index date. RESULTS: A total of 71,467 patients met inclusion criteria: 43.98% had not been prescribed NSAIDs/opioids, 40.76% had been prescribed NSAIDs, and 15.26% an opioid. Mean age was 71.36 years, and 68.2% of the patients were women. At least one comorbidity was present in 91.34% of the patients; 38.05% were newly diagnosed with OA. During 1-year of follow-up, 173,884 prescriptions with an associated diagnosis of OA were found: 47.36% had been prescribed an NSAID, 9.11% diclofenac, 8.30% codeine+paracetamol, and 7.32% ketoprofen. Nearly 15% of the patients had at least 1 request for a specialist visit and 23.82% had at least 1 request for exams. Orthopedic visits accounted for 60% of all specialist visits. Yearly mean costs per patient were €622, for approximately €2.5 billion per year in direct costs, considering 3.9 million patients with OA in Italy. Protheses were a major driver in annual costs: €143.45 in patients without a prosthesis and €10,090.91 in those with a joint prosthesis. CONCLUSION: This real-world analysis of direct costs of care of patients with OA in Italy confirms the substantial economic burden. Direct costs dramatically increased when joint replacement was needed.

Cost-Utility Analysis of Saxagliptin/Dapagliflozin Versus Gliclazide and Insulin Glargine: Economic Implications of the Outcomes of the CVD-Real Studies I and II.

Diabetes treatment cost represents an ever-growing problem. The adoption of new drugs in therapy, although they can guarantee an improvement in patient's quality of life, can meet obstacles when it involves an increase in costs. We decided to compare the costs and benefits of the new saxagliptin and dapagliflozin combination versus traditional therapies. Bodyweight loss and the sharp reduction in hypoglycemic episodes were the 2 main clinical outcomes that emerged from registered studies of saxagliptin and dapagliflozin compared with the sulfonylureas. These results, combined with the good cardiovascular risk profile, led to develop a cost-utility analysis. We aimed to show the economic value of this new association therapy. We carried out a cost-utility analysis from the Italian National Healthcare System (NHS) perspective, focused on direct costs related to the treatment and management of main diabetes complications. Utility scores adopted have been measured based on the patient's perception of weight changes. In light of the better durability profile of saxagliptin/dapagliflozin compared with gliclazide, we also considered a simulation scenario to assess the impact on costs of switching to basal insulin, starting from gliclazide and the fixed combination, respectively, and based on the related probabilities to switch. To assess the robustness of the results, a 1-way sensitivity analysis was performed by changing the main parameters by ±20%. Furthermore, the sensitivity of the results was tested considering the addition of a percent discount, because the purchase costs of drugs are usually subject to hidden discounts. We calculated the total direct annual cost per patient of saxagliptin/dapagliflozin versus gliclazide and insulin glargine for patients with type 2 diabetes mellitus not achieving glycemic control on metformin plus saxagliptin alone, dapagliflozin alone, or gliclazide at a lower dosage. Total treatment costs have been obtained adding the direct cost of the drug, needles, glycemic self-monitoring, hypoglycemic events, cardiovascular complications, and effect on consumption of other drugs. The total direct cost of saxagliptin/dapagliflozin fixed dose combination was €414.62 higher than gliclazide (€1.067.72 vs €653.10), and greater than basal insulin, with a difference of €166.99 (€1067.72 vs €900.72). Despite the higher annual direct total cost, the additional cost per quality-adjusted life year (QALY) gained, compared with gliclazide, has been €11 517, and €4639, when compared with insulin glargine in the base-case scenario, and the robustness of the results has been shown in the sensitivity analysis. The results of our cost-utility analysis, expressed as incremental cost-effectiveness ratios, were fully compliant with the threshold adopted for Italy. Then, saxagliptin/dapagliflozin can be considered a cost-effective oral hypoglycemic agent. The positive effect of this drug on the quality of life, induced by the bodyweight loss, has allowed this outcome, despite the higher annual cost per patient, mainly determined by the drug purchase cost.

Omalizumab for Severe Allergic Asthma Treatment in Italy: A Cost-Effectiveness Analysis from PROXIMA Study.

INTRODUCTION: Inadequately controlled severe asthma patients require additional therapy accounting for significant clinical and economic burden. Our analysis aims to determine the cost-effectiveness of omalizumab in the management of severe allergic asthma in Italy based on observational data from the PROXIMA study. METHODS: Observational data on efficacy, healthcare resource utilization and changes in quality of life at 12 months after the initiation of omalizumab were examined to estimate the cost-effectiveness compared to pre-omalizumab period and results were expressed with Incremental Cost-Effectiveness Ratio (ICER). The cost-utility analysis estimated the cost per quality-adjusted life-year (QALY) gained. Direct health costs were assessed from the perspective of the Italian National Health Service (NHS). RESULTS: Omalizumab reduced the incidence of exacerbations, number of hospitalizations, physician visits, and improved quality of life after 12 months of treatment. Omalizumab had a greater effectiveness than pre-omalizumab treatment involving 0.132 QALYs gained and led to a €3729 per patient reduction in direct healthcare costs, excluding the add-on treatment cost. Nevertheless, the addition of omalizumab cost led to €7478 increase in total direct costs with respect to pre-omalizumab period. Based on difference in total direct cost and difference in QALY between post and pre-omalizumab period, the ICER was €56,847. According to sensitivity analysis, omalizumab provided a cost-effective use of NHS resources, already at 20% discounted price. CONCLUSION: This study offers a real-world evidence of omalizumab effectiveness in Italy. Despite the high acquisition cost of the innovative drug, omalizumab is a sustainable treatment option for patients with uncontrolled severe allergic asthma.

Omalizumab and long-term quality of life outcomes in patients with moderate-to-severe allergic asthma: a systematic review.

BACKGROUND: Asthma is a highly prevalent chronic inflammatory airways disease, with a considerable impact on quality of life (QoL). To express the effects of asthma on patients' subjective experience, patient-reported outcomes (PROs) represent an important instrument. The asthma QoL questionnaire (AQLQ) is one of the main PROs among these. MATERIALS AND METHODS: To identify long-term asthma-related QoL outcomes associated with omalizumab therapy in patients with moderate-to-severe asthma, we developed a systematic review according to the PRISMA guidelines. Published real-world effectiveness studies of adults or adolescents (12 years or older) with moderate-to-severe allergic asthma treated with omalizumab for at least 48 weeks were reviewed. Sources used were Medline ( PubMed), the Cochrane Library and Google Scholar up to February 2018. In addition, a cross-referencing search was conducted to complete the revision. RESULTS: A total of 255 potential papers were identified in the first search through the database. After full-text viewing, eight articles were finally included in the review. We summarized the results according to the study design, patient baseline characteristics and effectiveness outcomes assessed by AQLQ score results: variation from baseline to the end of study. Results confirmed the long-term benefits of omalizumab as an add-on therapy in patients with uncontrolled moderate-to-severe allergic asthma. Since there is a lot of evidence on omalizumab effectiveness, we aimed to focus on how a therapy can change patient's QoL in a long time period. Data showed long-term effects of omalizumab treatment on subjective (PROs) and objective (lung function, corticosteroid use, hospitalizations, asthma exacerbation) effectiveness measures. CONCLUSION: Studies included in our review were observational trials that, due to their design, present a potential risk of selection bias in the patients included. Beyond this limit, the evaluation of QoL using the AQLQ showed a clear increase over time, following both 48 weeks and 9 years of observation, where QoL improvements still were significant over baseline values.

Shadow cost of oral corticosteroids-related adverse events: A pharmacoeconomic evaluation applied to real-life data from the Severe Asthma Network in Italy (SANI) registry.

BACKGROUND: Asthma is one of the most common non-communicable respiratory diseases, affecting about 6% of the general population. Severe asthma, even if afflicts a minority of asthmatics, drives the majority of costs of the disease. The aim of this study is to create a pharmacoeconomic model to predict the costs of corticosteroid-related adverse events in severe asthmatics and applying it to the first published epidemiologic data from the Severe Asthma Network in Italy (SANI) registry. METHODS: The analysis was conducted from the perspective of the Italian National Healthcare System (INHS). Model inputs, derived from literature, included: asthma epidemiology data, frequency of adverse events, percentage of severe asthma treated with OCS and adverse event cost (Diagnosis-Related Group (DRG) national tariffs). We estimated costs per different patient groups: non-asthma controls, mild/moderate and severe asthmatics. Final results report estimated direct cost per patient and total direct cost for overall target population, showing economic impact related to corticosteroid complication. RESULTS: Based on epidemiological data input, in Italy, asthmatic subjects resulted about 3,999,600, of which 199,980 with severe asthma. The number of patients with severe asthma OCS-treated was estimated at 123,988. Compared to the non-asthma control cohort and to that with moderate asthma annual cost per severe asthmatic patient resulted respectively about €892 and €606 higher, showing a corticosteroids shadow cost ranging from 45% to 30%.Applying the cost per patient to the target population identified for Italy, the budget impact model estimated a total annual cost related to OCS-related adverse events of €242.7 million for severe asthmatics. In respect with non-asthmatic and moderate population, an incremental expenditure of about € 110.6 million and €75.2, respectively, were shown. CONCLUSIONS: Our study provides the first estimates of additional healthcare costs related to corticosteroid induced adverse events in severe asthma patient. Budget impact model results highlighted the relevant economic impact of OCS-related adverse events in severe asthma patients. The future extrapolation of additional data from SANI registry will support the development of a model to investigate the role of corticosteroids sparing drugs.

Cost-minimization analysis of degludec/liraglutide versus glargine/aspart: economic implications of the DUAL VII study outcomes.

BACKGROUND: Diabetes represents a relevant public health problem worldwide due to its increasing prevalence and socioeconomic burden. There is no doubt that tight glycemic control reduces the development of diabetic complications such as the long-term costs related to the disease. The aim of our model was to calculate total direct costs associated with the two treatments considered in DUAL VII study, and hence evaluate the potential economic benefits for the National Health System (NHS) deriving from the use of insulin degludec plus liraglutide (IDegLira) in a once-daily fixed combination. MATERIALS AND METHODS: We applied the cost-minimization technique adopting the NHS point of view to the DUAL VII trial outcomes. In the model, developed in Microsoft Excel®, we calculated and compared annual costs per patient of the two therapeutic options for type 2 diabetes (T2D) patients not achieving glycemic control on basal insulin and metformin described in the trial, including costs of therapy management and side effects, both negative and positive. Annual treatment costs were calculated based on IDegLira and basal bolus end-of-trial doses resulting in a 1:2 ratio (40.4 U vs 84.1 U). Therefore, maintaining the IDegLira/basal bolus at 1:2 dose ratio, we calculated the correlation between the dose reduction and costs compared to DUAL VII doses base case scenario. RESULTS: Total treatment costs were obtained by adding annual cost of drug, needles, glycemic self-monitoring, hypoglycemic events, and effect on consumption of other drugs. Total annual costs of IDegLira combination resulted in €434 higher than basal bolus in DUAL VII base case (40.4 U); the two treatments reported equal costs at 34% dose reduction (26.7 U), while below this value IDegLira treatment became less expensive, with about €215 gain at 50% dose reduction (20.2 U). It is also important to notice that above the break-even point, until an IDegLira dose of 30 U, the cost difference is negligible in view of the clinical benefit provided by the fixed combination highlighted in DUAL VII trial. CONCLUSION: Adding the significant clinical findings derived from DUAL VII trial to our economic evaluation, IDegLira seems to offer an important alternative to basal-bolus therapy.

Patterns of treatment and costs of intermediate and advanced hepatocellular carcinoma management in four Italian centers.

BACKGROUND: Hepatocellular carcinoma (HCC) is a severe health condition associated with high hospitalizations and mortality rates, which also imposes a relevant economic burden. PURPOSE: The aim of the present survey is to investigate treatment strategies and related costs for HCC in the intermediate and advanced stages of the disease. PATIENTS AND METHODS: The survey was conducted in four Italian centers through structured interviews with physicians. Information regarding the stage of disease, treatments performed, and related health care resource consumption was included in the questionnaire. Direct health care cost per patient associated with the most relevant treatments such as sorafenib, transarterial chemoembolization (TACE), and transarterial radioembolization (TARE) was evaluated. RESULTS: Between 2013 and 2014, 285 patients with HCC were treated in the four participating centers; of these, 80 were in intermediate stage HCC (Barcelona Clinic Liver Cancer Classification [BCLC] B), and 57 were in the advanced stage of the disease (BCLC C). In intermediate stage HCC, the most frequent first-line treatment was TACE (63%) followed by sorafenib (15%), radiofrequency ablation (14%), and TARE (1.3%). In the advanced stage of HCC, the most frequently used first-line therapy was sorafenib (56%), followed by best supportive care (21%), TACE (18%), and TARE (3.5%). The total costs of treatment per patient amounted to €12,214.54 with sorafenib, €13,418.49 with TACE, and €26,106.08 with TARE. Both in the intermediate and in the advanced stage of the disease, variability in treatment patterns among centers was observed. CONCLUSION: The present analysis raises for the first time the awareness of the overall costs incurred by the Italian National Healthcare System for different treatments used in intermediate and advanced HCC. Further investigations would be important to better understand the effective health care resource usage.

Cost-effectiveness analysis on the use of rFSH + rLH for the treatment of anovulation in hypogonadotropic hypogonadal women.

BACKGROUND: Hypogonadotropic hypogonadal women are characterized by ovarian functionality deficiency, caused by low concentrations of follicle-stimulating hormone (FSH) and luteinizing hormone (LH). To recover reproduction functionality, recommended therapies for ovarian induction involve injections of FSH and LH medications. OBJECTIVE: Since important differences exist between recombinant and urinary gonadotropin therapies in terms of efficacy and cost, the objective of this study was to develop a cost-effectiveness model to compare recombinant FSH (rFSH) + recombinant LH (rLH) and highly purified human menopausal gonadotropin (HP-HMG). METHODS: A Markov model was developed, considering three cycles of therapy; probability of pregnancy and miscarriage were considered, and the efficacy was evaluated in terms of pregnancy occurrence. The perspective of the model was that of the Italian Health Service, so only direct cost (drugs, specialist visits, patient examinations, and hospitalizations) were included. RESULTS: rFSH + rLH is associated with a higher total cost (€3,453.50) and higher efficacy (0.87) compared with HP-HMG (€2,719.70 and 0.50). rFSH + rLH generated an incremental cost effectiveness ratio equal to €2,007.30 compared to HP-HMG; the average cost per pregnancy is estimated to be €3,990.00 for recombinant strategy and €5,439.80 for urinary strategy. Results of probabilistic sensitivity analysis were consistent with the abovementioned findings. CONCLUSION: Despite the higher acquisition cost in comparison to HP-HMG, rFSH + rLH resulted in a higher pregnancy rate, which makes it the recommended choice when considering cost-effectiveness of LH in supporting FSH-induced follicular gonadotropins in hypogonadotropic hypogonadal women.

The burden of endometriosis: costs and quality of life of women with endometriosis and treated in referral centres.

BACKGROUND: This study aimed to calculate costs and health-related quality of life of women with endometriosis-associated symptoms treated in referral centres. METHODS: A prospective, multi-centre, questionnaire-based survey measured costs and quality of life in ambulatory care and in 12 tertiary care centres in 10 countries. The study enrolled women with a diagnosis of endometriosis and with at least one centre-specific contact related to endometriosis-associated symptoms in 2008. The main outcome measures were health care costs, costs of productivity loss, total costs and quality-adjusted life years. Predictors of costs were identified using regression analysis. RESULTS: Data analysis of 909 women demonstrated that the average annual total cost per woman was €9579 (95% confidence interval €8559-€10 599). Costs of productivity loss of €6298 per woman were double the health care costs of €3113 per woman. Health care costs were mainly due to surgery (29%), monitoring tests (19%) and hospitalization (18%) and physician visits (16%). Endometriosis-associated symptoms generated 0.809 quality-adjusted life years per woman. Decreased quality of life was the most important predictor of direct health care and total costs. Costs were greater with increasing severity of endometriosis, presence of pelvic pain, presence of infertility and a higher number of years since diagnosis. CONCLUSIONS: Our study invited women to report resource use based on endometriosis-associated symptoms only, rather than drawing on a control population of women without endometriosis. Our study showed that the economic burden associated with endometriosis treated in referral centres is high and is similar to other chronic diseases (diabetes, Crohn's disease, rheumatoid arthritis). It arises predominantly from productivity loss, and is predicted by decreased quality of life.

[Management of acute pain in Italy. Economic aspects and quality of life]. / La gestione del dolore acuto in Italia: aspetti economici e qualità di vita.

OBJECTIVES: This study aims to measure quality of life and treatment costs of patients with acute pain due to osteoarticular diseases;2 to asses associations between pain, quality of life and treatment costs from NHS' perspective. PATIENTS AND METHODS: Observational, multicentric study; patients of both gender were enrolled sequentially from March to July 2002 and observed for 30 days. Type of pain was assessed by a McGill Pain Questionnaire, while degree of severity was measured by VAS (Visual Analogue Scale) ranging from 0 (no pain) to 10 (very severe pain). Quality of life was assessed by EQ-5D (EuroQol/Quality of Life in Europe-5 Dimensions). RESULTS: 188 patients matched by gender and age in to four group: male < or =65 years (51), female < or =65 years (59), male > 65 years (42), female > 65 years (36). Male's age ranged from 36 to 88 years, while female's ranged from 31 to 87 years. Groups differ for type of pain. Cost after enrollment (48.99 Euro) is due for about 60% to diagnostic examinations, while pharmacological therapies account for only 17.64 Euro. Mean score of EQ-5D shows a low level of utility (0.265) that reflects a rather law quality of life. Patient older than 65 years had lower score than patient of the same gender but younger. Differences are significant in the dimension anxiety/depression that afflicts women and older patients. CONCLUSIONS: This study valued utility of patients with non oncological acute pain in Italy and health cost of diagnostic and therapeutic path faced by patients.

[Quality of life and treatment costs in patients with non-cancer chronic pain]. / Qualità della vita e costi di trattamento in pazienti con dolore cronico benigno.

BACKGROUND: there is still little information about the use of generic questionnaires [e.g. the 36-item Short Form health survey (SF-36)] and utility questionnaires [e.g. the EuroQOL instrument (EQ-5D) and visual analogue scale (VAS)] in patients with non-cancer chronic pain. OBJECTIVES: (i) to measure quality of life and utility in patients with non-cancer chronic pain using the SF-36 and the EuroQOL questionnaires; (ii) to assess direct costs and to evaluate the impact of some clinical variables on diagnostic or treatment costs. PARTICIPANTS: 153 patients from 9 Italian Center for treatment non-cancer chronic pain (54% male, mean age 57 years) completed both quality of life questionnaires and diagnostic and treatment prescription questionnaire. RESULTS: the mean scores for the 8 domains of the SF-36 ranged from 23.7 (physical role) to 53.7 (mental health). The EuroQOL mean score was 0.28 in the self-classifier (EQ-5D) version and 6.4 in the visual analogue scale (VAS) version. The direct costs was assessed in 173 Euro, in two months observation period (the analysis was conducted from the National Health System perspectives); we found some differences between pathologies observed: about 180 Euro for back pain or arthrosis, about 155 Euro for periarthritis of the shoulder. CONCLUSIONS: our study, quantified the quality of life in patients with non-cancer chronic pain, showed that the presence of pain had an important role. The EuroQOL measurements obtained from these patients will aid evaluation of the cost-utility ratio for pain therapies.