Análise de custo-utilidade do eculizumabe no tratamento da hemoglobinúria paroxística noturna
Análise de custo-utilidade do eculizumabe no tratamento da hemoglobinúria paroxística noturna
Arquivos
Data
2019
Autores
Cruz, Daniela Souza
Journal Title
Journal ISSN
Volume Title
Publisher
Instituto Nacional de Cardiologia
Resumo
INTRODUÇÃO: A Hemoglobinúria Paroxística Noturna (HPN) é uma doença rara caracterizada por anemia hemolítica crônica, acarretando em sintomas como fadiga, dispneia e hemoglobinúria. Outros aspectos clínicos associados são o risco aumentado de tromboembolismo e de comprometimento da medula óssea. O eculizumabe é eficaz na redução da hemólise intravascular, consistindo no único medicamento específico para a doença. Contudo, o alto preço da terapia é um fator limitante para o acesso ao tratamento, estimulando a judicialização do medicamento no Brasil, com impactos financeiros significativos para o Ministério da Saúde. Apesar disto, a estimativa da razão de custo-utilidade incremental é desconhecida. OBJETIVO: Realizar análise de custo-utilidade do eculizumabe para o tratamento da HPN, na perspectiva do Sistema Único de Saúde (SUS). MÉTODOS: Foi desenvolvido um modelo de decisão de Markov para a população acima de 35 anos, com HPN sintomática. Foram considerados ciclos mensais e um horizonte temporal de 20 anos. Considerou-se os anos de vida ajustados pela qualidade (QALY) como medida de efetividade. Dados foram obtidos a partir de ensaios clínicos, coortes históricas e bases assistenciais do SUS. O uso de recursos e os custos foram estimados pela perspectiva do SUS. Análises de sensibilidade determinística e probabilísticas foram efetuadas. RESULTADOS: O ganho estimado em efetividade com o uso do eculizumabe foi de 1,08 QALY mediante ao custo incremental de R$ 10.934.545,67. A razão de custoefetividade incremental foi de R$10.138.511,04 por QALY, sendo 110,6 vezes maior que a disposição a pagar de três vezes o Produto Interno Bruto per capita. Na análise de sensibilidade determinística, os parâmetros relacionados à utilidade dos estados de saúde foram associados com maior impacto no modelo. Os resultados da análise de sensibilidade probabilística evidenciam que 100% das simulações não seriam consideradas custo-efetivas com a disposição a pagar arbitrada em R$ 30.548,40 e R$ 91.645,20 por QALY. CONCLUSÕES: O ganho em efetividade com o uso do eculizumabe foi modesto, associado com um custo incremental expressivo. Assim, o medicamento eculizumabe não é custo-efetivo, quando comparado à terapia convencional, no tratamento da HPN sob a perspectiva do SUS. Regras mais específicas para análises econômicas de medicamentos para doenças raras precisam ser elaboradas.
INTRODUCTION: The paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by chronic hemolytic anemia, resulting in symptoms such as fatigue, dyspnea and hemoglobinuria. The risk of thromboembolism and bone marrow abnormality are other related clinical aspects. The eculizumab is effective in reducing intravascular hemolysis, being the only specific drug to the disease. Although, the high price of therapy is a limiting factor to access to the treatment, encouraging the judicialization of the drug in Brazil, causing significant financial impact to the Brazilian Ministry of Health. Despite this, the estimate of incremental cost-utility ratio is unknown. OBJECTIVE: To perform a cost-utility analysis of eculizumab for the treatment of PNH from the perspective of the Brazilian Unified Health System (SUS). METHODS: A Markov decision model was developed for patients over 35 years old with symptomatic PNH. Monthly cycles and a time horizon of 20 years were considered. Quality-adjusted life years (QALYs) were considered as measures of effectiveness. Data were extracted from clinical trials, historical cohorts and SUS databases. Resource use and costs were estimated from the perspective of SUS. Deterministic and probabilistic sensitivity analysis were performed. RESULTS: The estimated gain in effectiveness with the use of eculizumab was 1.08 QALYs through the incremental cost of R$ 10,934,545.67. The incremental costeffectiveness ratio was R$ 10,138,511.04 per QALY, being 110.6 times greater than the willingness to pay of three times the 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 evidence that 100% of the simulations were not considered cost-effective with the arbitrated willingness to pay of 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 expressive incremental cost. Therefore, eculizumab is not a costeffective drug compared with the current standard of care in the treatment of PNH from the perspective of the SUS. More specific rules for the economic evaluations of drugs for rare diseases need to be created.
INTRODUCTION: The paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by chronic hemolytic anemia, resulting in symptoms such as fatigue, dyspnea and hemoglobinuria. The risk of thromboembolism and bone marrow abnormality are other related clinical aspects. The eculizumab is effective in reducing intravascular hemolysis, being the only specific drug to the disease. Although, the high price of therapy is a limiting factor to access to the treatment, encouraging the judicialization of the drug in Brazil, causing significant financial impact to the Brazilian Ministry of Health. Despite this, the estimate of incremental cost-utility ratio is unknown. OBJECTIVE: To perform a cost-utility analysis of eculizumab for the treatment of PNH from the perspective of the Brazilian Unified Health System (SUS). METHODS: A Markov decision model was developed for patients over 35 years old with symptomatic PNH. Monthly cycles and a time horizon of 20 years were considered. Quality-adjusted life years (QALYs) were considered as measures of effectiveness. Data were extracted from clinical trials, historical cohorts and SUS databases. Resource use and costs were estimated from the perspective of SUS. Deterministic and probabilistic sensitivity analysis were performed. RESULTS: The estimated gain in effectiveness with the use of eculizumab was 1.08 QALYs through the incremental cost of R$ 10,934,545.67. The incremental costeffectiveness ratio was R$ 10,138,511.04 per QALY, being 110.6 times greater than the willingness to pay of three times the 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 evidence that 100% of the simulations were not considered cost-effective with the arbitrated willingness to pay of 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 expressive incremental cost. Therefore, eculizumab is not a costeffective drug compared with the current standard of care in the treatment of PNH from the perspective of the SUS. More specific rules for the economic evaluations of drugs for rare diseases need to be created.
Description
Palavras-chave
Economic evaluation, Cost Effectiveness Analysis, Cost-Utility Analyses, Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, Avaliação econômica, Análise de custo-efetividade, Análise de custo-utilidade, Hemoglobinúria Paroxística Noturna, Eculizumabe
Citação
Cruz DS. Análise de custo-utilidade do eculizumabe no tratamento da hemoglobinúria paroxística noturna. Rio de Janeiro. Dissertação [Mestrado Profissional em Avaliação de Tecnologia em Saúde] - Instituto Nacional de Cardiologia; 2019.