Resumo Fundamento O choque cardiogênico (CC) refratário está associado com altas taxas de mortalidade, e o uso de oxigenação por membrana extracorpórea venoarterial (VA-ECMO, do inglês venoarterial extracorporeal membrane oxygenation) como uma opção terapêutica tem gerado discussões. Nesse sentido, sua custo-efetividade, principalmente em países de baixa e média renda como o Brasil, continua incerto.Objetivos: Conduzir uma análise de custo-efetividade na perspectiva do Sistema Único de Saúde (SUS) para avaliar a custo-efetividade de VA-ECMO combinado com o tratamento padrão em comparação ao tratamento padrão isolado em pacientes adultos com CC refratário. Métodos Acompanhamos uma coorte de pacientes com CC refratário tratados com VA-ECMO em centros de assistência terciária do sul brasileiro. Coletamos dados de desfechos e custos hospitalares. Realizamos uma revisão sistemática para complementar nossos dados e usamos o modelo de Markov para estimar a razão de custo-efetividade incremental (RCEI) por ano de vida ajustado pela qualidade (QALY) e por ano de vida ganho. Resultados Na análise do caso-base, a VA-ECMO gerou uma RCEI de Int$ 37 491 por QALY. Análises de sensibilidade identificaram o custo de internação, o risco relativo de sobrevida, e a sobrevida do grupo submetido à VA-ECMO como principais variáveis influenciando os resultados. A análise de sensibilidade probabilística mostrou um benefício do uso de VA-ECMO, com uma probabilidade de 78% de custo-efetividade no limiar recomendado de disposição a pagar. Conclusões Nosso estudo sugere que, dentro do SUS, VA-ECMO pode ser uma terapia custo-efetiva para o CC refratário. Contudo, a escassez de dados sobre a eficácia e de ensaios clínicos recentes que abordem seus benefícios em subgrupos específicos de pacientes destaca a necessidade de mais pesquisas. Ensaios clínicos rigorosos, incluindo perfis diversos de pacientes, são essenciais para confirmar a custo-efetividade com uso de VA-ECMO e assegurar acesso igualitário a intervenções médicas avançadas dentro dos sistemas de saúde, especialmente em países com desigualdades socioeconômicas como o Brasil. (CC mortalidade VAECMO, VAECMO VA ECMO, ECMO (VA-ECMO oxygenation discussões sentido custoefetividade, custoefetividade efetividade, efetividade Brasil incerto.Objetivos incertoObjetivos incerto Objetivos SUS (SUS brasileiro hospitalares (RCEI QALY (QALY ganho casobase, casobase caso base, base caso-base Int 3 49 internação resultados 78 pagar custoefetiva efetiva Contudo pesquisas rigorosos saúde 4 7

Abstract Background Refractory cardiogenic shock (CS) is associated with high mortality rates, and the use of venoarterial extracorporeal membrane oxygenation (VA-ECMO) as a therapeutic option has generated discussions. Therefore, its cost-effectiveness, especially in low- and middle-income countries like Brazil, remains uncertain.Objectives: To conduct a cost-utility analysis from the Brazilian Unified Health System perspective to assess the cost-effectiveness of VA-ECMO combined with standard care compared to standard care alone in adult refractory CS patients. Methods We followed a cohort of refractory CS patients treated with VA-ECMO in tertiary care centers located in Southern Brazilian. We collected data on hospital outcomes and costs. We conducted a systematic review to supplement our data and utilized a Markov model to estimate incremental cost-effectiveness ratios (ICERs) per quality-adjusted life year (QALY) and per life-year gained. Results In the base-case analysis, VA-ECMO yielded an ICER of Int$ 37,491 per QALY. Sensitivity analyses identified hospitalization cost, relative risk of survival, and VA-ECMO group survival as key drivers of results. Probabilistic sensitivity analysis favored VA-ECMO, with a 78% probability of cost-effectiveness at the recommended willingness-to-pay threshold. Conclusions Our study suggests that, within the Brazilian Health System framework, VA-ECMO may be a cost-effective therapy for refractory CS. However, limited efficacy data and recent trials questioning its benefit in specific patient subsets highlight the need for further research. Rigorous clinical trials, encompassing diverse patient profiles, are essential to confirm cost-effectiveness and ensure equitable access to advanced medical interventions within healthcare systems, particularly in socio-economically diverse countries like Brazil. (CS rates VAECMO VA ECMO (VA-ECMO discussions Therefore costeffectiveness, costeffectiveness cost effectiveness, effectiveness low middleincome middle income Brazil uncertain.Objectives uncertainObjectives uncertain Objectives costutility utility costs ICERs (ICERs qualityadjusted quality adjusted QALY (QALY lifeyear gained basecase base case Int 37491 37 491 37,49 results VAECMO, ECMO, 78 willingnesstopay willingness pay threshold that framework costeffective effective However research profiles systems socioeconomically socio economically 3749 3 49 37,4 7 374 4 37,

Resumen Objetivo: Evaluar el coste-efectividad de diferentes estrategias de vacunación universal infantil frente a la hepatitis A en España. Método: A partir de un modelo dinámico y un árbol de decisión, se realizó un análisis de coste-efectividad para comparar tres estrategias de vacunación frente a la hepatitis A: no vacunación y vacunación universal infantil con una y dos dosis. La perspectiva del estudio escogida fue la del Sistema Nacional de Salud (SNS) y se consideró como horizonte temporal toda la vida del paciente. Tanto los costes como los efectos se descontaron al 3% anual. Los resultados en salud se midieron en años de vida ajustados por calidad (AVAC) y la medida de coste-efectividad utilizada es la razón de coste-efectividad incremental (RCEI). Además, se llevaron a cabo análisis de sensibilidad determinísticos por escenarios. Resultados: En el caso particular de España, con baja endemicidad de hepatitis A, las diferencias en resultados en salud entre las distintas estrategias de vacunación (con una o dos dosis) y la no vacunación son prácticamente inexistentes, en términos de AVAC. Además, las RCEI obtenidas son elevadas, superando los límites establecidos de disposición a pagar obtenidos en España (22.000-25.000 €/AVAC). El análisis de sensibilidad determinístico muestra que los resultados son sensibles a las variaciones de los parámetros clave, aunque en ningún caso resultan coste-efectivos. Conclusiones: La vacunación universal infantil frente a la hepatitis A no sería una opción coste-efectiva desde la perspectiva del SNS en España en la actualidad.

Abstract Objective: To evaluate the cost-effectiveness of infant universal vaccination against hepatitis A in Spain. Method: Using a dynamic model and decision tree model, a cost-effectiveness analysis was performed to compare three vaccination strategies against hepatitis A: non-vaccination strategy versus universal childhood vaccination of hepatitis A with one or two doses. The perspective of the study was that of the National Health System (NHS) and a lifetime horizon was considered. Both costs and effects were discounted at 3% per year. Health outcomes were measured in terms of quality adjusted life years (QALY) and the cost-effectiveness measure used was the incremental cost-effectiveness ratio (ICER). In addition, deterministic sensitivity analysis by scenarios was performed. Results: In the particular case of Spain, with low endemicity for hepatitis A, the difference in health outcomes between vaccination strategies (with 1 or 2 doses) and non-vaccination are practically non-existent, terms of QALY. In addition, the ICER obtained is high, exceeding the limits of willingness to pay from Spain (€22,000-25,000/QALY). The deterministic sensitivity analysis showed that the results are sensitive to the variations of the key parameters, although in no case the vaccination strategies are cost-effective. Conclusions: Universal infant vaccination strategy against hepatitis A would not be a cost-effective option from the NHS perspective in Spain.

Abstract Objective: To evaluate the cost-effectiveness of infant universal vaccination against hepatitis A in Spain. Method: Using a dynamic model and decision tree model, a cost-effectiveness analysis was performed to compare three vaccination strategies against hepatitis A: non-vaccination strategy versus universal childhood vaccination of hepatitis A with one or two doses. The perspective of the study was that of the National Health System (NHS) and a lifetime horizon was considered. Both costs and effects were discounted at 3% per year. Health outcomes were measured in terms of quality adjusted life years (QALY) and the cost-effectiveness measure used was the incremental cost-effectiveness ratio (ICER). In addition, deterministic sensitivity analysis by scenarios was performed. Results: In the particular case of Spain, with low endemicity for hepatitis A, the difference in health outcomes between vaccination strategies (with 1 or 2 doses) and non-vaccination are practically non-existent, terms of QALY. In addition, the ICER obtained is high, exceeding the limits of willingness to pay from Spain (€22,000-25,000/QALY). The deterministic sensitivity analysis showed that the results are sensitive to the variations of the key parameters, although in no case the vaccination strategies are cost-effective. Conclusions: Universal infant vaccination strategy against hepatitis A would not be a cost-effective option from the NHS perspective in Spain.

Resumen Objetivo: Evaluar el coste-efectividad de diferentes estrategias de vacunación universal infantil frente a la hepatitis A en España. Método: A partir de un modelo dinámico y un árbol de decisión, se realizó un análisis de coste-efectividad para comparar tres estrategias de vacunación frente a la hepatitis A: no vacunación y vacunación universal infantil con una y dos dosis. La perspectiva del estudio escogida fue la del Sistema Nacional de Salud (SNS) y se consideró como horizonte temporal toda la vida del paciente. Tanto los costes como los efectos se descontaron al 3% anual. Los resultados en salud se midieron en años de vida ajustados por calidad (AVAC) y la medida de coste-efectividad utilizada es la razón de coste-efectividad incremental (RCEI). Además, se llevaron a cabo análisis de sensibilidad determinísticos por escenarios. Resultados: En el caso particular de España, con baja endemicidad de hepatitis A, las diferencias en resultados en salud entre las distintas estrategias de vacunación (con una o dos dosis) y la no vacunación son prácticamente inexistentes, en términos de AVAC. Además, las RCEI obtenidas son elevadas, superando los límites establecidos de disposición a pagar obtenidos en España (22.000-25.000 €/AVAC). El análisis de sensibilidad determinístico muestra que los resultados son sensibles a las variaciones de los parámetros clave, aunque en ningún caso resultan coste-efectivos. Conclusiones: La vacunación universal infantil frente a la hepatitis A no sería una opción coste-efectiva desde la perspectiva del SNS en España en la actualidad.

Abstract Aim: The study aimed to conduct a cost-utility analysis of traditional drug therapy (TDT) provided for hypertensive patients at primary care in comparison to the protocol based on combination with an exercise program (TDT+E) in real-life conditions, adopting a health system perspective. Methods: Longitudinal study based on enrollment of 49 hypertensive adults distributed into two groups, for 12 months. Quality-adjusted life years were estimated using health-related quality of life. Direct health care costs were calculated including inputs and human resources in primary care from medical records. Sensitivity analysis was performed based on multivariate and probabilistic scenarios. Results: Incremental cost-effectiveness ratios of TDT+E in comparison to TDT were +79.69. Sensitivity analysis showed that TDT+E presented advantages considering uncertainties. Conclusion: Our findings show that exercise programs may improve quality of life and life expectancy among hypertensive patients.

RESUMO Objetivo Avaliar a relação custo-utilidade do iStent inject® para o tratamento do glaucoma de ângulo aberto leve a moderado no Sistema Único de Saúde. Métodos Foi desenvolvido um modelo de Markov, no qual a medida de resultado de efetividade foi a razão custo-efetividade incremental (razão de custo-efetividade incremental: R$/ano de vida ajustado pela qualidade). Os custos médicos diretos foram obtidos por meio da perspectiva do Sistema Único de Saúde. O caso base foi composto de uma coorte hipotética de pacientes com glaucoma de ângulo aberto em uso de medicação tópica tratados de acordo com o Protocolo Clínico e Diretrizes Terapêuticas e um cenário do mundo real baseado em dados do Departamento de Informática do Sistema Único de Saúde. Foi testada a robustez do modelo por meio de análises de sensibilidade. Resultados No cenário base do Protocolo Clínico e Diretrizes Terapêuticas, o implante trabecular micro-bypass proporcionou ganhos de 0,47 ano de vida ajustado pela qualidade e razão de custo-efetividade incremental de R$7.996,66/ano de vida ajustado pela qualidade em relação ao tratamento com medicação tópica. No cenário real baseado em dados do Departamento de Informática do Sistema Único de Saúde, o implante trabecular proporcionou ganhos de 0,47 ano de vida ajustado pela qualidade e razão de custo-efetividade incremental de R$ 4.485,68/ano de vida ajustado pela qualidade em relação ao tratamento com medicação tópica. Os resultados foram robustos para análises de sensibilidade. Conclusão A incorporação do iStent inject® ao Sistema Único de Saúde proporciona melhora na qualidade de vida do paciente com um custo adicional que garante o benefício proporcionado a eles. Os resultados podem ser considerados custo-efetivos em comparação com a medicação tópica.

ABSTRACT Purpose To evaluate the cost-utility of the iStent inject® for the treatment of mild-to-moderate open-angle glaucoma (OAG) within the Brazilian Unified Health System (SUS). Methods A Markov model was developed, in which the effectiveness outcome measure was the incremental cost-effectiveness ratio (ICER: R$ / QALY quality-adjusted life-year). Direct medical costs were obtained from the SUS perspective. The base case comprised of a hypothetical cohort of patients with OAG using topical medication and being managed according to the Clinical Protocol and Therapeutic Guidelines (PCDT) and a real-world setting based on data from Datasus. The model’s robustness through sensitivity analyses was tested. Results In the PCDT base case setting, the trabecular micro-bypass implant provided gains of 0.47 QALYs and an ICER of R$7,996.66/QALY compared to treatment with topical medication. In the real-world setting based on data from Datasus, the trabecular micro-bypass implant, provided gains of 0.47 QALYs and an ICER of R$4,485.68/QALY compared to treatment with topical medication. The results were robust to sensitivity analyses. Conclusion Incorporating iStent inject® to SUS provides an improvement in the patient’s quality of life with an additional cost that warrants the benefit provided to patients. Results may be considered cost-effective compared to topical medication.

Abstract Background: Implantable cardiac defibrillators (ICDs) therapy for primary prevention (PP) of sudden cardiac arrest (SCA) is well-established but underutilized globally. The Improve SCA study has identified a cohort of patients called 1.5 primary prevention (1.5PP), based on PP patients with the presence of documented risk factors: non-sustained ventricular tachycardia, frequent premature ventricular contractions, left ventricular ejection fraction < 25%, and pre-syncope or syncope. Objective: This study evaluated the cost-effectiveness of ICD therapy compared to no ICD among 1.5PP patients in the Brazilian public healthcare system. Methods: Modified inputs to a published Markov model were applied to compare costs and outcomes of ICD therapy to no ICD therapy from the Brazilian payer’s perspective. Mortality and utility estimates were obtained from the IMPROVE SCA trial. Additional effectiveness inputs were sourced from the literature. Cost inputs were obtained from the Brazilian Unified Health System and the Ministry of Health. Costs were discounted at 4.7%; quality-adjusted life years (QALYs) were discounted at 1.45%. This study applied a willingness-to-pay (WTP) value of three times Brazil’s gross domestic product (GDP) in 2017, R$105,723 (Brazilian Real). Results: The total discounted lifetime costs for ICD therapy were R$100,920 compared to R$43,866 for no ICD therapy. Total discounted QALYs for ICD therapy and no ICD therapy were 9.85 and 7.15, respectively. The incremental cost effectiveness ratio was R$21,156 per QALY and less than the R$105,723 WTP threshold. Results from sensitivity analyses were consistent with base case results. Conclusions: ICD therapy compared to no ICD therapy is cost-effective in the 1.5PP population in Brazil. (Int J Cardiovasc Sci. 2021; [online].ahead print, PP.0-0) Background ICDs (ICDs (PP (SCA wellestablished well established globally 15 1 5 1. 1.5PP, 15PP , 5PP (1.5PP) factors nonsustained non sustained tachycardia contractions 25 25% presyncope pre syncope Objective costeffectiveness system Methods payers payer s perspective trial literature 4.7% 47 4 7 qualityadjusted quality adjusted (QALYs 145 45 1.45% willingnesstopay willingness pay (WTP Brazils Brazil GDP (GDP 2017 R105723 R 105 723 R$105,72 Real. Real . Real) R100920 100 920 R$100,92 R43866 43 866 R$43,86 985 9 85 9.8 715 7.15 respectively R21156 21 156 R$21,15 threshold results Conclusions costeffective effective Int Sci 2021 online.ahead onlineahead online .ahead ahead print PP.00 PP00 PP.0 0 PP.0-0 (1.5PP 2 4.7 14 1.45 201 R10572 10 72 R$105,7 R10092 92 R$100,9 R4386 86 R$43,8 98 8 9. 71 7.1 R2115 R$21,1 202 00 PP0 PP. PP.0- 4. 1.4 20 R1057 R$105, R1009 R$100, R438 R$43, 7. R211 R$21, R105 R$105 R100 R$100 R43 R$43 R21 R$21 R10 R$10 R4 R$4 R2 R$2 R1 R$1 R$

Resumo: Introdução e Objetivos: O cancro colorretal (CCR) é uma das neoplasias mais comuns na Europa. Recentemente, temos observado um aumento da incidência de cancro colorretal (CCR) em indivíduos <50 anos (não abrangidos pelos programas de rastreio), tanto na europa como nos Estados Unidos da América (EUA). Simultaneamente, a taxa de mortalidade (TM) permaneceu estável em doentes <55 anos e diminuiu >55 anos. Baseado nestes dados, a American Cancer Society (ACS) publicou uma recomendação qualificada advogando o início do rastreio aos 45 anos. Avaliar se as alterações na incidência/mortalidade de CCR observadas nos EUA/Europa também ocorrem em Portugal e realizar uma análise de custo-utilidade do início do rastreio de CCR aos 45 anos. Métodos: Ava liamos a incidência de CCR por faixa etária usando dados do Registro Oncológico Nacional (1993-2010) e TM de acordo com o Instituto Nacional de Estatística (2003-2016). A análise de custo-utilidade foi realizada com uma árvore de decisão sob uma perspetiva social, comparando o teste imunoquímico fecal bienal (FIT) com a realização de uma colonoscopia total aos 45 anos. Resultados: Em Portugal (1993-2010) observou-se um aumento na incidência de CRC de 17% (25/100.000 vs. 30/100.000), 35% (39/100.000 vs. 54/100.000) e 71% (52/100.000 vs. 97/100.000) em doentes com 45-49 anos, 50-54 anos e 55-59 anos, respetivamente. A TM de indivíduos com 45-54 anos permaneceu estável (12/100.000) ao contrário da diminuição moderada em indivíduos com 55-64 (38/100.000 vs. 35/100.000) e uma acentuada redução em 65-75 (93/100.000 vs. 75/100.000). O rastreio de CCR aos 45 anos não teve custo-utilidade na presente incidência (FIT/colonoscopia total). O rastreio com FIT forneceu um RCEI de € 84.304/QALY, enquanto a colonoscopia forneceu um RCEI de € 3.112.244/QALY. Em análise de sensibilidade unilateral, o rastreio com FIT apresentaria custo-utilidade com o custo atual da colonoscopia sob sedação (€ 150) e taxas de aceitação apenas se a incidência subir acima de 47,5/100.000; o rastreio com colonoscopia nesta idade nunca teria custo-utilidade, apesar das mudanças nos custos e/ou taxas de incidência. Conclusão: Em Portugal, a incidência de CCR em doentes com idades entre 45-55 anos tem aumentado (TM estável). Este cenário é semelhante ao descrito nos EUA/restante europa. No entanto, o início do rastreio do CCR aos 45 anos em Portugal apenas terá custo-utilidade se incidência for >47.5/100.000 (vs. 30/100.000).

Abstract: Background and Objective: Colorectal cancer (CRC) is one of the most common cancers in Europe. Recently, new data from the USA and Europe revealed an increase in the incidence of CRC in individuals aged <55 years and a reduction in those aged >65 years. Mortality rate was stable in patients aged <55 years and decreased after the age of 55 years. Based on the USA data, the American Cancer Society (ACS) published a qualified recommendation advocating the start of CRC screening at the age of 45 years. We aimed to evaluate if the changes in the CRC incidence/mortality observed in the USA and the rest of Europe also occur in Portugal, and then perform a cost-utility analysis of CRC screening that starts at 45 years of age. Methods: We evaluated the incidence of CRC by age group using data from the National Cancer Registry, and the mortality rate according to the National Statistics Institute in the periods 1993-2010 and 2003-2016. A cost-utility analysis was performed with a decision tree from a societal perspective comparing biennial fecal immunochemical test (FIT) or a single colonoscopy screening versus nonscreening at the age of 45 years in Portugal. Results: In Portugal, in 1993-2010, there was an increase in CRC incidence of 17% (from 25 to 30/100,000), 35% (from 39 to 54/100,000), and 71% (from 52 to 97/100,000) in patients aged 45-49 years, 50-54 years, and 55-59 years, respectively. The mortality rate of patients aged 45-54 years remained stable between 2003 and 2016 (12/100,000) as a counterpoint to a moderate decrease in those aged 55-64 years (from 38 to 35/100,000) and a sharp reduction in those aged 65-75 years (from 93 to 75/100,000). Screening for CRC at the age of 45 years has no cost utility with the current incidence. FIT screening provided an ICUR of EUR 84,304/quality-adjusted life years (QALY) while colonoscopy provided an ICUR of EUR 3,112,244/QALY. On one-way sensitivity analysis, FIT screening would only have cost utility at the present cost of colonoscopy under sedation (EUR 150) and acceptance rates if the incidence rate rises above 47.5/100,000; colonoscopy at this age would have no cost utility despite changes in costs and/or incidence rates. Conclusion: In Portugal, the incidence of CRC in patients aged 45-55 years has been increasing with a stable mortality rate, in contrast to the decrease in mortality in the age groups covered by the current CRC screening program. However, at present, CRC screening in Portugal at the age of 45 years has no cost utility and will only have this if the incidence rate rises above 47.5/100,000 (vs. the actual incidence of 30/100,000).

Abstract Background: Ceftazidime-avibactam (C/A), has shown reduction in mortality rates and risk of nephrotoxicity, compared to colistin, conventional therapy. Aim: To estimate the cost-effectiveness of C/A versus colistin + meropenem in the treatment of infections due to carbapenem-resistant Enterobacteriaceae (CRE) in Chile. Methods: An economic decision tree type model was adapted. The perspective of the public payer was used with a time horizon of 30 days and extrapolation to life expectancy. The clinical information was derived from an observational study. Medication and care costs correspond to local reports. The results are expressed as incremental cost-effectiveness ratio (ICER) per life year gained (LYG) and per quality adjusted life year (QALY) in Chilean pesos and US dollars (US$ 1.00 = $792.2218). Results: 8.65 and 6.48 LYGs and 6.44 and 4.27 QALYs were obtained, for C/A and colistin + meropenem, respectively. The estimated ICER for C/A was $940,488 (US$1,187.2) per AVG and $938,715 (US$1,184.9) per QALY. Discussion: Given the lack of publications or evidence, the model is based on an observational study. C/A would reduce the death rate and increase LYGs and QALYs, resulting in a cost-effective alternative vs. colistin + meropenem for CRE.

Resumen Introducción: Ceftazidima-avibactam (C/A), ha demostrado reducir las tasas de mortalidad y el riesgo de nefrotoxicidad, comparado con colistin, la terapia convencional. Objetivo: Estimar la costo-efectividad de C/A versus colistin + meropenem en el tratamiento de infecciones por Enterobacteriaceae resistentes a carbapenémicos (ERC) en Chile. Material y Métodos: Se adaptó un modelo económico tipo árbol de decisión. Se utilizó la perspectiva del pagador público, un horizonte de tiempo de 30 días con extrapolación a la expectativa de vida. La información clínica se derivó de un estudio observacional. Los costos de los medicamentos y de atención corresponden a reportes locales. Los resultados se expresan como razón de costo-efectividad incremental (RCEI) por año de vida ganado (AVG) y por año de vida ajustado por calidad (AVAC) en pesos chilenos y en dólares estadounidenses (US$ 1,00 = $792,2218). Resultados: Se obtuvieron 8,65 y 6,48 AVGs y 6,44 y 4,27 AVACs, para C/A y colistin + meropenem, respectivamente. La RCEI estimada de C/A fue $940.488 (US$1.187,2) por AVG y $938.715 (US$1.184,9) por AVAC. Discusión: Dada la falta de publicaciones o evidencia, el modelo se basa en un estudio observacional. C/A reduciría la proporción de muertes e incrementaría los AVG y los AVAC, resultando en una alternativa costo-efectiva versus colistin + meropenem para ERC.

Resumen Este trabajo tiene por objetivo identificar los principales instrumentos de medición de estados de salud y las metodologías de valoración de preferencias que se utilizan para calcular los años de vida ajustados por calidad -AVAC-. A partir de una revisión de literatura con una estrategia de búsqueda equilibrada para la identificación de referencias bibliográficas relevantes, se presentan las características técnicas de los sistemas de clasificación en salud, como lo son los conceptos funcionales -sociales, mentales, físicos y sensoriales-, la combinación de atributos, el número de estados de salud diferentes, la duración de la encuesta y su administración. Asimismo, referente a las metodologías de valoración de preferencias, se resumen las particularidades sobre la consistencia con la teoría de los AVAC, la flexibilidad en la descripción de estados de salud, los estados peores que la muerte, entre otros aspectos. Se concluye que no existe un sistema de clasificación de estados de salud o método de valoración de preferencias mejor que otro, por lo que el investigador debe elegir la herramienta que mejor se adapte a la condición bajo análisis y al diseño de estudio en específico. JEL: I10, B41, D60.

Abstract The purpose of this research is to identify the main instruments for measuring health states and the methodologies for evaluating preferences, which are used to calculate quality-adjusted life years -QALYs-. From a literature review with a balanced search strategy to identify relevant bibliographic references, the technical characteristics of health classification systems are presented, such as functional concepts -social, mental, physical and sensory-, the combination of attributes, the number of different health states, the duration of the survey and its administration. Likewise, referring to the methodologies of valuation of preferences, the particularities on the consistency with the QALY theory, the flexibility in the description of health states, states worse than death, among other aspects, are summarized. It is concluded that there is no one better health status classification system or preference assessment method than another, so the researcher must choose the tool that best suits the condition under analysis and the specific study design. JEL: I10, B41, D60.

Resumen Objetivo En este estudio se realiza una evaluación económica de un programa de vigilancia para prevenir la luxación de cadera en niños y niñas con parálisis cerebral. Método Se desarrolló un modelo que comparó los costes y los resultados en salud de niños y niñas con parálisis cerebral incluidas y no incluidas en un programa de vigilancia. Se combinó la información proveniente de diversas fuentes en un modelo analítico de decisión, principalmente basado en datos de un estudio comparativo con 20 años de seguimiento. La efectividad se midió empleando los años de vida ajustados por calidad (AVAC). El análisis tomó la perspectiva del Sistema Nacional de Salud de España. Se realizó un extenso análisis de sensibilidad, incluyendo un análisis de sensibilidad probabilístico. Resultados El programa de vigilancia estuvo asociado a más AVAC y mayores costes sanitarios, con un coste incremental por AVAC ganado estimado en 12.282 €. Los resultados fueron robustos a los supuestos del modelo. La probabilidad de que el programa fuera coste-efectivo se estimó en un valor por encima del 80% para el umbral de 25.000 € por AVAC recomendado en España. Conclusión Este estudio indica que es probable que los programas de vigilancia para prevenir la luxación de cadera en niños y niñas con parálisis cerebral sean coste-efectivos.

Abstract Objective In this study we conducted an economic evaluation of a surveillance programme to prevent hip dislocation in children with cerebral palsy. Method We developed a model that compared costs and health outcomes of children with cerebral palsy with and without a surveillance programme. Information from a number of sources was combined into a decision analytical model, primarily based on data from a comparative study with a 20-year follow-up. Effectiveness was measured using Quality-Adjusted Life Years (QALYs). The analysis took the perspective of the Spanish National Health Service. We undertook extensive sensitivity analyses including a probabilistic sensitivity analysis. Results The surveillance programme led to higher QALYs and higher health care costs, with an estimated incremental cost per QALY gained of 12,282€. The results were robust to model assumptions. The probability that the programme was cost-effective was estimated to be over 80% at the threshold of 25.000€/QALY recommended in Spain. Conclusion This study indicates that surveillance programmes to prevent hip dislocation in children with cerebral palsy are likely to be cost-effective.

ABSTRACT Objectives: To evaluate the cost-effectiveness of direct-acting antiviral (DAAs) treatment versus non-treatment in prisoners awaiting treatment for chronic hepatitis C (CHC) and to analyse the clinical and economic impact of the treatment on liver complications and mortality. Material and method: A lifetime Markov model was developed to simulate treatment and disease progression from an estimated cohort of 4,408 CHC prisoners treated with DAAs over 2 years (50% of patient each year) versus no treatment. In the treated cohort, a sustained viral response of 95% was associated. Patient characteristics, transition probabilities, utilities and costs (pharmacological and healthcare states) were obtained from published literature. The model estimated healthcare costs and benefits, incremental cost-utility ratio (ICUR) based on total costs and the quality-adjusted life year (QALY) and avoided clinical events. A National Healthcare System perspective was adopted with a 3% annual discount rate for both costs and health outcomes. Sensitivity analyses were performed to assess uncertainty. Results: In the DDA treated cohort, the model estimated a decrease of 92% of decompensated cirrhosis and 83% of hepatocellular carcinoma, 88% liver-related mortality cases were reduced, 132 liver transplants were avoided. The treatment achieved an additional 5.0/QALYs (21.2 vs. 16.2) with an incremental cost of €3,473 (€24,088 vs. €20,615) per patient with an ICUR of €690 per QALY gained. Discussion: Considering the willingness-to-pay threshold used in Spain (€22,000-30,000/QALY), DAAs treatment for prisoners with CHC is a highly cost-effective strategy, reduces infection transmission, increases survival and reduces complications due to liver disease, as well as the cost associated with its management.

RESUMEN Objetivos: Evaluar, en presos con hepatitis C crónica (HCc) pendientes de tratamiento, el coste-efectividad de tratarlos con antivirales de acción directa (AAD) frente a no tratarlos, y analizar el impacto clínico y económico del tratamiento sobre las complicaciones hepáticas y la mortalidad. Material y método: Se desarrolló un modelo de Markov para simular el tratamiento y proyectar la progresión de la enfermedad de una cohorte estimada de 4.408 reclusos con HCc tratados con AAD en dos años (el 50% cada año) frente a ningún tratamiento. En los tratados, se asoció una respuesta viral sostenida del 95%. Las características de los pacientes, las probabilidades de transición, las utilidades y los costes (farmacológicos y estados de salud) se obtuvieron de publicaciones científicas. El modelo estimó costes y resultados en salud, la relación coste-utilidad incremental (RCUI), a partir de los costes totales y años de vida ajustados por calidad (AVAC) y los eventos clínicos evitados. La perspectiva fue la del Sistema Nacional de Salud (SNS) y se aplicó una tasa de descuento anual (el 3%) para costes y resultados en salud. La robustez de los resultados se evaluó mediante un análisis de sensibilidad. Resultados: En la población tratada, el modelo estimó una reducción del 92% de los casos de cirrosis descompensada y del 83% de carcinoma hepatocelular, se evitaron 132 trasplantes hepáticos y disminuyó el 88% la mortalidad hepática. El tratamiento consiguió 5,0/AVAC adicionales (21,2 frente al 16,2), con un coste incremental de 3.473 euros (24.088 € frente a 20.615 €) por paciente y una RCUI de 690 €/AVAC ganado. Discusión: Considerando el umbral de disponibilidad a pagar utilizado en España (22.000-30.000 €/AVAC), el tratamiento con AAD en los presos con HCc es una estrategia altamente coste-efectiva, reduce la transmisión, aumenta la supervivencia y reduce las complicaciones hepáticas, así como los costes asociado a su manejo.

Abstract Objective In this study we conducted an economic evaluation of a surveillance programme to prevent hip dislocation in children with cerebral palsy. Method We developed a model that compared costs and health outcomes of children with cerebral palsy with and without a surveillance programme. Information from a number of sources was combined into a decision analytical model, primarily based on data from a comparative study with a 20-year follow-up. Effectiveness was measured using Quality-Adjusted Life Years (QALYs). The analysis took the perspective of the Spanish National Health Service. We undertook extensive sensitivity analyses including a probabilistic sensitivity analysis. Results The surveillance programme led to higher QALYs and higher health care costs, with an estimated incremental cost per QALY gained of 12,282€. The results were robust to model assumptions. The probability that the programme was cost-effective was estimated to be over 80% at the threshold of 25.000€/QALY recommended in Spain. Conclusion This study indicates that surveillance programmes to prevent hip dislocation in children with cerebral palsy are likely to be cost-effective.

Resumen Objetivo En este estudio se realiza una evaluación económica de un programa de vigilancia para prevenir la luxación de cadera en niños y niñas con parálisis cerebral. Método Se desarrolló un modelo que comparó los costes y los resultados en salud de niños y niñas con parálisis cerebral incluidas y no incluidas en un programa de vigilancia. Se combinó la información proveniente de diversas fuentes en un modelo analítico de decisión, principalmente basado en datos de un estudio comparativo con 20 años de seguimiento. La efectividad se midió empleando los años de vida ajustados por calidad (AVAC). El análisis tomó la perspectiva del Sistema Nacional de Salud de España. Se realizó un extenso análisis de sensibilidad, incluyendo un análisis de sensibilidad probabilístico. Resultados El programa de vigilancia estuvo asociado a más AVAC y mayores costes sanitarios, con un coste incremental por AVAC ganado estimado en 12.282 €. Los resultados fueron robustos a los supuestos del modelo. La probabilidad de que el programa fuera coste-efectivo se estimó en un valor por encima del 80% para el umbral de 25.000 € por AVAC recomendado en España. Conclusión Este estudio indica que es probable que los programas de vigilancia para prevenir la luxación de cadera en niños y niñas con parálisis cerebral sean coste-efectivos.

RESUMO Objetivo: Estudar o custo-efetividade do ranibizumabe e bevacizumabe no tratamento da degeneração macular relacionada à idade neovascular. Métodos: Utilizamos um modelo de árvore de decisão para analisar a relação custo-efetividade do ranibizumabe e bevacizumabe no tratamento da degeneração macular relacionada à idade, sob a perspectiva do Sistema Único de Saúde. O ranibizumabe e bevacizumabe foram administrados a pacientes com o mesmo procedimento de tratamento, e a diferença nos custos do tratamernto foi calculada com base no custo dos medicamentos. Os custos diretos foram estimados utilizando as informações fornecidas pelo SUS. A efetividade foi determinada em anos de vida ajustados pela qualidade (QALY) baseados em valores de utilidade em deficiênciavisual. A razãoincremental custo-efetividadefoicalculada comparando os dois tratamentos. O horizonte analítico foi de um ano. Resultados: A análise da árvore de decisão mostrou que a diferença na efetividade do tratamento foi de 0,01 QALY. A razão incremental de custo-efetividade mostrou que o tratamento com ranibizumabe exigiu um custo anual incremental de R$ 2 milhões para gerar um QALY adicional, em comparação ao bevacizumabe. Conclusões: Do ponto de vista do SUS, o bevacizumabe é mais custo-efetivo que o ranibizumabe no tratamento da degeneração macular relacionada à idade neovascular. O seu uso poderia gerar uma grande economia anual para o orçamento em saúde.

ABSTRACT Purpose: To study the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration. Methods: We used a decision tree model to analyze the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration, from the Brazilian Public Health System (SUS) perspective. Ranibizumab and bevacizumab were administered to patients with the same treatment procedure, and the difference in treatment costs was calculated based on the cost of the drugs. Direct costs were estimated using the information provided by the Brazilian SUS. Effectiveness in terms of quality-adjusted life years (QALYs) was calculated based on the utility values for visual impairment. Incremental cost-effectiveness ratio was calculated by comparing both treatments. The analytical horizon was one year. Results: The decision tree analysis showed that the difference in treatment effectiveness was 0.01 QALY. Incremental cost-effectiveness ratio showed that ranibizumab treatment required an incremental annual cost of more than R$ 2 million to generate 1 additional QALY, as compared to bevacizumab. Conclusions: From the Brazilian SUS perspective, bevacizumab is more cost-effective than ranibizumab for the treatment of neovascular age-related macular degeneration. Its use could allow potential annual savings in health budget.

ABSTRACT Objective To carry out a cost-utility analysis of the application of the Oncotype genomic test to inform the decision to use or not to use chemotherapy in the Basque Country (Spain). Method The cost-utility study was carried out using a discrete event simulation model representing the natural history of breast cancer. The decision of treatment with chemotherapy based on Oncotype was compared with the standard of treatment based on clinical-pathological criteria. The model included clinical data from Basque hospitals and the literature and was processed by deterministic and probabilistic analysis to calculate the incremental cost-effectiveness ratio (ICER), the cost-effectiveness plane, the acceptability curve and the expected value of perfect information. The study adopted both a health and societal perspective. Results From a health perspective, the deterministic analysis estimated an ICER for Oncotype of 17,453 euros/quality-adjusted life year (QALY), discount included, and 9,613 euros/QALY without the discount. Eighty five percent (85%) of the simulations were below the efficiency threshold for Spain. The parametric variability associated with the Oncotype results was the main uncertainty factor in the decision. Conclusions Oncotype is a cost-effective intervention from a health system perspective since each QALY gained costs less than 25,000 euros. From a societal perspective, it is dominant since it provides greater health and is accompanied by cost savings.

RESUMEN Objetivo Llevar a cabo un análisis de coste-utilidad del uso del test genómico Oncotype en el País Vasco (España) para informar la decisión de uso de quimioterapia. Método El estudio de coste-utilidad se realizó mediante un modelo de simulación de eventos discretos que representó la evolución natural del cáncer de mama. La decisión de tratamiento con quimioterapia basada en Oncotype se comparó con el estándar de tratamiento basado en criterios clínico-patológicos. El modelo incluyó datos clínicos de hospitales vascos y la literatura para calcular la ratio de coste-efectividad incremental (RCEI) mediante análisis determinista y probabilístico, el plano coste-efectividad, la curva de aceptabilidad y el valor esperado de la información perfecta. El estudio adoptó una perspectiva tanto sanitaria como social. Resultados El análisis determinista estimó una RCEI para Oncotype de 17.453 euros/año de vida ajustado por calidad (AVAC) con descuento y 9613 euros euros/AVAC sin descuento, desde la perspectiva sanitaria. El 85% de las simulaciones estuvieron por debajo el umbral de aceptabilidad para España. La variabilidad paramétrica asociada a los resultados de Oncotype fue el mayor factor de incertidumbre de la decisión. Conclusiones Oncotype constituye una intervención coste-efectiva, ya que cada AVAC ganado tiene asociado un coste inferior a 25.000 euros. El test es dominante desde una perspectiva social al lograr mayor salud acompañada de ahorros.

Resumen Objetivo Llevar a cabo un análisis de coste-utilidad del uso del test genómico Oncotype en el País Vasco (España) para informar la decisión de uso de quimioterapia. Método El estudio de coste-utilidad se realizó mediante un modelo de simulación de eventos discretos que representó la evolución natural del cáncer de mama. La decisión de tratamiento con quimioterapia basada en Oncotype se comparó con el estándar de tratamiento basado en criterios clínico-patológicos. El modelo incluyó datos clínicos de hospitales vascos y la literatura para calcular la ratio de coste-efectividad incremental (RCEI) mediante análisis determinista y probabilístico, el plano coste-efectividad, la curva de aceptabilidad y el valor esperado de la información perfecta. El estudio adoptó una perspectiva tanto sanitaria como social. Resultados El análisis determinista estimó una RCEI para Oncotype de 17.453 euros/año de vida ajustado por calidad (AVAC) con descuento y 9613 euros euros/AVAC sin descuento, desde la perspectiva sanitaria. El 85% de las simulaciones estuvieron por debajo el umbral de aceptabilidad para España. La variabilidad paramétrica asociada a los resultados de Oncotype fue el mayor factor de incertidumbre de la decisión. Conclusiones Oncotype constituye una intervención coste-efectiva, ya que cada AVAC ganado tiene asociado un coste inferior a 25.000 euros. El test es dominante desde una perspectiva social al lograr mayor salud acompañada de ahorros.

Abstract Objective To carry out a cost-utility analysis of the application of the Oncotype genomic test to inform the decision to use or not to use chemotherapy in the Basque Country (Spain). Method The cost-utility study was carried out using a discrete event simulation model representing the natural history of breast cancer. The decision of treatment with chemotherapy based on Oncotype was compared with the standard of treatment based on clinical-pathological criteria. The model included clinical data from Basque hospitals and the literature and was processed by deterministic and probabilistic analysis to calculate the incremental cost-effectiveness ratio (ICER), the cost-effectiveness plane, the acceptability curve and the expected value of perfect information. The study adopted both a health and societal perspective. Results From a health perspective, the deterministic analysis estimated an ICER for Oncotype of 17,453 euros/quality-adjusted life year (QALY), discount included, and 9,613 euros/QALY without the discount. Eighty five percent (85%) of the simulations were below the efficiency threshold for Spain. The parametric variability associated with the Oncotype results was the main uncertainty factor in the decision. Conclusions Oncotype is a cost-effective intervention from a health system perspective since each QALY gained costs less than 25,000 euros. From a societal perspective, it is dominant since it provides greater health and is accompanied by cost savings.