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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.
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BACKGROUND: Gefitinib, a tyrosine kinase inhibitor, is an effective treatment in advanced non-small cell lung cancer (NSCLC) patients with an activating mutation in the epidermal growth factor receptor (EGFR). Randomised clinical trials showed a benefit in progression free survival for gefitinib versus doublet chemotherapy regimens in patients with an activated EGFR mutation (EGFR M+). From a patient perspective, progression free survival is important, but so is health-related quality of life. Therefore, this analysis evaluates the Quality Adjusted progression free survival of gefitinib versus three relevant doublet chemotherapies (gemcitabine/cisplatin (Gem/Cis); pemetrexed/cisplatin (Pem/Cis); paclitaxel/carboplatin (Pac/Carb)) in a Dutch health care setting in patients with EGFR M+ stage IIIB/IV NSCLC. This study uses progression free survival rather than overall survival for its time frame in order to better compare the treatments and to account for the influence that subsequent treatment lines would have on overall survival analysis. METHODS: Mean progression free survival for Pac/Carb was obtained by extrapolating the median progression free survival as reported in the Iressa-Pan-Asia Study (IPASS). Data from a network meta-analysis was used to estimate the mean progression free survival for therapies of interest relative to Pac/Carb. Adjustment for health-related quality of life was done by incorporating utilities for the Dutch population, obtained by converting FACT-L data (from IPASS) to utility values and multiplying these with the mean progression free survival for each treatment arm to determine the Quality Adjusted progression free survival. Probabilistic sensitivity analysis was carried out to determine 95% credibility intervals. RESULTS: The Quality Adjusted progression free survival (PFS) (mean, (95% credibility interval)) was 5.2 months (4.5; 5.8) for Gem/Cis, 5.3 months (4.6; 6.1) for Pem/Cis; 4.9 months (4.4; 5.5) for Pac/Carb and 8.3 (7.0; 9.9) for gefitinib. CONCLUSIONS: In the Dutch health care setting, the previously established progression free survival benefit of first-line gefitinib in advanced NSCLC EGFR M+ patients in comparison to standard doublet chemotherapy is further supported by the Quality Adjusted PFS, which takes into account the additional health-related quality of life benefits of gefitinib over doublet chemotherapy.