Cabotegravir and Rilpivirine Long-Acting Antiretroviral Therapy Administered Every 2 Months is Cost-Effective for the Treatment of HIV-1 in Spain.

INTRODUCTION: Current antiretroviral therapies (ARTs) have improved outcomes for people living with HIV. However, the requirement to adhere to lifelong daily oral dosing may be challenging for some people living with HIV, leading to suboptimal adherence and therefore reduced treatment effectiveness. Treatment with long-acting (LA) ART may improve adherence and health-related quality of life. The objective of this study was to evaluate the cost-effectiveness of cabotegravir + rilpivirine (CAB+RPV) LA administered every 2 months (Q2M) compared with current ART administered as daily oral single-tablet regimens (STRs) from a Spanish National Healthcare System perspective. METHODS: A hybrid decision-tree and Markov state-transition model was used with pooled data from three phase III/IIIb trials (FLAIR, ATLAS, and ATLAS-2M) over a lifetime horizon, with health states defined by viral load and CD4+ cell count. Direct costs (in €) were taken from Spanish public sources from 2021 and several deterministic and probabilistic analyses were carried out. An annual 3% discount rate was applied to both costs and utilities. RESULTS: Over the lifetime horizon, CAB+RPV LA Q2M was associated with an additional 0.27 quality-adjusted life years (QALYs) and slightly greater lifetime costs (€4003) versus daily oral ART, leading to an incremental cost-effectiveness ratio of €15,003/QALY, below the commonly accepted €30,000/QALY willingness-to-pay threshold in Spain. All scenario analyses showed consistent results, and the probabilistic sensitivity analysis showed cost-effectiveness compared with daily oral STRs in 62.4% of simulations, being dominant in 0.3%. CONCLUSION: From the Spanish National Health System perspective, CAB+RPV LA Q2M is a cost-effective alternative compared with the current options of daily oral STR regimens for HIV treatment. CLINICAL TRIALS REGISTRATION: ATLAS, NCT02951052; ATLAS-2M, NCT03299049; FLAIR, NCT02938520.

Over the past decades, treatments for HIV infection have improved outcomes for people living with HIV. However, most of the treatments available consist of daily oral administration, which may present challenges for some people. These challenges may lead to a less optimal intake of the medicines and, therefore, to a potential reduction of treatment effectiveness. A new long-acting treatment alternative for HIV with two drugs is now available: cabotegravir + rilpivirine long-acting is the first injectable treatment administered in the muscle every 2 months by a healthcare professional. Long-acting injectables may improve treatment administration and health-related quality of life of people living with HIV. This study estimated the cost-effectiveness of cabotegravir + rilpivirine long-acting in Spain compared with daily oral single-tablet treatment for HIV. An economic model using clinical data and Spanish inputs was used to estimate cost-effectiveness and health outcomes over a lifetime. Cabotegravir + rilpivirine long-acting compared with daily oral single-tablet treatment showed an increase in health-related quality of life, leading to a cost-effectiveness ratio of €15,003, below the Spanish willingness-to-pay threshold of €30,000. All different scenarios tested showed consistent results, with cabotegravir + rilpivirine long-acting being cost-effective in 62.4% of the simulations and less costly and more effective in 0.3%. This study demonstrated that, in Spain, cabotegravir + rilpivirine long-acting administered every 2 months is a cost-effective alternative to the current daily oral single-tablet treatment options for HIV.

Cost-Effectiveness of Once-Daily Single-Inhaler COPD Triple Therapy in Spain: IMPACT Trial.

Purpose: Given between-country differences in healthcare systems, treatment costs, and disease management guidelines, country-specific cost-effectiveness analyses are important. This study evaluated the cost-effectiveness of once-daily fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) versus FF/VI and UMEC/VI among patients with symptomatic chronic obstructive pulmonary disease (COPD) at risk of exacerbations from a Spanish healthcare system perspective. Patients and Methods: Baseline data and treatment effects from the IMPACT trial were populated into the validated GALAXY COPD progression model. Utilities were estimated using Spanish observational data. Direct healthcare costs (2019 €) were informed by Spanish public sources. A 3% discount rate for costs and benefits was applied. The time horizon and treatment duration were 3 years (base case). One-way sensitivity, scenario, and probabilistic sensitivity analyses were performed. Results: FF/UMEC/VI treatment resulted in fewer exacerbations over 3 years (4.130 vs 3.648) versus FF/VI, with a mean (95% confidence interval [CI]) incremental cost of €444 (€149, €713) per patient and benefit of 0.064 (0.053, 0.076) quality-adjusted life years (QALYs), resulting in an incremental cost-effectiveness ratio (ICER) of €6887 per QALY gained. FF/UMEC/VI was a dominant treatment strategy versus UMEC/VI, resulting in fewer exacerbations (4.130 vs 3.360), with a mean (95% CI) incremental cost of -€450 (-€844, -€149) and benefit of 0.054 (0.043, 0.064) QALYs. FF/UMEC/VI was cost-effective versus FF/VI and UMEC/VI across all analyses. Conclusion: FF/UMEC/VI was predicted to be a cost-effective treatment option versus FF/VI or UMEC/VI in symptomatic COPD patients at risk of exacerbations in Spain, across all scenarios and sensitivity analyses.

Contribution of cabotegravir + rilpivirine long-acting for the treatment of HIV-1 infection.

OBJECTIVE: To determine the value contribution of cabotegravir + rilpivirine, the first injectable every two months long-acting antiretroviral  regimen, using multi-criteria decision analysis. METHOD: The study was developed in two phases. After a small pilot, a field  ork study with a larger number of multidisciplinary experts was carried out.  Seven single-tablet regimens, currently recommended by the GeSIDA  guidelines, were selected as comparators. EVIDEM methodology was followed,  with a framework composed by 12 quantitative and 5 contextual criteria. Mean  and standard deviations were calculated for quantitative criteria (1 to 5 scale;  comparative criteria -5 to +5), whereas qualitative criteria were analyzed as  percentages of experts that considered a positive, neutral or negative impact  for the National Health System. RESULTS: 35 experts participated in the study. Human immunodeficiency virus- 1 infection was considered severe (mean ± standard deviation: 3.0 ± 1.0),  with moderate size of affected population (2.7 ± 1.2) and unmet needs (2.8 ±  1.0). Minimal differences were found in comparative efficacy/effectiveness (0.1  ± 0.5), safety/tolerability (-0.5 ± 0.7), and cost criteria: cost of the  intervention (0.5 ± 2.0), other medical costs (0.2 ± 1.8) and non- medical/indirect costs (0.5 ± 1.6). Experts perceived an improvement with  cabotegravir + rilpivirine long-acting, compared  to current daily oral single-tablet regimens, in patient-reported outcomes (2.7 ± 1.4). Therapeutic benefit  of the long-acting regimen was considered moderate-to-high (3.5 ± 1.2).  Experts considered the evidence provided by cabotegravir + rilpivirine long- actingrobust (4.3 ± 0.8), with elevated consensus on its future  recommendation in guidelines (3.2 ± 1.0). In contextual criteria, most experts  considered positive the impact on population priorities and access (91%),  common goal and specific interests (63%) and political, historical, and cultural  context criteria (60%). Impact was neutral in system capacity and appropriate  use (40%), and opportunity costs and affordability criteria (51%). Result of the  weighted global value contribution of cabotegravir + rilpivirine long-acting  was 0.34 (-1 to +1 scale), with Patient Reported Outcomes comparative  criterion bringing the highest added value. CONCLUSIONS: Cabotegravir + rilpivirine long-acting provides added value  contribution to human immunodeficiency virus-1 management in Spain  compared to daily oral single-tablet regimens. Patient Reported Outcomes and  therapeutic benefit of cabotegravir + rilpivirine long-acting were highly valued  by experts, as the expected benefit in adherence and stigma-related issues  would improve overall quality of life for people living with human  immunodeficiency virus-1.

OBJETIVO: Determinar la contribución de valor de cabotegravir + rilpivirina, el  primer tratamiento antirretroviral inyectable de acción prolongada, utilizando  metodología de análisis de decisión multicriterio.Método: El estudio se desarrolló en dos fases: una prueba piloto y una fase de  extensión, con un grupo multidisciplinar más grande. Se seleccionaron siete regímenes de comprimido único orales diarios  recomendados en las guías GeSIDA como comparadores. Se utilizó el marco  EVIDEM, compuesto por 12 criterios cuantitativos y 5 contextuales. Los  criterios cuantitativos se analizaron calculando la media y desviación estándar,  y los cualitativos se analizaron mediante el porcentaje de expertos que  consideraron el impacto positivo, neutro o negativo para el Sistema Nacional de Salud. RESULTADOS: Un total de 35 expertos participaron en el estudio. La infección  por virus de la inmunodeficiencia humana 1 se consideró grave (media ±  desviación estándar: 3,0 ± 1,0), con un tamaño de población afectada (2,7 ±  1,2) y unas necesidades no cubiertas (2,8 ± 1,0) moderadas. Las diferencias  fueron mínimas en los criterios comparativos de eficacia/efectividad (0,1 ±  0,5), seguridad/tolerabilidad (­0,5 ± 0,7) y coste: coste del tratamiento (0,5 ±  2,0), otros costes médicos (0,2 ± 1,8) y costes no-médicos/indirectos (0,5  ± 1,6). Los expertos observaron una emtrimejora con cabotegravir + rilpivirina  de acción prolongada en los resultados reportados por los pacientes  (2,7 ± 1,4). El beneficio terapéutico (3,5 ± 1,2) se consideró moderado-alto.  La evidencia de cabotegravir + rilpivirina de acción prolongada fue considerada  robusta (4,3 ± 0,8), con elevado consenso sobre su futura  recomendación en las guías (3,2 ± 1,0). En los criterios contextuales, el  impacto fue positivo en los criterios de prioridades de acceso (91%), objetivo  común (63%) y contexto político (60%). El impacto fue neutro en la capacidad  del sistema (40%) y los costes de oportunidad (51%). El resultado  promedio de la contribución del valor global de cabotegravir + rilpivirina de  acción prolongada fue de 0,34 (escala de ­1 a +1), siendo el criterio de  resultados reportados por el paciente el que proporcionó la mayor contribución de valor (0,04). CONCLUSIONES: Cabotegravir + rilpivirina de acción prolongada aporta un valor  añadido en el manejo del virus de la inmunodeficiencia humana 1 en  España en comparación con los regímenes de comprimido único utilizados  actualmente. Los expertos valoraron positivamente los resultados reportados  por los pacientes y el beneficio terapéutico de cabotegravir + rilpivirina de  acción prolongada, considerando que el beneficio esperado en la adherencia y  los problemas relacionados con el estigma produciría una mejora en la calidad  de vida de las personas con virus de la inmunodeficiencia humana 1.

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.

Contribución de cabotegravir + rilpivirina de acción prolongada al tratamiento de la infección por VIH-1 / Contribution of cabotegravir + rilpivirine long-acting for the treatment of HIV-1 infection

Objetivo: Determinar la contribución de valor de cabotegravir + rilpivirina, el primer tratamiento antirretroviral inyectable de acción prolongada,utilizando metodología de análisis de decisión multicriterio.Método: El estudio se desarrolló en dos fases: una prueba piloto y unafase de extensión, con un grupo multidisciplinar más grande. Se seleccionaron siete regímenes de comprimido único orales diarios recomendadosen las guías GeSIDA como comparadores. Se utilizó el marco EVIDEM,compuesto por 12 criterios cuantitativos y 5 contextuales. Los criterioscuantitativos se analizaron calculando la media y desviación estándar, ylos cualitativos se analizaron mediante el porcentaje de expertos que consideraron el impacto positivo, neutro o negativo para el Sistema Nacionalde Salud.Resultados: Un total de 35 expertos participaron en el estudio. Lainfección por virus de la inmunodeficiencia humana 1 se consideró grave(media ± desviación estándar: 3,0 ± 1,0), con un tamaño de poblaciónafectada (2,7 ± 1,2) y unas necesidades no cubiertas (2,8 ± 1,0) moderadas. Las diferencias fueron mínimas en los criterios comparativos deeficacia/efectividad (0,1 ± 0,5), seguridad/tolerabilidad (–0,5 ± 0,7) ycoste: coste del tratamiento (0,5 ± 2,0), otros costes médicos (0,2 ± 1,8)y costes no-médicos/indirectos (0,5 ± 1,6). Los expertos observaron una mejora con cabotegravir + rilpivirina de acción prolongada en los resultados reportados por los pacientes (2,7 ± 1,4). El beneficio terapéutico(3,5 ± 1,2) se consideró moderado-alto. La evidencia de cabotegravir+ rilpivirina de acción prolongada fue considerada robusta (4,3 ± 0,8),con elevado consenso sobre su futura recomendación en las guías(3,2 ± 1,0). En los criterios contextuales, el impacto fue positivo en loscriterios de prioridades de acceso (91%), objetivo común (63%) y contextopolítico (60%). (AU)

Objective: To determine the value contribution of cabotegravir + rilpivirine, the first injectable every two months long-acting antiretroviral regimen, using multi-criteria decision analysis.Method: The study was developed in two phases. After a small pilot,a field work study with a larger number of multidisciplinary experts wascarried out. Seven single-tablet regimens, currently recommended by theGeSIDA guidelines, were selected as comparators. EVIDEM methodology was followed, with a framework composed by 12 quantitative and5 contextual criteria. Mean and standard deviations were calculated forquantitative criteria (1 to 5 scale; comparative criteria –5 to +5), whereasqualitative criteria were analyzed as percentages of experts that considered a positive, neutral or negative impact for the National Health System.Results: 35 experts participated in the study. Human immunodeficiencyvirus-1 infection was considered severe (mean ± standard deviation:3.0 ± 1.0), with moderate size of affected population (2.7 ± 1.2) andunmet needs (2.8 ± 1.0). Minimal differences were found in comparative efficacy/effectiveness (0.1 ± 0.5), safety/tolerability (–0.5 ± 0.7),and cost criteria: cost of the intervention (0.5 ± 2.0), other medical costs(0.2 ± 1.8) and non-medical/indirect costs (0.5 ± 1.6). Experts perceived an improvement with cabotegravir + rilpivirine long-acting, compared to current daily oral single-tablet regimens, in patient-reported outcomes(2.7 ± 1.4). Therapeutic benefit of the long-acting regimen was considered moderate-to-high (3.5 ± 1.2). (AU)

Direct Medical Costs of Four Vaccine-Preventable Infectious Diseases in Older Adults in Spain.

OBJECTIVE: Protection against vaccine-preventable diseases is especially relevant in older adults due to age-related decline in immunity (immunosenescence). However, adult vaccination remains a challenge with overall low coverage rates, which has an impact on both the patients who have these diseases and the health care system in terms of resource use and costs derived. This study aimed to estimate the direct economic impact of herpes zoster, pneumococcal disease, influenza and pertussis in Spanish adults 45 years and older. METHODS: Data from 2015 were extracted from two Spanish public databases: the Minimum Basic Data Set for Hospitalisations and the Clinical Database of Primary Care. Codes from the International Classification of Diseases and the International Classification of Primary Care were used to identify and classify the diseases analysed. The variables extracted and calculated were hospitalisation (cases, percentage, length of stay, costs, mortality), primary care (cases, percentage, costs) and referrals (cases, percentage, costs). Results were presented for the age groups 45-64 years, 65-74 years, > 74 years and all ages. RESULTS: In adults 45 years and older, total costs amounted to €134.1 million in 2015 (i.e. 63.9% of the total direct costs for all age groups): 44.4% due to pneumococcal disease, 39.5% due to influenza, 16.0% due to herpes zoster and 0.1% due to pertussis. Hospitalisations represented 58.1% (€77.9 million) of the total costs, with 15,910 admissions, 144,752 days of hospitalisation and 1170 deaths. Primary care registered 566,556 visits with a cost of €35.0 million, and 269,186 referrals with a cost of €21.1 million. CONCLUSION: The direct economic burden of herpes zoster, pneumococcal disease, influenza and pertussis in adults 45 years and older was high in Spain, and may be underestimated as it only considered medical assistance and not other applicable direct or indirect costs. Increasing vaccination rates in adults may potentially reduce the economic burden derived from these diseases, although future cost-effectiveness analysis including other disease-related costs, vaccination costs and vaccination effectiveness would be needed.

Cost-consequence analysis of fluticasone furoate/vilanterol for asthma management in Spain: an analysis based on the Salford Lung Study in asthma.

OBJECTIVES: The Salford Lung Study in asthma (SLS asthma) is a 12-month, open-label randomised clinical trial comparing clinical effectiveness of initiating once-daily inhaled combination of fluticasone furoate/vilanterol (FF/VI) 184/22 mcg or 92/22 mcg, with continuing optimized usual care (UC) with inhaled corticosteroids (ICS) alone, or in combination with a long-acting ß2-agonist (ICS/LABA), in asthmatic patients followed in primary care in the UK. The objective of the analysis is to estimate the economic impact of these results when applied in Spain. METHODS: A 1-year cost-consequence model was populated with SLS asthma, adopting the Spanish National Health System (NHS) perspective. 775,900 of diagnosed asthmatic patients ≥ 18 years old currently managed with UC in Spain were included in the analysis. Effectiveness data included the percentage of patients per Asthma Control Test (ACT) category at 24 and 52 weeks from SLS asthma. Direct costs (pharmacological and per ACT category) were estimated from Spanish public sources and literature (€, 2018). Base case analysis assumed an increased use of FF/VI from 10 to 20% within 1 year. One-way sensitivity analyses were performed. RESULTS: Within the 775,900 asthmatic patients analysed, substitution of UC with FF/VI was associated with reduced costs due to ACT improvement, leading to potential total annual savings of €4,927,672. Sensitivity analyses ranged from €6,012,975 to €14,783,015 cost savings associated with FF/VI. An analysis considering patients only on ICS/LABA showed potential cost savings of €8,207,448. CONCLUSIONS: The improved asthma control for FF/VI compared with UC observed in SLS asthma could be translated into potential savings for the Spanish NHS. These results may be useful for decision makers.

Correction to: Cost-consequence analysis of fluticasone furoate/vilanterol for asthma management in Spain: an analysis based on the Salford Lung Study in asthma.

In the published article, the Table 1 was published incorrectly.

Cost-consequence analysis of fluticasone furoate/vilanterol 92/22 mcg for the management of COPD in the Spanish NHS.

OBJECTIVES: The Salford Lung Study in Chronic Obstructive Pulmonary Disease (SLS COPD) is a 12-month, open-label randomized clinical trial comparing clinical effectiveness and safety of initiating once-daily fluticasone furoate/vilanterol (FF/VI) 92/22 mcg with continuing usual care (UC) in patients with COPD followed in primary care in the UK. The objective of this analysis is to estimate the economic impact of these results when applied to Spain. MATERIALS AND METHODS: An Excel-based cost-consequence model with a one-year time horizon was populated with SLS COPD results, adopting the Spanish National Health System (NHS) perspective. Patients analyzed were diagnosed COPD patients ≥40 years old, currently managed with maintenance treatment and with a history of exacerbations (total number estimated from Spanish data). Mean least squares annual rates of moderate/severe exacerbations after 1 year for the intention-to-treat population from SLS COPD were included in the model (1.50 [FF/VI] and 1.64 [UC]); serious adverse events were excluded from the analysis as no differences between treatment arms were found. Medication and exacerbation management costs in euros were estimated from Spanish public sources for 2016. Model base-case analysis assumed an increased usage of FF/VI from 4% to 10% within 1 year, and a 100% proportion of days covered with study medications. Deterministic sensitivity analyses were performed for mitigating uncertainty. RESULTS: At base case, within 50,522 COPD patients analyzed, substitution of UC with FF/VI 92/22 mcg was associated with reduced medication and exacerbation management costs, leading to potential total annual savings of €353,623. Deterministic sensitivity results ranged from €218,333 up to €1,532,366 potential cost savings associated with FF/VI, showing the robustness of base-case results. CONCLUSION: The decreased rate of exacerbations with FF/VI 92/22 mcg compared with UC observed in SLS COPD could be translated into potential health care savings for the Spanish NHS. These results may be useful to inform decision-making processes.

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)

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

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 (€, 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€ per life year gained, with an incremental cost-utility ratio (ICUR) of 23,158€ 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€/ QALY. CONCLUSION: Belimumab can be regarded as a cost-effective alternative from the Spanish social perspective.

Objetivo: Estimar el coste-efectividad (CE) de belimumab en aquellos pacientes con biomarcadores positivos y enfermedad activa a pesar del tratamiento estandar (TE) desde la perspectiva social espanola. Métodos: A partir de un modelo de microsimulacion, que permite simular la evolucion natural de la enfermedad, se estimo el CE de belimumab + TE vs. TE. Se considero una duracion del tratamiento de dos anos y un horizonte temporal de toda la vida. La extrapolacion de eficacia a largo plazo se baso en los ensayos clinicos 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 espanoles publicados (€, 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 terminos de anos de vida ganados (AVG) y anos de vida ajustados por calidad (AVAC). Se realizaron analisis de sensibilidad deterministicos (TD al 0% y 5%, duracion de tratamiento 5 anos y exclusion de costes indirectos) asi como probabilisticos (PSA). Resultados: El ICER de belimumab + TE vs. TE fue de 16.647€/ AVG y 23.158€/AVAC respectivamente. La variacion de la TD supuso la mayor variacion 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€/AVAC. Conclusiones: Belimumab puede considerarse una alternativa coste-efectiva desde la perspectiva social espanola.