Emma S. Knowles

Background: The current work was designed to estimate the cost–effectiveness of trifluridine/tipiracil (T/T) versus best supportive care (BSC) for patients with advanced stage or metastatic gastroe...

Introduction: Systemic juvenile idiopathic arthritis (sJIA) is a rare, complex autoinflammatory disease with substantial morbidity, often characterized by fever, rash, and muscle pain, amongst other symptoms. Biologic agents, such as anakinra, have been successfully used to treat patients internationally, but their usage in some regions is limited to patients that have failed to achieve clinically inactive disease with corticosteroids and conventional synthetic disease-modifying anti-rheumatic drugs (csDMARDs). Use of anakinra early in the disease course leads to better clinical outcomes; however, longer-term costs for this treatment strategy have not been established. This study compares the economic implications of first-line versus later-line availability of anakinra for patients with sJIA. Methods: Data for patients treated with first-line anakinra were identified from a single-center, prospective study and compared to a combination of published trial and economic evaluation information to facilitate a comparison to later-line anakinra (ie, following corticosteroids + csDMARDs). Costs were estimated for product acquisition and medical resource utilization (MRU), including planned outpatient visits and unplanned hospital admissions. Total costs over a 5-year horizon were compared. Results: Total 5-year product acquisition cost for the first-line anakinra strategy was € 24,021, and for later-line anakinra was € 20,471. The corresponding MRU costs were € 19,197 (first-line) versus € 25,425 (later-line). Overall 5-year costs (product acquisition and MRU) were lower for the first-line strategy (€ 43,218 versus € 45,896). Conclusion: The use of anakinra for patients with sJIA in the first-line setting is efficacious to induce and sustain inactive disease, and the findings of this study show that this treatment strategy leads to cost savings through reduced medical expenditure.

Background A range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established. Objective This study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors. Methods A published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate^®, Sanofi), (2) EHL PEGylated rFVIII (PEG-rFVIII, Adynovate^®, Takeda), and (3) standard half-life (SHL) rFVIII (antihemophilic factor [recombinant], Advate^®, Takeda), used as a proxy for all SHL rFVIII treatments. Acquisition costs were included based on published dosing and weight data. Benefits were incorporated through published annualized bleeding rates, rates of target joint development/resolution, and improvements in the modified hemophilia joint health score. Results were presented as total, discounted costs, and quality-adjusted life-years (QALYs). Results rFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing. Conclusions rFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study.