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Introdução: A CONITEC é o órgão vinculado ao Ministério da Saúde (MS) responsável por avaliar a incorporação de novas tecnologias no Sistema Único de Saúde (SUS). O Brasil não possui um padrão pragmático no processo de tomada de decisões, avaliando individualmente cada uma das tecnologias submetidas no que tange critérios clínicos e econômicos, como custo-efetividade. Este tema ganhou destaque em 2021, onde foi pauta de uma oficina realizada pelo MS, o qual através de discussão com especialistas identificou que o valor de 1 PIB per capita seria adequado para critério de aceite de novos medicamentos. Ainda que o valor estabelecido tenha sido de 1 PIB per capita, casos especiais podem ser discutidos se o parâmetro for de até 3 PIB per capita. Objetivo: Avaliar o impacto econômico da utilização da diretriz de limiar de custo efetividade, atualmente em consulta pública, no SUS. Métodos: Estudo retrospectivo coletou dados da tomada de decisão da CONITEC, no período de 2012 a 2021, para medicamentos indicados para hematologia, imunologia, doenças raras e oncologia. Além da recomendação positiva ou não do órgão, informações econômicas foram coletadas, sendo elas a razão de custo-efetividade incremental (RCEI) e impacto orçamentário (IO). A partir deste levamento, foi realizada uma comparação entre as decisões efetivamente tomadas e as que teriam sido realizadas frente à diretriz de limiar de custo efetividade de 1 PIB per capita. Resultados: Ao longo do período analisado, 59 medicamentos foram avaliados. Destes, 43 foram submetidos para avaliação com valor menor que 1 PIB per capita, 14 medicamentos com valor entre 1 e 2 PIB per capita e 2 medicamentos com valor entre 2 e 3 PIB per capita. Dentre as 43 medicações do primeiro subgrupo, 40% foram aprovados, enquanto na faixa de 1 a 2 PIB per capita o índice de aprovação foi de 29% e para 2 a 3 PIB per capita foi de 50%. No geral, a CONITEC teve taxa de aceitação de 37%, o que representou gasto total de R$ 4.032.341.086,18 em 5 anos. Caso a nova diretriz de limiar de custo efetividade seja aprovada, é esperado que medicamentos com RCEI menor que 1 PIB per capita sejam incorporados. Dessa forma, a taxa de aceitação da CONITEC mudaria para 81%, gerando um impacto orçamentário incremental de R$ 2.722.461.560,91. O que corresponde a aumento de 67,5% nos gastos com medicamentos em até 5 anos. Conclusão: A adoção do limiar de custo-efetividade em 1 PIB per capita faria com que a taxa de aprovação da CONITEC aumentasse consideravelmente e, consequentemente, representasse um aumento no impacto orçamentário relevante. Isso acontece principalmente pelo fato da maioria dos medicamentos analisados estarem dentro do limiar de custo-efetividade proposto. O Brasil vêm atualizando os critérios para tomada de decisão em saúde, com a possibilidade de utilizar o limiar de custo efetividade associado a outros parâmetros relevantes no processo de avaliação de tecnologias em saúde. Introdução: A CONITEC é o órgão vinculado ao Ministério da Saúde (MS) responsável por avaliar a incorporação de novas tecnologias no Sistema Único de Saúde (SUS). O Brasil não possui um padrão pragmático no processo de tomada de decisões, avaliando individualmente cada uma das tecnologias submetidas no que tange critérios clínicos e econômicos, como custo-efetividade. Este tema ganhou destaque em 2021, onde foi pauta de uma oficina realizada pelo MS, o qual através de discussão com especialistas identificou que o valor de 1 PIB per capita seria adequado para critério de aceite de novos medicamentos. Ainda que o valor estabelecido tenha sido de 1 PIB per capita, casos especiais podem ser discutidos se o parâmetro for de até 3 PIB per capita. Objetivo: Avaliar o impacto econômico da utilização da diretriz de limiar de custo efetividade, atualmente em consulta pública, no SUS. Métodos: Estudo retrospectivo coletou dados da tomada de decisão da CONITEC, no período de 2012 a 2021, para medicamentos indicados para hematologia, imunologia, doenças raras e oncologia. Além da recomendação positiva ou não do órgão, informações econômicas foram coletadas, sendo elas a razão de custo-efetividade incremental (RCEI) e impacto orçamentário (IO). A partir deste levamento, foi realizada uma comparação entre as decisões efetivamente tomadas e as que teriam sido realizadas frente à diretriz de limiar de custo efetividade de 1 PIB per capita. Resultados: Ao longo do período analisado, 59 medicamentos foram avaliados. Destes, 43 foram submetidos para avaliação com valor menor que 1 PIB per capita, 14 medicamentos com valor entre 1 e 2 PIB per capita e 2 medicamentos com valor entre 2 e 3 PIB per capita. Dentre as 43 medicações do primeiro subgrupo, 40% foram aprovados, enquanto na faixa de 1 a 2 PIB per capita o índice de aprovação foi de 29% e para 2 a 3 PIB per capita foi de 50%. No geral, a CONITEC teve taxa de aceitação de 37%, o que representou gasto total de R$ 4.032.341.086,18 em 5 anos. Caso a nova diretriz de limiar de custo efetividade seja aprovada, é esperado que medicamentos com RCEI menor que 1 PIB per capita sejam incorporados. Dessa forma, a taxa de aceitação da CONITEC mudaria para 81%, gerando um impacto orçamentário incremental de R$ 2.722.461.560,91. O que corresponde a aumento de 67,5% nos gastos com medicamentos em até 5 anos. Conclusão: A adoção do limiar de custo-efetividade em 1 PIB per capita faria com que a taxa de aprovação da CONITEC aumentasse consideravelmente e, consequentemente, representasse um aumento no impacto orçamentário relevante. Isso acontece principalmente pelo fato da maioria dos medicamentos analisados estarem dentro do limiar de custo-efetividade proposto. O Brasil vêm atualizando os critérios para tomada de decisão em saúde, com a possibilidade de utilizar o limiar de custo efetividade associado a outros parâmetros relevantes no processo de avaliação de tecnologias em saúde.

The mainstays of cutaneous leishmaniasis (CL) treatment, in several world regions, are pentavalent antimony (Sbv) compounds administered parenterally, despite their recognized toxicity, which requires frequent laboratory monitoring and complicates their use in areas with scarce infrastructure. As result of these drawbacks, the WHO Expert Committee on leishmaniasis has expanded the recommendations for the use of local therapies, including Sbv intralesional infiltration (IL-Sbv), as CL therapy alternatives even in the New World. However, the efficacy of these approaches has never been compiled. The aim of this study was to critically and systematically assess the efficacy of IL-Sbv for CL treatment.The PRISMA guidelines for systematic reviews and the Cochrane manual were followed. The sources used were the MEDLINE and LILACS databases and the International Clinical Trials Registry Platform of the World Health Organization. The outcome of interest was a clinical cure, defined as complete re-epithelialization of all lesions. The IL-Sbv pooled cure rate was estimated for several subgroups and direct comparisons were performed when possible.Thirty nine articles (40 studies) involving 5679 patients treated with IL-Sbv infiltration were included. In direct comparison, only three studies involving 229 patients compared IL-Sbv infiltration versus placebo and no difference was observed (OR: 1,9; 95%IC 0,93 to 3,82) based on cure rate 69.6% (95%CI 17.6-96.1%) and 83,2% (95%CI 66-92.7%) for placebo and IL-Sbv, respectively. In an alternative and non-comparative analysis, gathering all study arms using the intervention, the pooled IL-Sbv efficacy rate was 75% (95%CI 68-81%). In the Old World, the observed overall IL-Sbv efficacy rate was 75% (95%CI 66-82%), and the cure rates were significantly higher with sodium stibogluconate (SSG) than with meglumine antimoniate (MA): 83% (95%CI 75-90%) versus 68% (95%CI 54-79%), p = 0.03. Studies directly comparing IL-Sbv with topical 15% paromomycin ointment, IL hypertonic saline, radiofrequency-induced heat therapy, topical trichloroacetic acid and cryotherapy showed no significant difference in efficacy between the interventions. The analyses suggested a higher efficacy of IL-Sbv combined with cryotherapy (81.8%, 95%IC 62.4-92.4%) when compared with IL-Sbv alone (53.3%, 95%IC 46.1-66%), OR: 3.14 (95%CI 1.1-8.9), p = 0.03. In the New World, the global IL-Sbv efficacy was 77%(95%CI 66-85%). In contrast with the Old World, a significant difference favoring MA in relation to SSG was observed: 61% (95%CI 49-73%) versus 82% (95%CI 70-89%).By comparing IL infiltration schedules, it was determined that patients submitted to IL-Sbv treatments longer than 14 days had higher cure rates.Despite the high heterogeneity and low methodological quality of studies, an indirect comparison shows that the antimony infiltration efficacy rate is similar to that reported for antimony systemic use. The evidence gathered thus far is insufficient to identify the ideal IL therapeutic regime or estimate the rates of adverse events and mucosal late complications.

Introduction: The National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. This study estimated the budget impact of the supply of methionine-free amino acid formula (MFAAf) for patients with classical homocystinuria (HCU) in the SUS. Methods: The incidence of one case per 250,000 live births in Brazil and the registration of a Brazilian association of patients with HCU was assumed to calculate the population. Mortality and responsiveness to pyridoxine rates were applied. The costs of treatment were estimated according to the recommended dosage in literature and public purchasing prices. For calculating the dose of MFAAf patients, a median age of 19 years and weight of 60 kg were assumed, according to Brazilian study data. Results: The annual cost of treatment was estimated at BRL 77,000 (USD 21,084) per patient. The incorporation of MFAAf for HCU would generate a budget impact in SUS of around BRL 37 million (USD 10.1 million) in 2019 and BRL 188 million (USD 51.5 million) after five years which considers the epidemiological data, and a budget impact of around BRL 6.4 million (USD 1.75 million) in 2019 and BRL 33 million (USD 9 million) after five years which considers the information of a Brazilian association of patients with HCU. The wide range of values in the incremental budgetary impact is due to the lack of information on the epidemiology of the disease in Brazil. Conclusions: The incorporation of the MFAAf in the SUS represents an important budgetary impact and covers a small number of patients. CONITEC recommended the incorporation of the MFAAf in the SUS, according to clinical protocol. [ABSTRACT FROM AUTHOR]

Background: The mainstays of cutaneous leishmaniasis (CL) treatment, in several world regions, are pentavalent antimony (Sbv) compounds administered parenterally, despite their recognized toxicity, which requires frequent laboratory monitoring and complicates their use in areas with scarce infrastructure. As result of these drawbacks, the WHO Expert Committee on leishmaniasis has expanded the recommendations for the use of local therapies, including Sbv intralesional infiltration (IL-Sbv), as CL therapy alternatives even in the New World. However, the efficacy of these approaches has never been compiled. The aim of this study was to critically and systematically assess the efficacy of IL-Sbv for CL treatment. Methodology: The PRISMA guidelines for systematic reviews and the Cochrane manual were followed. The sources used were the MEDLINE and LILACS databases and the International Clinical Trials Registry Platform of the World Health Organization. The outcome of interest was a clinical cure, defined as complete re-epithelialization of all lesions. The IL-Sbv pooled cure rate was estimated for several subgroups and direct comparisons were performed when possible. Results: Thirty nine articles (40 studies) involving 5679 patients treated with IL-Sbv infiltration were included. In direct comparison, only three studies involving 229 patients compared IL-Sbv infiltration versus placebo and no difference was observed (OR: 1,9; 95%IC 0,93 to 3,82) based on cure rate 69.6% (95%CI 17.6–96.1%) and 83,2% (95%CI 66–92.7%) for placebo and IL-Sbv, respectively. In an alternative and non-comparative analysis, gathering all study arms using the intervention, the pooled IL-Sbv efficacy rate was 75% (95%CI 68–81%). In the Old World, the observed overall IL-Sbv efficacy rate was 75% (95%CI 66–82%), and the cure rates were significantly higher with sodium stibogluconate (SSG) than with meglumine antimoniate (MA): 83% (95%CI 75–90%) versus 68% (95%CI 54–79%), p = 0.03. Studies directly comparing IL-Sbv with topical 15% paromomycin ointment, IL hypertonic saline, radiofrequency-induced heat therapy, topical trichloroacetic acid and cryotherapy showed no significant difference in efficacy between the interventions. The analyses suggested a higher efficacy of IL-Sbv combined with cryotherapy (81.8%, 95%IC 62.4–92.4%) when compared with IL-Sbv alone (53.3%, 95%IC 46.1–66%), OR: 3.14 (95%CI 1.1–8.9), p = 0.03. In the New World, the global IL-Sbv efficacy was 77%(95%CI 66–85%). In contrast with the Old World, a significant difference favoring MA in relation to SSG was observed: 61% (95%CI 49–73%) versus 82% (95%CI 70–89%).By comparing IL infiltration schedules, it was determined that patients submitted to IL-Sbv treatments longer than 14 days had higher cure rates. Conclusions: Despite the high heterogeneity and low methodological quality of studies, an indirect comparison shows that the antimony infiltration efficacy rate is similar to that reported for antimony systemic use. The evidence gathered thus far is insufficient to identify the ideal IL therapeutic regime or estimate the rates of adverse events and mucosal late complications. [ABSTRACT FROM AUTHOR]

Introduction: The National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. The Ministry of Health (MS) guideline recommends treatment of phenylketonuria (PKU) with restricted phenylalanine diet and phenylalanine-free amino acid formula (PFAAf) supplementation. CONITEC evaluated the inclusion of sapropterin dihydrochloride for PKU in the SUS. Methods: The population eligible for treatment was evaluated by the number of patients receiving PFAAf between 2014 and 2017 registered in the SUS. Patients were stratified by age/weight and a simple linear regression was performed to estimate the future population. The costs of treatment and testing the responsiveness of sapropterin dihydrochloride were estimated according to the recommended dosage guideline of the MS, leaflet and public purchasing prices. A univariate deterministic sensitivity analysis was performed to evaluate different prices, responsiveness test methods and variations in the reduction of formula use. Results: The incorporation of sapropterin dihydrochloride would generate an incremental budget impact in the SUS of around BRL 79 million (USD 21.7 million) in 2019 and BRL 300 million (USD 82.1 million) in five years (2019-2023). The univariate sensitivity analysis estimated that the incremental budget impact could be between BRL 66 and BRL 103 million (USD 18 and USD 28 million) in the first year and between BRL 251 and BRL 388 million (USD 69 and USD 106 million) in five years. Sensitivity analysis showed that the price of sapropterin dihydrochloride was the most sensitive variable in the model. Conclusions: The incorporation of sapropterin dihydrochloride in the SUS represents a significant budgetary impact and covers a small number of patients. Sapropterin dihydrochloride was recommended by CONITEC for the treatment of women with PKU, with a positive drug responsiveness test, and who are in the preconception period or in the gestational period. [ABSTRACT FROM AUTHOR]