Real-World Clinical Outcomes and Replacement Factor VIII Consumption in Patients with Haemophilia A in Italy: A Comparison between Prophylaxis Pre and Post Octocog Alfa (BAY 81-8973).

(1) Background: new generations of rFVIII products offered the possibility to improve personalized therapeutic approaches, reducing the number of infusions or increasing the protection against bleeding risk. The aim of this study was to assess the effectiveness of prophylaxis with BAY 81-8973 (octocog alfa, Kovaltry®, Bayer Pharma AG) in the real-world setting and its impact on FVIII consumption compared to previous standard half-life treatments. (2) Methods: a retrospective observational study was conducted in five Italian Haemophilia Centers. Patients with haemophilia A under prophylactic treatment with BAY 81-8973 for at least one year, and previously on prophylaxis with a different product were included in the study. Annual bleeding rate (ABR) and annual FVIII consumption were compared. (3) Results: forty-four patients were included in the study. After switching to BAY 81-8973, ABR was significantly reduced (1.76 vs. 0.23; p = 0.015), the percentage of patients with zero bleeds increased from 54.6% to 84.1% (p = 0.003), and the overall FVIII consumption decreased by 25,542 (-7.2%, p = 0.046) IU per patient-year. Patients treated every 3 days or 2 times per week increased from 0% to 27.3%. (4) Conclusion: our results suggest that prophylaxis with BAY 81-8973 can improve clinical outcomes and reduce FVIII consumption, in the real-world practice, compared with the previous prophylaxis regimen with standard half-life products.

Efficiency measures of emergency departments: an Italian systematic literature review.

Life expectancy globally increased in the last decades: the number of people aged 65 or older is consequently projected to grow, and healthcare demand will increase as well. In the recent years, the number of patients visiting the hospital emergency departments (EDs) rocked in almost all countries of the world. These departments are crucial in all healthcare systems and play a critical role in providing an efficient assistance to all patients. A systematic literature review covering PubMed, Scopus and the Cochrane Library was performed from 2009 to 2019. Of the 718 references found in the literature research, more than 25 studies were included in the current review. Different predictors were associated with the quality of EDs care, which may help to define and implement preventive strategies in the near future. There is no harmonisation in efficiency measurements reflecting the performance in the ED setting. The identification of consistent measures of efficiency is crucial to build an evidence base for future initiatives. The aim of this study is to review the literature on the problems encountered in the efficiency of EDs around the world in order to identify an organisational model or guidelines that can be implemented in EDs to fill inefficiencies and ensure access optimal treatment both in terms of resources and timing. This review will support policy makers to improve the quality of health facilities, and, consequently of the entire healthcare systems.

Sleeve gastrectomy with tailored 360° fundoplication according to Rossetti in patients affected by obesity and gastroesophageal reflux: a prospective observational study.

BACKGROUND: Laparoscopic sleeve gastrectomy (LSG) is the most frequently performed bariatric procedure. Recent studies demonstrated the correlation between LSG and gastroesophageal reflux disease (GERD). OBJECTIVES: To evaluate the effectiveness of LSG + Rossetti antireflux fundoplication in patients affected by morbid obesity and GERD. SETTING: High-volume bariatric center, Italy. METHODS: This is a prospective, observational cohort study that enrolled 58 patients affected by obesity and GERD who underwent surgery. All the patients had a 12-month follow-up. Gastroscopies were performed preoperatively and at month 12 for 35 patients. RESULTS: At 1 year after surgery, patients had a consistent decrease in body mass index, from 41.9 ± 4.6 kg/m2 to 28.2 ± 3.7 kg/m2. GERD improved in 97.1% of patients. Co-morbidities, such as hypertension, type 2 diabetes, respiratory dysfunction, and arthropathies improved as well. The visual analogue scale score regarding the global state of health increased significantly, from 58.1 ± 17.1% before surgery to 98.8 ± 4.1% at 1 year after surgery. Two patients had a fundoplication perforation and needed reparative surgery (3.5%). One patient had anemia that needed a blood transfusion (1.7%). Complications were reduced with a learning curve. CONCLUSION: LSG + Rossetti fundoplication was shown to be a safe and effective intervention. It could be considered an option in obese patients affected by GERD. A longer follow-up is needed to establish the long-term outcomes.

The socioeconomic burden of patients affected by hemophilia with inhibitors.

Hemophilia is associated with a high financial burden on individuals, healthcare systems, and society. The development of inhibitors significantly increases the socioeconomic burden of the diseases. This study aimed to review and describe the burden of hemophilia with inhibitors, providing a reference scenario to assess the impact of new products in the real word. Two systematic literature reviews were performed to collect data on (i) health economics and (ii) health-related quality of life evidences in hemophilic patients with inhibitors. The costs associated with patients with hemophilia and inhibitors are more than 3 times greater than the costs incurred in those without inhibitors, with an annual cost per patient that can be higher than €1 000 000. The costs of bypassing agents account for the large majority of the total healthcare direct costs for hemophilia treatment. The quality of life is more compromised in patients with hemophilia and inhibitors compared to those without inhibitors, in particular the physical domains, whereas mental domains were comparable to that of the general population. The development of an inhibitor has a high impact on costs and quality of life. New treatments have the potential to change positively the management and socioeconomic burden of hemophilia with inhibitors.

Stroke prevention, 1-year clinical outcomes and healthcare resource utilization in patients with atrial fibrillation in France: Data from the GARFIELD-AF registry.

BACKGROUND: GARFIELD-AF is a non-interventional worldwide study of adults with atrial fibrillation. AIMS: To analyse the characteristics of the 1399 patients recruited in France from August 2010 to July 2015, their 1-year outcomes and healthcare resource utilization. METHOD: Patients aged ≥18 years with newly diagnosed atrial fibrillation (≤6 weeks' duration) and ≥1 stroke risk factor were eligible. Patient demographics, medical history and antithrombotic treatment were recorded at baseline. The incidences of stroke/systemic embolism, major bleeding, all-cause mortality, cardiovascular and non-cardiovascular mortality, new acute coronary syndrome and congestive heart failure were recorded during a 1-year follow-up. RESULTS: The median age was 76.0 years; 44.5% of patients were female. The median CHA2DS2-VASc and HAS-BLED scores were 4.0 and 2.0, respectively. At diagnosis, 78.9% of patients received anticoagulant therapy±antiplatelet therapy; more patients received vitamin K antagonists (VKAs; 46.0%) than direct oral anticoagulants (DOACs; 32.9%). The median proportion of time in the therapeutic range for VKAs was 65.6%. Between 2010 and 2015, anticoagulant prescription increased, driven by the growing use of DOACs±antiplatelet therapy (1.1% to 50.0%), whereas prescription of VKAs±antiplatelet therapy decreased (74.4% to 32.3%). All-cause mortality was the most frequent event (6.75 per 100 person-years). Risk-adjusted event rates for France showed that stroke/systemic embolism and all-cause mortality occurred more frequently than in GARFIELD-AF overall, whereas the rates of major bleeding were similar. In terms of healthcare resource utilization, the highest cost was associated with inpatients. CONCLUSIONS: Patients enrolled in France had higher rates of mortality and stroke/systemic embolism than in GARFIELD-AF overall. Conversely, the risk of major bleeding was not higher.

Costs and effectiveness of influenza vaccination: a systematic review.

BACKGROUND: Seasonal influenza can cause a significant public health burden. Vaccination is proposed as the most effective measure to prevent influenza and related undesired outcomes. OBJECTIVE: To estimate the efficiency of influenza vaccination. METHODS: A literature review of economic evaluations of influenza vaccinations, published over the last 5 years, was performed using MEDLINE (through PubMed), Web of Science and Scopus. RESULTS: 935 papers were identified and 30 were selected, including studies performed in different population subgroups: general population, children, adults, elderly, pregnant women and high risk patients. Twenty-one studies were performed in Europe and in US. The majority of the studies were carried out on elderly patients and children. All except one were cost-effectiveness analyses and reported influenza vaccination as a cost-saving or cost-effective intervention. CONCLUSIONS: Vaccination strategies are economically favourable in a range of countries and sub-groups of patients.

Cost and Cost Effectiveness of Treatments for Psoriatic Arthritis: A Systematic Literature Review.

BACKGROUND: Psoriatic arthritis is a long-term inflammatory arthropathy occurring in a subgroup of patients with psoriasis. In addition to irreversible bone erosions, joint destruction, and skin manifestations, psoriatic arthritis is associated with numerous comorbid conditions. Over the last 5 years, new treatments emerged; the analysis and comparisons of their additional costs and the added benefits have become increasingly important to optimize the limited resources available. METHODS: A systematic literature review covering PubMed, EMBASE, and the Cochrane Library was performed from May 2012 to October 2017 focusing on the most recent evidence of costs, benefits, and burden of psoriatic arthritis and its treatments. All economic evaluations assessing the burden of patients with psoriatic arthritis and written in English were eligible for inclusion. We also performed an assessment of the quality of the studies. RESULTS: Of the 1652 references found in the literature search, nine cost-effectiveness analyses and 12 cost-of-illness studies were included in the current review. Patients with psoriatic arthritis incur substantially higher direct and indirect costs, as compared with patients with psoriasis without arthritis or patients with other inflammatory diseases. The cost of treatment with biologic therapies is the major predictor of the total cost. However, individuals with psoriatic arthritis are also affected by substantial productivity losses and indirect costs. Biologic therapies are generally cost effective vs. conventional therapies (e.g., synthetic drugs) for treating psoriatic arthritis. CONCLUSIONS: Psoriatic arthritis is associated with a significant economic burden and biologic therapies contribute significantly to these costs. Biologic therapies are more effective than disease-modifying anti-rheumatic drugs for the symptoms and signs of psoriatic arthritis and for improving quality of life and inhibiting structural radiological damage. Therefore, biologic therapies are cost effective compared with conventional therapies: the increased direct cost associated with biologic drugs is offset by the significant improvement in the efficacy of treatments and in patient management of psoriatic arthritis.

Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions.

BACKGROUND: Cost is currently one of the most important aspects in haemophilia care. Factor concentrates absorb more than 90% of healthcare direct costs of haemophilia care, and the debate regarding the high cost of haemophilia treatments and their different use across different countries is increasing. OBJECTIVE: The objective of this study was to review cost-effectiveness analyses conducted on treatment options in haemophilia, focusing on their results and their strengths and limitations; to highlight the possible issues associated with economic evaluations of new treatment options. METHODS: Electronic searches in PubMed and EMBASE were performed to retrieve papers published between November 2015 and September 2017 to update the previous review of economic evaluations of haemophilia treatments by Drummond et al. Reference lists of included articles and reviews were examined for relevant studies, which were assessed for their quality and their empirical results. RESULTS: Twenty-six relevant economic analyses were identified; 15 (57.7%) were conducted in patients with haemophilia with inhibitors while 11 (42.3%) involved patients without inhibitors. There were methodological variations among the included studies, and differences in the treatment schemes make a comparative assessment of interventions for patients with haemophilia difficult. Only immune tolerance induction showed consistent results in its cost-saving profile compared with the treatment with bypassing agents. CONCLUSIONS: Economic evaluations of haemophilia treatments are increasing, but the identification of general cost-effectiveness trends is still difficult in these studies. We are now facing a new era in haemophilia management with a soaring need for high-quality economic evaluations, performed through proactive collaboration between clinical experts, budget holders and health economists.

Cost-effectiveness analysis of oral fentanyl formulations for breakthrough cancer pain treatment.

Breakthrough cancer Pain (BTcP) has a high prevalence in cancer population. Patients with BTcP reported relevant health care costs and poor quality of life. The study assessed the cost-effectiveness of the available Oral Fentanyl Formulations (OFFs) for BTcP in Italy. A decision-analytical model was developed to estimate costs and benefits associated with treatments, from the Italian NHS perspective. Expected reductions in pain intensity per BTcP episodes were translated into, percentage of BTcP reduction, resource use and Quality-Adjusted-Life-Years (QALYs). Relative efficacy, resources used and unit costs data were derived from the literature and validated by clinical experts. Probabilistic and deterministic sensitivity analyses were performed. At base-case analysis, Sublingual Fentanyl Citrate (FCSL) compared to other oral formulations reported a lower patient's cost (€1,960.8) and a higher efficacy (18.7% of BTcP avoided and 0.0507 QALYs gained). The sensitivity analyses confirmed the main results in all tested scenarios, with the highest impact reported by BTcP duration and health care resources consumption parameters. Between OFFs, FCSL is the cost-effective option due to faster reduction of pain intensity. However, new research is needed to better understand the economic and epidemiologic impact of BTcP, and to collect more robust data on economic and quality of life impact of the different fentanyl formulations. Different fentanyl formulations are available to manage BTcP in cancer population. The study is the first that assesses the different impact in terms of cost and effectiveness of OFFs, providing new information to better allocate the resources available to treat BTcP and highlighting the need of better data.

Italian population-based values of EQ-5D health states.

OBJECTIVE: To estimate a value set for the calculation of Italian-specific quality-adjusted life years (QALYs), based on preferences elicited on EuroQol five-dimensional (EQ-5D) questionnaire health states using the time trade-off technique. METHODS: The revised standard Measurement and Valuation of Health protocol was followed. Twenty-five health states, divided into three groups and given to 450 subjects, were selected to obtain 300 observations per state. Subjects aged 18 to 75 years were recruited to be representative of the Italian general adult population for age, sex, and geographical distribution. To improve efficiency, face-to-face interviews were conducted by using the Computer Assisted Personal Interviewing approach. Several random effects regression models were tested to predict the full set of EQ-5D questionnaire health states. Model selection was based on logical consistency of the estimates, sign and magnitude of the regression coefficients, goodness of fit, and parsimony. RESULTS: The model that satisfied the criteria of logical consistency and was more efficient includes 10 main effect dummy variables for the EQ-5D questionnaire domain levels and the D1 interaction term, which accounts for the number of dimensions at levels 2 or 3 beyond the first. This model has an R(2) of 0.389 and a mean absolute error of 0.03, which are comparable to or better than those of models used in other countries. The utility estimates after state 11111 range from 0.92 (21111) to -0.38 (33333). Italian utility estimates are higher than those estimated in the United Kingdom and Spain and used so far to assess QALYs and conduct cost-utility evaluations in Italy. CONCLUSIONS: A specific value set is now available to calculate QALYs for the conduction of health economic studies targeted at the Italian health care system.

Systemic vasculitis: how little we know about their societal and economic burden.

This article attempts to perform an evaluation of the state of the art of the economic and societal burden of systemic vasculitis (VAs). Due to the rarity of these diseases and their variable clinical picture, few data are available in the literature on their health economic issues, and only some papers have been published that marginally examine the problem. Since VAs are severe conditions with a high medical and societal impact and determine high healthcare resource consumption, studies able to define societal, quality of life and economic burden of these pathologies are needed. Policy makers, private and public organisations involved in the care of VAs need data to programme future investment or make cost-effectiveness analysis for introducing new drugs or protocols.

Systematic literature review on economic implications and pharmacoeconomic issues of psoriatic arthritis.

The introduction of anti tumour necrosis factors-α (TNF-α) agents has greatly advanced the management of psoriatic arthritis (PsA). Functional disability in patients with PsA may result in significant impairment of Quality of Life (QoL), psychosocial disability and productivity loss. Although many patients respond adequately to methotrexate and other therapies, in patients who have incomplete responses, anti TNF-α agents reduce inflammation and minimise joints damage, increasing functional capacity and QoL, and decreasing the progression rate of structural damage in peripheral joints. Because of the high costs associated to anti TNF-α agents therapy, an increasing number of economic evaluations have been performed over the last few years, and several cost-of-illness and cost-effectiveness studies have been published concerning use of anti TNF-α agents in management of PsA. We performed a systematic literature review to better understand the pharmacoeconomic perspective of PsA. The pharmacoeconomic studies analysed have demonstrated the high socioeconomic burden of PsA and that TNF-α blockers treatment options provide value for money in the musculoskeletal and cutaneous manifestations of psoriatic disease.