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.

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.

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-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.