Clinical and Economic Impact of Long-Term Inhaled Corticosteroid Withdrawal in Patients with Chronic Obstructive Pulmonary Disease Treated with Triple Therapy in Spain.

Purpose: To determine the clinical and economic impact of inhaled corticosteroid (ICS) withdrawal in Spanish patients with COPD receiving triple therapy (TT) with ICS, long-acting ß2-agonist (LABA), and long-acting muscarinic antagonist (LAMA). Patients and Methods: This was an observational, retrospective study of BIG-PAC database medical records. Patients aged ≥40 years receiving TT from 2016 to 2018 were followed for 1 year. Two cohorts were identified: patients continuing TT (ICS+LABA+LAMA), and patients receiving TT with ICS withdrawn (LABA+LAMA). Variables included medication, exacerbations (moderate and severe), pneumonia, mortality, health resource use (HRU), and cost per patient/year. Cohorts were compared using propensity score matching (PSM). Multivariate statistical analysis using analysis of covariance and Cox proportional risks was conducted. Results: Of 6541 patients included, 5740 (87.8%) continued TT and 801 (12.2%) had ICS withdrawn. Patients with ICS withdrawal were younger, had lower disease burden, higher ICS doses, and more exacerbations compared with those continuing ICS. PSM matched 795 patients in each cohort. Mean age was 68.5 years (SD: 11.2), 69.9% were male, and mean Charlson index was 2.0. Patients with ICS withdrawal had more total exacerbations in the 12 months following withdrawal compared with patients continuing TT (36.6% vs 31.4%; p=0.030). No significant differences were found for pneumonia (3.3% vs 3.6%; p=0.583) and mortality (9.9% vs 7.5%; p=0.092). Median time to first exacerbation was shorter in patients with ICS withdrawal compared with those continuing ICS (HR: 0.69, 95% CI: 0.57-0.83; p<0.001). Mean health cost per patient/year among patients with ICS withdrawal was higher than those continuing TT (€2993 vs €2130; p<0.001). Conclusion: ICS withdrawal in patients with COPD receiving TT was associated with increased exacerbations, HRU, and costs compared with continuing TT, with health and economic impacts on patients and the Spanish National Healthcare System, respectively. Pneumonia and mortality rates were similar between groups.

Análisis coste-efectividad de Belimumab en pacientes con lupus eritematoso sistémico en España / Cost-effectiveness analysis of Belimumab in patients with systemic lupus erythematosus in Spain

Objetivo: Estimar el coste-efectividad (CE) de belimumab en aquellos pacientes con biomarcadores positivos y enfermedad activa a pesar del tratamiento estándar (TE) desde la perspectiva social española. Métodos: A partir de un modelo de microsimulación, que permite simular la evolución natural de la enfermedad, se estimó el CE de belimumab + TE vs. TE. Se consideró una duración del tratamiento de dos años y un horizonte temporal de toda la vida. La extrapolación de eficacia a largo plazo se basó en los ensayos clínicos de belimumab y en la cohorte de pacientes John Hopkins de Estados Unidos; los datos de utilidades se obtuvieron de la literatura. Se calcularon costes directos e indirectos en base a datos españoles publicados (Euros, 2014), aplicando una tasa de descuento (TD) del 3% tanto a costes como a efectos. Los resultados se expresaron como ratio coste-efectividad incremental (ICER) en términos de años de vida ganados (AVG) y años de vida ajustados por calidad (AVAC). Se realizaron análisis de sensibilidad determinísticos (TD al 0% y 5%, duración de tratamiento 5 años y exclusión de costes indirectos) así como probabilísticos (PSA). Resultados: El ICER de belimumab + TE vs. TE fue de 16.647 Euros/ AVG y 23.158 Euros/AVAC respectivamente. La variación de la TD supuso la mayor variación de los resultados respecto al escenario base. En el 68% de los escenarios simulados en el PSA, belimumab fue una alternativa coste-efectiva considerando como umbral 30.000 Euros/AVAC. Conclusiones: Belimumab puede considerarse una alternativa coste-efectiva desde la perspectiva social española (AU)

Objective: To estimate the cost-effectiveness of belimumab in patients with systemic lupus erythematosus (SLE) presenting positive biomarkers and active disease despite standard treatment (ST), from the Spanish social perspective. Methods: A microsimulation model was used to estimate the cost-effectiveness of belimumab plus ST versus ST alone. A treatment duration of two years with a life-time horizon were considered. Efficacy data were obtained from belimumab clinical trials and the evolution of the disease was simulated from John Hopkins' patient cohort data in the United States. Utility data were obtained from literature review. Direct and indirect costs were calculated based on Spanish published data (Euros, 2014), applying a discount rate (DR) of 3% to both costs and effects. Results were expressed as incremental cost-effectiveness ratio (ICER) in terms of gained life years (LY) and quality of life adjusted life years (QALYs). Probabilistic (PSA) and deterministic sensitivity analyses (DR of 0% and 5%, 5-years treatment duration and excluding indirect costs) were performed to determine the robustness of the model. Results: The incremental cost-effectiveness ratio (ICER) was 16,647 Euros per life year gained, with an incremental cost-utility ratio (ICUR) of 23,158 Euros per additional QALY gained. In 68% of the scenarios simulated in the PSA, belimumab was found to be a cost-effective alternative, considering a threshold of 30,000 Euros/ QALY. Conclusion: Belimumab can be regarded as a cost-effective alternative from the Spanish social perspective (AU)