ABSTRACT
BACKGROUND: Based on the results of the phase III randomized 20120215 trial, the European Medicines Agency granted the approval of blinatumomab for the treatment of pediatric patients with high-risk first-relapsed Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL). In France, blinatumomab received reimbursement for this indication in May 2022. This analysis assessed the cost effectiveness of blinatumomab compared with high-risk consolidation chemotherapy (HC3) in this indication from a French healthcare and societal perspective. METHODS: A partitioned survival model with three health states (event-free, post-event and death) was developed to estimate life-years (LYs), quality-adjusted life-years (QALYs) and costs over a lifetime horizon. Patients who were alive after 5 years were considered to be cured. An excess mortality rate was applied to capture the late effects of cancer therapy. Utility values were based on the TOWER trial using French tariffs, and cost input data were identified from French national public health sources. The model was validated by clinical experts. RESULTS: Treatment with blinatumomab over HC3 was estimated to provide gains of 8.39 LYs and 7.16 QALYs. Total healthcare costs for blinatumomab and HC3 were estimated to be 154,326 and 102,028, respectively, resulting in an increment of 52,298. The incremental cost-effectiveness ratio was estimated to be 7308 per QALY gained from a healthcare perspective. Results were robust to sensitivity analyses, including analysis from the societal perspective. CONCLUSIONS: Blinatumomab administered as part of consolidation therapy in pediatric patients with high-risk first-relapsed ALL is cost effective compared with HC3 from the French healthcare and societal perspective.
ABSTRACT
Background: Quantification of COVID-19 burden may be useful to support the future allocation of resources. Objective: To evaluate the public health impact of COVID-19 in French ambulatory patients with at least one risk factor for severe disease. Study design: A Markov model was used to estimate life years, costs, number of hospitalisations, number of deaths and long/prolonged COVID forms over a time horizon of 2 years. The hospitalisation probabilities were derived from an early access cohort, and the hospitalisation stay characteristics were derived from the French national hospital discharge database. Several scenario analyses were conducted. Results: The number of hospitalisations reached 256 per 1,000 patients over the acute phase (first month of simulation), and 382 per 1,000 patients over 2 years. The number of deaths was 37 per 1,000 patients, and the number of long/prolonged COVID forms reached 407 per 1,000 patients. These translated into a reduction of 0.7 days of life per patient in the first month, with an associated cost of 1,578, and a reduction of 27 days of life over the time horizon, with an associated cost of 4,280. The highest burden was observed for patients over 80 years old, and those not vaccinated. The scenarios with a less severe situation or new treatments available showed a non-negligible burden reduction. Conclusion: This study allowed us to quantify the considerable burden related to COVID-19 in infected patients, with at least one risk factor for severe form. Strategies with the ability to substantially reduce this burden in France are urgently required.
ABSTRACT
BACKGROUND: Newer direct-acting antiviral therapies are increasingly becoming the therapy of choice in patients with hepatitis C virus (HCV) infection. Here, we report the safety and effectiveness of sofosbuvir/velpatasvir (SOF/VEL) and ledipasvir/sofosbuvir (LDV/SOF) in real-world cohorts in Germany. METHODS: Patients initiated on SOF/VEL 12 weeks or LDV/SOF 8, 12 or 24 weeks regimens in a single German centre were included in this study. Data on treatment outcomes and adverse events (AE) were analysed in patients with available sustained virologic response 12 weeks after cessation of treatment (SVR12) information overall and by subgroups. RESULTS: This study included 115 patients who received SOF/VEL from July-2016 to July-2017, and 249 patients who received LDV/SOF from November-2014 to September-2015. Overall, SVR12 was achieved in 99% of patients on SOF/VEL ± ribavirin 12 weeks independent of HCV genotype, treatment history, or cirrhosis status, and in 96% of patients treated with LDV/SOF 8 weeks or LDV/SOF ± ribavirin 12 or 24 weeks. In genotype 1 treatment-naïve, non-cirrhotic patients, ≥99% achieved SVR12 across SOF/VEL and LDV/SOF regimens. Likewise, 100% of genotype 3-cirrhotic patients on SOF/VEL ± ribavirin regimens achieved SVR12. Grade 3/4 AE were reported in 13 (5.2%) patients on LDV/SOF and in 1 (<1%) patient on SOF/VEL. CONCLUSION: Overall, SOF/VEL and LDV/SOF achieved high SVR rates in a broad patient population. We showed the effectiveness of SOF/VEL as a pan-genotypic regimen, and regardless of treatment history or cirrhosis status. Use of such therapies improves outcomes and contributes towards the global efforts to eradicate HCV.
