Health inequity assessment in Brazil: is EQ-5D-3L sensible enough to detect differences among distinct socioeconomic groups?

BACKGROUND: Multidimensional health-related quality of life (HRQOL) instruments, such as the EQ-5D, are increasingly used to assess inequalities in health. However, it is necessary to explore the ability of these instruments to capture differences between population groups, especially in low/middle-income countries. This study aimed to investigate whether the EQ-5D-3L instrument can detect differences in HRQOL between groups of different socioeconomic status (SES) in Brazil. METHODS: Data collection occurred during the Brazilian EQ-5D-3L valuation study and included respondents aged 18 to 64 years enrolled in urban areas. SES was aggregated into three categories: "higher" (A and B), "intermediate" (C) and "lower" (D and E). EQ-5D-3L index was calculated considering the Brazilian value set. A mixed-effects regression model was estimated with random effects on individuals and marginal effects on SES, sex, and educational attainment. Odds ratios for the chance of reporting problems for each EQ-5D dimension were estimated by logistic regression. RESULTS: A total of 9,148 respondents were included in the study. Mean age was 37.80 ± 13.13 years, 47.4% were men and the majority was ranked as classes B or C (38.4% and 50.7%, respectively). Participants in lower SES classes reported increasingly poorer health compared to individuals in higher classes. The mean EQ-5D-3L index decreased as SES deteriorates being significantly higher for classes A and B (0.874 ± 0.14) compared to class C (0.842 ± 0.15) and classes D and E (0.804 ± 0.17) (p < 0.001). The same was observed for the mean EQ-VAS scores (84.0 ± 13.8 in classes A and B, 81.0 ± 17 in class C and 78.3 ± 18.7 in class C [p < 0.001]). The multivariate analysis confirmed that SES is an independent factor that effects EQ-5D-3L index measures. Participants in intermediate and lower SES classes have a statistically significant lower EQ-5D-3L index compared to participants in classes A and B, regardless of age, sex, and educational attainment. CONCLUSION: In a Brazilian population sample, the EQ-5D-3L instrument was able to detect important differences between groups with distinct socioeconomic statuses (SES). The EQ-5D-3L is useful for exploring inequities in health.

Measurement properties of the EQ-5D in children and adolescents: a systematic review protocol.

BACKGROUND: Although the EQ-5D instruments have been initially designed for adult populations, there are new studies evaluating and applying these instruments to children and adolescents. The EuroQol Group adapted and created two versions designed for these groups, i.e., the EQ-5D-Y versions. The measurement properties of the EQ-5D have been systematically reviewed in different health conditions. However, there is a lack of a proper systematic assessment including the studies' risk of bias and focusing on recent studies assessing the EQ-5D instruments in children and adolescents. The lack of a systematic assessment of the EQ-5D versions does not allow us to have a comprehensive evaluation of the validity, reliability, and responsiveness of these instruments among children and adolescents. This systematic review aims to critically appraise and summarize the evidence on the measurement properties of the EQ-5D instruments (self-reported version - answered by children and adolescents; and proxy versions - versions reported by parents, caregivers, or health professionals) in children and adolescents. METHODS: A systematic review searching the following electronic databases: MEDLINE, EMBASE, CINAHL, EconLit, National Health Service Economic Evaluation Database (NHS-EED), Health Technology Assessment (HTA) database. Two independent reviewers will screen titles and abstracts and select full texts for eligibility. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology will be followed to conduct three main assessment steps: risk of bias, quality criteria for measurement properties, and evidence synthesis. DISCUSSION: This systematic review will provide comprehensive information about the evidence regarding the measurement properties of EQ-5D instruments in children and adolescents of different settings and countries. SYSTEMATIC REVIEW REGISTRATION: Open Science Framework with Registration https://osf.io/r8kt9/ and PROSPERO: CRD42020218382.

A cost-effectiveness analysis of a novel algorithm to sequentially diagnose leprosy based on manufactured tests under the SUS perspective.

Brazil has the second largest number of leprosy cases (a disease with a significant burden) in the world. Despite global and local efforts to eliminate this public health problem, inadequate or late diagnosis contribute to perpetuate its transmission, especially among household contacts. Tests such as the rapid IgM antibody detection (RT) and real-time polymerase chain reaction (RT-PCR) were developed to overcome the challenges of early diagnosis of leprosy. This study aimed to analyze the cost-effectiveness of a new diagnostic algorithm recommended by the Brazilian government to diagnose leprosy in household contacts of confirmed leprosy cases, which includes the RT and RT-PCR tests. A decision tree model was constructed and the perspective of the Brazilian Unified National Health System (SUS) and a 1-year time horizon were adopted. Only direct medical costs related to diagnostic tests were included. Effectiveness was measured as the number of avoided undiagnosed leprosy cases. Different scenarios were analyzed. The sequential use of RT, slit-skin smear (SSS) microscopy, and RT-PCR as recommended by the Brazilian Ministry of Health was compared to a base case (isolated SSS microscopy), yielding an incremental cost-effectiveness ratio of USD 616.46 per avoided undiagnosed leprosy case. Univariate sensitivity analysis showed that the prevalence of leprosy among household contacts was the variable that influenced the model the most. This is the first economic model to analyze a diagnostic algorithm of leprosy. Results may aid managers to define policies and strategies to eradicate leprosy in Brazil.

Cost-Utility Model of Nirmatrelvir/Ritonavir in Brazil: Analysis of a Vaccinated Population.

OBJECTIVES: The aim of this study is to conduct a cost-utility analysis of the use of the antiviral nirmatrelvir/ritonavir, applied to a vaccinated Brazilian population against COVID-19, from the perspective of the Brazilian Public Health System (SUS). METHODS: A microsimulation model was created with individual-level data and daily cycles, with a 1-year time horizon, to compare the current scenario of standard care with a scenario in which nirmatrelvir/ritonavir is offered to the population. Adults of any age group that received ≥2 doses of the COVID-19 vaccine formed the investigated population. Direct medical costs of the outpatients and inpatients admitted to the ward or intensive care unit were included. The effectiveness of the model was measured in quality-adjusted life-years (QALYs). RESULTS: In all simulations, the use of nirmatrelvir/ritonavir resulted in incremental costs per patient of US dollar (USD)245.86 and incremental effectiveness of 0.009 QALY, over a year. The incremental cost-utility ratio was USD27 220.70/QALY. The relative risk of the vaccinated population was the factor that affected the outcome most, according to the univariate sensitivity analysis. The probabilistic sensitivity analysis resulted in 100% of the simulations being more costly and effective, but that only 4% of them were below the established cost-effectiveness threshold of USD24 000.00/QALY. In the scenario considering only the population over 60 years old and immunosuppressed (of any age), the incremental cost-utility ratio was USD7589.37/QALY. CONCLUSIONS: The use of nirmatrelvir/ritonavir in the treatment of COVID-19 in a vaccinated population was cost-effective only for immunosuppressed individuals and people over 60 years of age.

A cost-effectiveness analysis of a novel algorithm to sequentially diagnose leprosy based on manufactured tests under the SUS perspective / Análise de custo-efetividade de um novo algoritmo para exame sequencial de hanseníase baseado em testes fabricados sob a perspectiva do SUS / Análisis de costo-efectividad de un nuevo algoritmo para el examen secuencial de lepra basado en pruebas fabricadas bajo la perspectiva del SUS

Abstract: Brazil has the second largest number of leprosy cases (a disease with a significant burden) in the world. Despite global and local efforts to eliminate this public health problem, inadequate or late diagnosis contribute to perpetuate its transmission, especially among household contacts. Tests such as the rapid IgM antibody detection (RT) and real-time polymerase chain reaction (RT-PCR) were developed to overcome the challenges of early diagnosis of leprosy. This study aimed to analyze the cost-effectiveness of a new diagnostic algorithm recommended by the Brazilian government to diagnose leprosy in household contacts of confirmed leprosy cases, which includes the RT and RT-PCR tests. A decision tree model was constructed and the perspective of the Brazilian Unified National Health System (SUS) and a 1-year time horizon were adopted. Only direct medical costs related to diagnostic tests were included. Effectiveness was measured as the number of avoided undiagnosed leprosy cases. Different scenarios were analyzed. The sequential use of RT, slit-skin smear (SSS) microscopy, and RT-PCR as recommended by the Brazilian Ministry of Health was compared to a base case (isolated SSS microscopy), yielding an incremental cost-effectiveness ratio of USD 616.46 per avoided undiagnosed leprosy case. Univariate sensitivity analysis showed that the prevalence of leprosy among household contacts was the variable that influenced the model the most. This is the first economic model to analyze a diagnostic algorithm of leprosy. Results may aid managers to define policies and strategies to eradicate leprosy in Brazil.

Resumo: O Brasil tem o segundo maior número de casos de hanseníase (doença com carga significativa) do mundo. Apesar dos esforços globais e locais para eliminar esse problema de saúde pública, o diagnóstico inadequado ou tardio contribui para perpetuar sua transmissão, especialmente entre contatos intradomiciliares. Exames como o teste rápido de anticorpos IgM (RT) e a reação em cadeia da polimerase em tempo real (RT-PCR) foram desenvolvidos para superar as barreiras do diagnóstico precoce da hanseníase. Este estudo teve como objetivo analisar a relação custo-efetividade de um novo algoritmo de diagnóstico recomendado pelo governo brasileiro para diagnosticar a hanseníase em contatos domiciliares de casos confirmados de hanseníase, que inclui os testes RT e RT-PCR. Foi construído um modelo de árvore de decisão e adotada a perspectiva do Sistema Único de Saúde (SUS) considerando o período de um ano. Foram incluídos apenas os custos médicos diretos relacionados aos exames diagnósticos. A efetividade foi medida considerando o número de casos evitados de hanseníase. Diferentes cenários foram analisados. O uso sequencial de RT, baciloscopia e RT-PCR, conforme recomendado pelo Ministério da Saúde, foi comparado a um caso base (baciloscopia isolada), obtendo-se uma razão de custo-efetividade incremental de USD 616,46 por caso evitado de hanseníase. A análise de sensibilidade univariada mostrou que a prevalência de hanseníase entre contatos intradomiciliares foi a variável que mais influenciou o modelo. Este é o primeiro modelo econômico a analisar um algoritmo diagnóstico da hanseníase. Os resultados poderão auxiliar os gestores na definição de políticas e estratégias para a erradicação da hanseníase no Brasil.

Resumen: Brasil tiene el segundo mayor número de casos de lepra (enfermedad con carga significativa) del mundo. A pesar de los esfuerzos globales y locales para eliminar ese problema de salud pública, el diagnóstico inadecuado o tardío contribuye a perpetuar su transmisión, sobre todo entre contactos intradomiciliarios. Los exámenes como la prueba rápida de anticuerpos IgM (RT) y la reacción en cadena de la polimerasa en tiempo real (RT-PCR) se desarrollaron para superar las barreras del diagnóstico precoz de la lepra. El objetivo de este estudio fue analizar la relación de costo-efectividad de un nuevo algoritmo de diagnóstico recomendado por el gobierno brasileño para diagnosticar la lepra en contactos domiciliarios de casos confirmados de lepra, que incluye las pruebas RT y RT-PCR. Se construyó un modelo de árbol de decisión y se adoptó la perspectiva del Sistema Único de Salud (SUS) teniendo en cuenta el periodo de un año. Solo se incluyeron los costos médicos directos relacionados con los exámenes diagnósticos. Se midió la efectividad teniendo en cuenta el número de casos de lepra evitados. Se analizaron distintos escenarios. Se comparó el uso secuencial de RT, baciloscopia y RT-PCR, conforme el Ministerio de Salud recomienda, con un caso base (baciloscopia aislada), y se obtuvo un cociente de costo-efectividad incremental de USD 616,46 por cada caso de lepra evitado. El análisis de sensibilidad univariante mostró que la prevalencia de lepra entre contactos intradomiciliarios fue la variable que más influyó el modelo. Este es el primer modelo económico que analiza un algoritmo diagnóstico de lepra. Los resultados podrán ayudar los gestores a definir políticas y estrategias para erradicar la lepra en Brasil.

Clinical, Economic, and Humanistic Impact of Short-Bowel Syndrome/Chronic Intestinal Failure in Portugal (PARENTERAL Study).

Introduction: This study aimed to assess the clinical, economic, and humanistic impact of short-bowel syndrome/chronic intestinal failure (SBS/CIF) in Portugal. Methods: This is a retrospective multicenter cohort chart review study, with a cross-sectional component for quality-of-life (QoL) evaluation. Inclusion criteria comprised patients with SBS/CIF, aged ≥1 year, with stable parenteral nutrition (PN). Data collection included patient chart review over a 12-month period and patient/caregiver self-report and SF-36/PedsQL™ questionnaires. Main endpoints comprised clinical and PN characterization, healthcare resource use (HRU), direct costs, and patient QoL. Results: Thirty-one patients were included (11 adults and 20 children). Patients' mean age (standard deviation [SD]) was 57.9 (14.3) years in adults and 7.5 (5.0) years in children, with a mean time since diagnosis of 10.2 (5.9) and 6.6 (4.2) years, respectively. PN was administered for a mean of 5.2 and 6.6 days/week in adults and children, respectively; home PN occurred in 81.8% of adults and 90.0% of children for a mean of 9.6 and 10.8 months/year, respectively. The mean annual number of hospitalizations was 1.9 and 2.0 which lasted for a mean of 34.0 and 29.4 days in adults and children, respectively. Twenty-one and forty hospitalization episodes were reported in adults and children, respectively, of which 71.4% and 85.0% were due to catheter-related complications. Mean annual direct costs per patient amounted to 47,857.53 EUR in adults and 74,734.50 EUR in children, with PN and hospitalizations as the main cost-drivers. QoL assessment showed a clinically significant impaired physical component in adults and a notable deterioration in the school functioning domain in children. Conclusion: In Portugal, SBS/CIF patient management is characterized by a substantial therapeutic burden and HRU, translating into high direct costs and a substantial impairment of the adults' physical function and children's school functioning.

Introdução: Este estudo teve como objetivo avaliar o impacto clínico, económico e social da síndrome do intestino curto/falência intestinal crónica (SIC/FIC) em Portugal. Métodos: Estudo de coorte retrospectivo e multicêntrico de revisão dos processos clínicos incluindo uma componente transversal para avaliação da qualidade de vida (QV). Os critérios de elegibilidade incluíram doentes com SIC/FIC, idade ≥1 ano, em nutrição parenteral (NP) e clinicamente estáveis. A recolha de dados incluiu a revisão dos processos clínicos ao longo de um período de 12 meses e a aplicação de questionários auto-administrados a doentes e cuidadores e de questionários de QV (SF-36/PedsQL™). Os indicadores principais foram a caracterização clínica e da NP, a utilização de recursos de saúde, custos diretos e QV dos doentes. Resultados: Foram incluídos 31 doentes (11 adultos e 20 crianças). A idade média (desvio padrão: DP) foi de 57.9 (14.3) anos nos adultos e de 7.5 (5.0) nas crianças com um tempo médio desde o diagnóstico de 10.2 (5.9) e 6.6 (4.2) anos, respetivamente. A NP foi administrada durante uma média de 5.2 e 6.6 dias por semana, em adultos e crianças respetivamente, em 81.8% e 90.0% dos adultos/crianças foi feita em casa durante uma média de 9.6 ou 10.8 meses por ano, respetivamente. O número médio anual de hospitalizações foi de 1.9 (1.6) e 2.0 (1.5) com uma duração média de 34.0 (47.4) e 29.4 (32.3) dias, em adultos e crianças, respetivamente. Foram reportados 21 e 40 episódios de hospitalização em adultos/crianças, dos quais 71.4% e 85.0% foram devido a complicações relacionadas ao uso de cateter. Os custos diretos anuais médios por doente ascenderam a 47,857.53 EUR nos adultos e a 74,734.50 EUR nas crianças, sendo que os maiores responsáveis foram a NP e as hospitalizações. A avaliação da QV mostrou um comprometimento clinicamente significativo da componente física nos adultos e uma deterioração relevante da dimensão escolar nas crianças. Conclusões: A gestão dos doentes com SIC/FIC em Portugal é caracterizada por uma sobrecarga substancial a nível terapêutico e de utilização de recursos de saúde, o que se traduz em elevados custos diretos e comprometimento substancial da componente física nos adultos e do desempenho escolar nas crianças.

Cost-effectiveness analysis of extended thromboprophylaxis with rivaroxaban versus no prophylaxis in high-risk patients after hospitalisation for COVID-19: an economic modelling study.

Background: In patients at high risk of thromboembolism who were discharged after hospitalisation due to COVID-19, thromboprophylaxis with rivaroxaban 10 mg/day for 35 days significantly improved clinical outcomes, reducing thrombotic events compared with no post-discharge anticoagulation. The present study aimed to estimate the cost-effectiveness of this anticoagulation strategy. Methods: Using the database of the MICHELLE trial, we developed a decision tree to estimate the cost-effectiveness of thromboprophylaxis with rivaroxaban 10 mg/day for 35 days versus no thromboprophylaxis in high-risk post-discharge patients for COVID-19 through an incremental cost-effectiveness analysis. Findings: 318 patients in 14 centres in Brazil were enrolled in the primary MICHELLE trial. The mean age was 57.1 years (SD 15.2), 127 (40%) were women, 191 (60%) were men, and the mean body-mass index was 29.7 kg/m2 (SD 5.6). Rivaroxaban 10 mg per day orally for 35 days after discharge decreased the risk of events defined by the primary efficacy outcome by 67% (relative risk 0.33, 95% CI 0.12-0.90; p = 0.03). The mean cost for thromboprophylaxis with rivaroxaban was $53.37/patient, and no prophylaxis was $34.22/patient, with an incremental cost difference of $19.15. The effectiveness means obtained in the intervention group was 0.1457, while in the control group was 0.1421, determining an incremental QALY difference of 0.0036. The estimated incremental cost-effectiveness ratio (ICER) was $5385.52/QALY. Interpretation: Extended treatment with Rivaroxaban as thromboprophylaxis after hospital discharge for high-risk patients with COVID-19 is a cost-effective treatment option. Funding: Modest funding was provided by Science Valley Research Institute, São Paulo, Brazil.

Economic analysis of antenatal screening for human T-cell lymphotropic virus type 1 in Brazil: an open access cost-utility model.

BACKGROUND: Human T-cell lymphotropic virus type 1 (HTLV-1) is a retrovirus that causes severe diseases, such as aggressive cancer or progressive neurological disease. HTLV-1 affects mainly people in areas with low human development index and can be transmitted from mother to child, primarily through breastfeeding. Refraining from breastfeeding is an effective intervention to reduce the risk of infection in infants. However, HTLV-1 antenatal screening is not offered globally. According to WHO, the scarcity of cost-effectiveness studies is considered one of the major barriers to the implementation of policies to prevent HTLV-1 infection. Therefore, this study aimed to assess the cost-effectiveness of antenatal screening and postnatal interventions to prevent HTLV-1 mother-to-child transmission in Brazil and to develop an open-access, editable, mathematical model that can be used by other countries and regions to assess different scenarios. METHODS: In this cost-utility analysis, we constructed a decision tree and a Markov model to assess the cost-effectiveness of HTLV-1 antenatal screening and postnatal interventions (ie, avoidance of breastfeeding, by suppression of lactation with cabergoline, and provision of formula feed) to reduce transmission. For our model, we used data from Brazil and we took the perspective of the public health-care system to estimate costs. FINDINGS: The implementation of both screening and interventions would result in the prevention of 1039 infections in infants every year in Brazil with an incremental cost-effectiveness ratio (ICER) of US$11 415 per quality-adjusted life-year (QALY). 88% of all probabilistic sensitivity analysis simulations had ICER values lower than the Brazilian cost-effectiveness threshold ($18 107·74 per QALY). HTLV-1 prevalence in pregnant women, the risk of HTLV-1 transmission when breastfeeding lasts for 6 months or more, and the cost of screening tests were the variables with the largest effect on ICER. INTERPRETATION: HTLV-1 antenatal screening is cost-effective in Brazil. An open-access model was developed, and this tool could be used to assess the cost-effectiveness of such policy globally, favouring the implementation of interventions to prevent HTLV-1 mother-to-child transmission worldwide. FUNDING: None. TRANSLATIONS: For the Portuguese and Spanish translations of the abstract see Supplementary Materials section.

Economic analysis of antenatal screening for human T-cell lymphotropic virus type 1 in Brazil: an open access cost-utility model

Background Human T-cell lymphotropic virus type 1 (HTLV-1) is a retrovirus that causes severe diseases, such as aggressive cancer or progressive neurological disease. HTLV-1 affects mainly people in areas with low human development index and can be transmitted from mother to child, primarily through breastfeeding. Refraining from breastfeeding is an effective intervention to reduce the risk of infection in infants. However, HTLV-1 antenatal screening is not offered globally. According to WHO, the scarcity of cost-effectiveness studies is considered one of the major barriers to the implementation of policies to prevent HTLV-1 infection. Therefore, this study aimed to assess the cost-effectiveness of antenatal screening and postnatal interventions to prevent HTLV-1 mother-to-child transmission in Brazil and to develop an open-access, editable, mathematical model that can be used by other countries and regions to assess different scenarios. Methods In this cost-utility analysis, we constructed a decision tree and a Markov model to assess the cost-effectiveness of HTLV-1 antenatal screening and postnatal interventions (ie, avoidance of breastfeeding, by suppression of lactation with cabergoline, and provision of formula feed) to reduce transmission. For our model, we used data from Brazil and we took the perspective of the public health-care system to estimate costs. Findings The implementation of both screening and interventions would result in the prevention of 1039 infections in infants every year in Brazil with an incremental cost-effectiveness ratio (ICER) of US$11415 per quality-adjusted lifeyear (QALY). 88% of all probabilistic sensitivity analysis simulations had ICER values lower than the Brazilian costeffectiveness threshold ($18 107·74 per QALY). HTLV-1 prevalence in pregnant women, the risk of HTLV-1 transmission when breastfeeding lasts for 6 months or more, and the cost of screening tests were the variables with the largest effect on ICER. Interpretation HTLV-1 antenatal screening is cost-effective in Brazil. An open-access model was developed, and this tool could be used to assess the cost-effectiveness of such policy globally, favouring the implementation of interventions to prevent HTLV-1 mother-to-child transmission worldwide. (AU)

Guidelines for Utility Measurement for Economic Analysis: The Brazilian Policy.

INTRODUCTION: Health-related quality of life is expressed in utilities, also referred to as utility estimates or parameters. Considerations about the source and type of utility values are especially important in a modeling context, where the lack of transparency, including the lack of a hierarchy for utility data sources, is a major issue to any estimation and can potentially compromise model reliability. OBJECTIVES: This document aims to present the first version of the Brazilian guidelines for utility measurement to support economic analysis. METHODS: A virtual workshop and a modified Delphi panel with 10 health technology specialists followed a rapid evaluation of 110 technical documents and indexed publications. The recommendations are based on the proposition that has received the most votes, although contentious issues are addressed in the suggestion or discussion. The rationale for the final decision is included in the text. RESULTS: The consensus includes 50 recommendations with the following topics: Transparency and Reliability, Model Design, Conditions Under Which Generic Questionnaires Are Not Sensible or Valid, Utility Evidence Hierarchy, Utility Data Searching, Modeling Utility Values, Extrapolating Quality Adjusted Life-Years for Models With Lifetime Horizons, Caregiver Utility, Utility Data Synthesis, Quality/Certainty of the Evidence, and Utility Estimates in End-of-Life Conditions. CONCLUSIONS: The goal of this project is to create unified national standards for using utility metrics in economic analysis in Brazil. This set of recommendations is not obligatory, but it is meant to serve as a guide and lead to the development of better and more transparent economic models in the country.

Cost-Utility of the CDK 4/6 Inhibitors for Postmenopausal Women With Luminal Advanced Breast Cancer in Brazil.

OBJECTIVES: Several trials have demonstrated the benefit of the CDK 4/6 inhibitors for postmenopausal women with luminal advanced breast cancer. This research aims to compare the cost-utility of the CDK 4/6 inhibitors in patients with no history of resistance to endocrine therapy. METHODS: A Markov model was constructed to estimate the incremental cost per quality-adjusted life-years (QALYs) of treatments from the Brazilian public health system perspective over a lifetime horizon (30 years) with 5% annual discount rate for both benefits and costs. Efficacy parameters were extracted from the pivotal studies. Costs were based on open data from the Brazilian Ministry of Health. The utilities were calculated according to the overall population preferences from a British study. Deterministic and probabilistic sensitivity analyses evaluated the robustness of the results. RESULTS: The most cost-effective drug was ribociclib (US$50 748/QALY), followed by abemaciclib (US$64 052/QALY) and palbociclib (US$65 289/QALY). The univariate analysis showed that the incremental cost-utility ratio (ICUR) was mainly sensitive to the overall survival hazard ratio. The one thousand-probabilistic simulation showed that all ICUR values were above classical thresholds such as 1 to 3 gross domestic product (GDP) per capita per QALY. CONCLUSIONS: Even though there is no established willingness to pay threshold in Brazil, the estimated ICUR for CDK 4/6 inhibitors is >6 times the Brazilian GDP per capita (GDP per capita = US$5694.73), which might be a barrier to their inclusion in the Brazilian public health system.

Cost Utility of Prasugrel in Postangioplasty Diabetic Patients.

OBJECTIVES: To prevent thrombotic events after angioplasty, current guidelines recommend dual antiplatelet therapy with aspirin and thienopyridine. Clopidogrel is the only thienopyridine currently available in the Brazilian National Health System. The purpose of this study was to determine the cost-effectiveness of prasugrel, an alternative thienopyridine, compared with clopidogrel in patients with acute coronary syndrome and diabetes mellitus who underwent angioplasty. METHODS: A state-transition Markov model was created to simulate the progression of diabetic patients after angioplasty. The model had a lifetime horizon and discounted outcomes at a 5% annual rate. The risks of myocardial infarction and death were calculated using data from the diabetes subgroup, and the risks of bleeding were calculated using data from the overall group from the Trial to Assess Improvement in Therapeutic Outcomes by Optimizing Platelet Inhibition With Prasugrel Thrombolysis in Myocardial Infarction 38 trial. Direct costs were estimated using official Brazilian open data. Quality of life values were obtained through literature search. Univariate and multivariate sensitivity analyses were performed. RESULTS: Prasugrel was associated with more quality-adjusted life-years (QALYs) (5.03 vs 4.94) and higher costs (US$975.11 vs US$575.97), resulting in an incremental cost-utility ratio (ICUR) of US$4303.86/QALY. In one-way sensitivity analysis, the costs of prasugrel had the greatest impact on ICUR, followed by the initial age entering the cohort. In the probabilistic sensitivity analysis, all ICUR values simulations were less than one Brazilian gross domestic product per capita/QALY (US $5802.86). CONCLUSIONS: Given the appealing economic profile, the clinical debate between reducing the risk of myocardial infarction and increasing the risk of bleeding may overcome economic concerns in the Brazilian National Health System.

Cost Utility of Vaccination Against COVID-19 in Brazil.

OBJECTIVES: The objective of this study was to evaluate the cost-utility of the Oxford, CoronaVac, and Janssen COVID-19 vaccines from the perspective of the Brazilian public health system. METHODS: A total of 3 microsimulation models were constructed with individual data to evaluate the 3 vaccines. The simulation contains 7 transition states that are related to the natural history of the disease. The model with a daily cycle has a time horizon of 1 year and uses data from 289 days of the pandemic. The analysis was conducted from the perspective of the Brazilian public health system considering direct medical costs. For the model inputs, outpatient and hospital databases were used with information on treated patients stratified by age. Information on mortality was also stratified based on patients' age in the mortality database (SIM). The efficacy of vaccines to reduce the likelihood of patients becoming ill was evaluated independently for each vaccine. Information on the quality of life of patients in outpatient or hospital treatment and the sequelae resulting from the disease were extracted from the literature. The main outcome of the analysis was quality-adjusted life-years (QALYs). RESULTS: The vaccines showed incremental cost-utility ratios ranging from R$-23 161.3/QALY (Oxford) to R$17 757.85/QALY (CoronaVac). The older the population, the lower was the incremental cost-utility ratio. Given a willingness-to-pay threshold of R$17 586/QALY, all the vaccines were considered cost-effective in the probabilistic sensitivity analysis. CONCLUSIONS: The results of the analysis by age group can help in the preparation of a vaccination prioritization plan.

Cost-Utility Analysis of Eculizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria From the Perspective of the Brazilian Public Health System.

OBJECTIVE: To perform a first cost-utility analysis of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria from the perspective of the Brazilian Unified Health System. METHODS: A Markov decision model was developed for 35-year-old patients with symptomatic paroxysmal nocturnal hemoglobinuria. We used a cycle length of one month and a time horizon of 20 years. The effectiveness measure was the quality-adjusted life year (QALY). Data were extracted from clinical trials, historical cohorts, and Unified Health System databases. Resource use and costs were estimated from the perspective of the Unified Health System. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: The estimated gain in effectiveness with the use of eculizumab was 1.08 QALY through the incremental cost of R$10,959,375.95. The incremental cost-effectiveness ratio was R$10,139,542.84 per QALY, being 331.92 times greater than the Brazilian gross domestic product per capita. In the deterministic sensitivity analysis, the parameters related to the utilities of health states were associated with greater impact in the model. The results of the probabilistic sensitivity analysis with 1000 simulations evidence that 100% of the simulations were not considered cost-effective with the arbitrated willingness to pay R$30,548.40 and R$91,645.20 per QALY. CONCLUSIONS: The gain in effectiveness with the use of eculizumab was modest, associated with an unjustifiable incremental cost. Therefore, eculizumab is not a cost-effective drug compared with the current standard of care in the treatment of paroxysmal nocturnal hemoglobinuria from the Brazilian Unified Health System perspective.

EQ-5D Brazilian population norms.

BACKGROUND: The EQ-5D-3L is a widely used generic health-related quality of life measure commonly applied to describe health outcomes and to measure disease burden. The aim of this study was to generate Brazilian population norms, stratified by age and gender, based on Brazilian preference weights for EQ-5D-3L. METHODS: A multicenter cross-sectional study was conducted in three Brazilian urban areas. The final sample consisted of 5774 respondents, aged from 18 to 64 years. Amongst other information, respondents were asked to self-report their health status using the EQ-5D-3L descriptive system and visual analog scale (EQ-VAS). Data on socio-demographic characteristics was obtained through specific questionnaires. The Brazilian TTO scoring algorithm was used to derive the utility values. Multivariate logistic regression models were fitted to analyze the influence of age, sex, education status and sample site on the presence of any problem for each dimension of EQ5D. RESULTS: Mean values were computed for both weighted index scores and self-rated health status (EQ-VAS), and stratified by gender and age groups. Health status declines with age, ranging between 0.87 for the youngest group 18-29 year-olds and 0.76 for 60-64-year-old. Men reported higher scores (0.85) than the woman (0.79). Lower education levels were associated with lower EQ-5D index score in most age groups. CONCLUSION: This study provides EQ-5D reference values for the Brazilian population. These values can be used by local decision-makers and researchers in economic evaluations and population health studies.

Application of the MCDA method for assessing new technologies for familial hypercholesterolaemia treatment / Aplicação do método MCDA para avaliar novas tecnologias para o tratamento da hipercolesterolemia familiar

Objective: Familial hypercholesterolaemia is a hereditary disease characterized by very high levels of low-density lipoprotein cholesterol and an elevated risk of early-onset cardiovascular disorders. New drugs provide alternatives for the treatment of patients with homozygous familial hypercholesterolaemia. The study aims to explore a practical application of multiple-criteria decision analysis on prioritization of new and emerging technologies for familial hypercholesterolaemia. Methods: The decision model was constructed using the MACBETH method. There were three stages: structuring the problem, measuring the performance of alternatives, and building the model. The weights for alternatives and levels were obtained by indirect comparisons, which evaluated the attractiveness of the performance levels of the criteria using the swing weights technique. Results: The drugs lomitapide, ezetimibe, evolocumab, and mipomersen were selected as alternatives for decision-making. "Cardiovascular Death", "Stroke" and "Acute Myocardial Infarction" had the three most significant weights. The criteria with the lowest weights were "Comfort" and "LDL-C Reduction". The top-ranked technology was evolocumab, with an overall score of 59.87, followed by ezetimibe, with a score of 37.21. Conclusion: How to apply the result of a higher score in the actual decisionmaking process still requires further studies. The case in question showed that evolocumab has more performance benefits than other drugs but with a cost approximately 50 times higher

Objetivo: A hipercolesterolemia familiar é uma doença hereditária caracterizada por níveis muito elevados de lipoproteína de baixa densidade (LDL-colesterol) e um risco elevado de doenças cardiovasculares de início precoce. Novos medicamentos oferecem alternativas para o tratamento de pacientes com hipercolesterolemia familiar homozigótica. Esse estudo tem como objetivo explorar uma aplicação prática da análise de decisão multicritério na priorização de tecnologias novas e emergentes para hipercolesterolemia familiar. Métodos: O modelo de decisão foi construído usando o método MACBETH. Três etapas foram criadas: estruturação do problema, mensuração do desempenho das alternativas e construção do modelo. Os pesos para alternativas e níveis foram obtidos por comparações indiretas, que avaliaram a atratividade dos níveis de desempenho dos critérios usando a técnica de pesos de balanço. Resultados: Os medicamentos lomitapida, ezetimiba, evolocumabe e mipomersen foram selecionados como alternativas para a tomada de decisão. "Morte Cardiovascular", "Acidente vascular cerebral" e "Infarto Agudo do Miocárdio" tiveram os três pesos mais significativos. Os critérios com os menores pesos foram "Conforto" e "Redução do LDL-C". A tecnologia mais bem avaliada foi o evolocumabe, com pontuação geral de 59,87, seguido da ezetimiba, com pontuação de 37,21. Conclusão: Ainda são necessários estudos para determinar como aplicar o resultado de uma pontuação mais alta no processo de tomada de decisão. O caso em questão demonstrou que o evolocumabe tem benefícios mais significativos em relação aos outros medicamentos, mas com um custo cerca de 50 vezes maior

Structuring of a proposal for the nursing process theoretical and methodological basis: professors' motivations / Estructuración de una propuesta para las bases teóricas y metodológicas del proceso de enfermería: motivaciones docentes / Estruturação de uma proposta para o embasamento teórico metodológico do processo de enfermagem: motivações docentes

ABSTRACT Objective: to describe the motivations that mobilize professors-nurses in the construction of a proposal for the theoretical and methodological basis for the Nursing Process teaching. Method: a Convergent Assistential research, conducted with professors-nurses. Six convergence groups were developed, with triggering questions on the theme. The reports were audio-recorded, validated by the participants and analyzed in the light of the Convergent Assistance Research. Results: there are three thematic approaches: professors' perception of the nurses' professional practices; dissatisfaction with the current health model; and dissatisfaction with the current teaching model. The thematic approaches reveal the motivation to overcome biomedical/cartesian practices in teaching and nursing care, which converges to the development of a proposal with a philosophical, theoretical and methodological framework for the Nursing Process teaching, aligned with the assumptions of the Unified Health System and consistent with health expanded clinic. Conclusion: this study allowed for the expression of concerns and discomforts with the current model of education and health, triggering reflections on the paradigm that guides the field of health teaching and dominates our life in society. The results reveal what is desired in the health scenario, as educating individuals and society, enhancing the perspective of social changes in the health sector, acclaimed and desired politically and socially, basing the development of philosophical, theoretical and methodological proposals for nursing care, consistent with society's current demands.

RESUMEN Objetivo: describir las motivaciones que impulsan a los enfermeros profesores a elaborar una propuesta para definir las bases teórica y metodológica para la enseñanza del Proceso de Enfermería. Método: investigación Convergente Asistencial, realizada con profesores enfermeros. Se desarrollaron seis grupos de convergencia con preguntas desencadenantes sobre la temática. Los relatos se grabaron en audio, fueron validados por los participantes y analizados bajo la óptica de la Investigación Convergente Asistencial. Resultados: surgen tres enfoques temáticos: prácticas profesionales de los enfermeros en la percepción de los profesores; la insatisfacción con el modelo de salud vigente; y la insatisfacción con el modelo de enseñanza vigente. Los enfoques temáticos revelan la motivación para superar prácticas biomédicas/cartesianas en la enseñanza y en la atención de enfermería, lo que converge para desarrollar una propuesta de referencial filosófico, teórico y metodológico para la enseñanza del Proceso de Enfermería, alineado con las presunciones del Sistema Único de Salud y en coincidencia con una clínica ampliada en salud. Conclusión: este estudio permitió expresar inquietudes y malestares con el modelo vigente de enseñanza y de salud, iniciando reflexiones sobre el paradigma que orienta al campo de la enseñanza en salud y rige nuestra vida en sociedad. Los resultados revelan lo que se desea en el ámbito de la salud, como individuos formadores y sociedad, potenciando la perspectiva de cambios sociales en el sector de la salud, aclamados y deseados tanto política como socialmente, y sirviendo como base para desarrollar propuestas filosóficas, teóricas y metodológicas para el cuidado de enfermería, en coincidencia con las demandas actuales de la sociedad.

RESUMO Objetivo: descrever as motivações que mobilizam enfermeiros professores na construção de uma proposta para o embasamento teórico e metodológico para o ensino do Processo de Enfermagem. Método: pesquisa Convergente Assistencial, com professores enfermeiros. Desenvolveram-se seis grupos de convergência com questões deflagradoras sobre a temática. Os relatos foram audiogravados, validados pelos participantes e analisados à luz da Pesquisa Convergente Assistencial. Resultados: surgem três enfoques temáticos: práticas profissionais dos enfermeiros na percepção dos professores; a insatisfação com o modelo de saúde vigente; e a insatisfação com o modelo de ensino vigente. Os enfoques temáticos revelam a motivação para superar práticas biomédicas/cartesianas no ensino e na assistência de enfermagem, o que converge para o desenvolvimento de uma proposta de referencial filosófico, teórico e metodológico para o ensino do Processo de Enfermagem, alinhado com os pressupostos do Sistema Único de Saúde e condizente com uma clínica ampliada em saúde. Conclusão: este estudo permitiu a expressão de inquietações e desconfortos com o modelo vigente de ensino e de saúde, disparando reflexões sobre o paradigma que orienta o campo do ensino na saúde e domina a nossa vida em sociedade. Os resultados revelam o que se almeja no cenário da saúde, como indivíduos formadores e sociedade, potencializando a perspectiva de mudanças sociais no setor da saúde, aclamadas e desejadas política e socialmente, alicerçando o desenvolvimento de propostas filosóficas, teóricas e metodológicas para o cuidado em enfermagem, condizentes com as demandas atuais da sociedade.

Multi-Criteria Model for Evaluating Drugs to Prevent Deep Venous Thrombosis Associated With Orthopedic Surgery: A Hospital-Based Case Study.

BACKGROUND: It's estimated that 40% to 60% of patients undergoing major orthopedic surgery of the hip or knee who do not receive thromboprophylaxis will develop deep venous thrombosis Instituto Nacional de Traumatologia e Ortopedia has established a guideline to prevent DVT with the administration of the Enoxaparin. Recently, institute stakeholders have been questioning this guideline as new oral anticoagulants that offer more comfort and efficacy, but present higher risk of bleeding, have been appearing in the market for treating deep venous thrombosis. OBJECTIVE: This study aims to validate the application of a multicriteria decision analysis in a real-world problem, the use of rivaroxaban and enoxaparin to prevent deep venous thrombosis. METHODS: The multicriteria method MACBETH (Measuring Attractiveness by a Categorical Based Evaluation Technique) was used in a decision conferencing process to develop an evaluation model for measuring the relative value of the drugs on each evaluation criterion, separately and globally. The model-building process was informed by a literature review and meta-analysis of randomized clinical trials with a critical appraisal of the evidence. RESULTS: We report a model-structure with eight criteria, each one associated with a weighting coefficient and value function. Following a simple additive aggregation process, the model-outputs showed that Rivaroxaban was considered a robust option for DVT. Sensitivity analysis and robustness analysis were performed and testify the consistency of the results. CONCLUSION: This article contributes to literature by showing how MACBETH method can be combined with scientific evidence and participatory group processes, for health technology assessment in hospitals.