Acordo de Compartilhamento de Risco: projeto-piloto no Sistema Único de Saúde / Risk Sharing Agreement: a pilot project in the Brazilian Unified Health System

O Acordo de Compartilhamento de Risco é definido como um acordo no qual o Estado concorda em oferecer acesso temporário a um novo medicamento, enquanto a indústria farmacêutica aceita receber pelo produto conforme o desempenho do medicamento em reais condições de uso. A partilha de risco depende, necessariamente, da coleta de evidências adicionais, que podem se referir aos benefícios terapêuticos ou ao volume de pacientes, conforme avaliação de seu uso na prática. Os autores descreveram a experiência do projeto-piloto de Acordo de Compartilhamento de Risco no Sistema Único de Saúde.

Risk Sharing Agreement is defined as an agreement in which the State agrees to offer temporary access to a new drug, while the pharmaceutical industry accepts to receive the product according to the performance of the drug in real conditions of use. Risk sharing necessarily depends on the collection of additional evidence that may refer to the therapeutic benefits or the volume of patients, according to the assessment of its use in practice. The authors described the experience of the pilot project of a Risk Sharing Agreement in the Unified Health System.

Risk Sharing Agreement: a pilot project in the Brazilian Unified Health System

Risk Sharing Agreement is defined as an agreement in which the State agrees to offer temporary access to a new drug, while the pharmaceutical industry accepts to receive the product according to the performance of the drug in real conditions of use. Risk sharing necessarily depends on the collection of additional evidence that may refer to the therapeutic benefits or the volume of patients, according to the assessment of its use in practice. The authors described the experience of the pilot project of a Risk Sharing Agreement in the Unified Health System.

Epidemiological characteristics and survival outcomes of children with medulloblastoma treated at the National Cancer Institute (INCA) in Rio de Janeiro, Brazil.

BACKGROUND: Medulloblastoma (MB),the most common malignant brain tumor of childhood has survival outcomes exceeding 80% for standard-risk and 60% for high-risk patients in high-income countries (HICs). These results have not been replicated in low- and middle-income countries (LMICs), where 80% of children with cancer live. METHODS: This is a retrospective review of 114 children aged 3-18 years diagnosed with MB from 1997 to 2016 at National Cancer Institute (INCA). Sociodemographic, clinical, and treatment data were extracted from the medical records and summarized descriptively. Overall survival (OS) and progression-free survival (PFS) were calculated using the Kaplan-Meier method. RESULTS: The male-to-female ratio was 1.32 and the median age at diagnosis was 8.2 years. Headache (83%) and nausea/vomiting (78%) were the most common presenting symptoms. Five-year OS was 59.1% and PFS was 58.4%. The OS for standard-risk and high-risk patients was 69% and 53%, respectively. The median time to diagnosis interval was 50.5 days and the median time from surgery to radiation therapy initiation was 50.4 days. Patients who lived >40 km from INCA fared better (OS = 68.2% vs. 51.1%, p = .032). Almost 20% of families lived below the Brazilian minimum wage. Forty-five patients (35%) had metastatic disease at admission. Gross total resection was achieved in 57% of the patitents. CONCLUSIONS: Although there are considerable barriers to deliver effective MB treatment in countries like Brazil, the OS seen in the present study demonstrates that good outcomes are not only feasible but can and should be increased with appropriate interventions.

Cost-Utility of Gastric Bypass Surgery Compared to Clinical Treatment for Severely Obese With and Without Diabetes in the Perspective of the Brazilian Public Health System.

PURPOSE: Obesity is associated with increased morbidity and mortality. Weight loss due to gastric bypass (GBP) surgery improves clinical outcomes and may be a cost-effective intervention. To estimate the cost-effectiveness of GBP compared to clinical treatment in severely obese individuals with and without diabetes in the perspective of the Brazilian public health system. MATERIALS AND METHODS: A Markov model was developed to compare costs and outcomes of gastric bypass in an open approach to clinical treatment. Health states were living with diabetes, remission of diabetes, non-fatal and fatal myocardial infarction, and death. We also included the occurrence of complications related to surgery and plastic surgery after the gastric bypass surgery. The direct costs were obtained from primary data collection performed in three public reference centers for obesity treatment. Utility values also derived from this cohort, while transition probabilities came from the international literature. A sensitivity analysis was performed to evaluate uncertainties. The model considered a 10-year time horizon and a 5% discount rate. RESULTS: Over 10 years, GBP increased quality-adjusted life years (QALY) and costs compared to clinical treatment, resulting in an incremental cost-effectiveness ratio (ICER) of Int$1820.17/QALY and Int$1937.73/QALY in individuals with and without diabetes, respectively. Sensitivity analysis showed that utility values and direct costs of treatments were the parameters that affected the most the ICERs. CONCLUSION: The study demonstrated that GBP is a cost-effective intervention for severely obese individuals in the Brazilian public health system perspective, with a better result in individuals with diabetes.

Ideal vial size for bortezomib: real-world data on waste and cost reduction in treatment of multiple myeloma in Brazil.

OBJECTIVES: Single-size vials of drugs may be a source of waste and increase in treatment costs. Bortezomib, indicated for multiple myeloma (MM) treatment, is available in 3.5-mg vials, a quantity higher than the average dose commonly prescribed. This analysis aimed to demonstrate, through real-world data, which would be the optimal vial presentation for bortezomib in Brazil and quantify the reduction in medication waste related to this option. METHODS: From November 2007 to October 2009 all patients with MM treated with bortezomib were identified via the Evidências database. Analysis of prescribed, dispensed, and wasted doses, their costs and projections of the ideal vial size were performed. RESULTS: Thirty-five patients (mean body surface area of 1.73 m(2)) received 509 infusions in 131 cycles of treatment (average of 3.77 cycles per patient). The average dose prescribed was 2.1 mg per infusion (95% confidence interval [CI] 1.97-2.26) with average waste of 39.5% of the vial content (95% CI 35.35-43.76). The mean waste per patient per day was 1.38 mg (95% CI 1.24-1.52). If a 3-mg vial were available, the average drug waste per patient per day would be 0.88 mg (95% CI 0.74-1.03) or 36.2% less. With a 2.5-mg vial the waste would be 1.05 mg (95% CI 0.81-1.29) or 23.9% less. If two presentations were available (2.5 mg and 0.5 mg), the waste would be 0.52 mg (95% CI 0.4-0.63) or 62.5% less. Considering the price of the different vials to be proportional to the original 3.5-mg vial, the cost would be also reduced by the same rates described above. CONCLUSIONS: A simple adjustment in vial size may reduce the waste of bortezomib by 36% to 62% and can also reduce the cost of treatment.

Há relação entre custo-efetividade de acordo com diferentes metas?: [revisão] / Is there relationship between cost-effectiveness in accordance with different goals?: [review]

Análise de custo-efetividade é uma razão, tal como custo por ano de vida salvo, em que o denominador reflete o ganho em saúde oriundo de uma intervenção específica (exemplos: anos de vida ganhos, número de acidentes cerebrovasculares evitados) e o numerador reflete o custo monetário para se obter o ganho em saúde. As relações de custo-efetividade vão variar de acordo com a meta de tratamento e o tipo de população que recebeu a intervenção.Quanto maior o risco cardiovascular da população tratada, maior o benefício da intervenção, em termos de redução de eventos cardiovasculares.

Cost-effectiveness analysis produces a ratio, such as the cost per year of life gained, where the denominator reflects the gain in health from a specific intervention (e.g., life-years gained, number of strokes averted) and the numerator reflects the monetary cost of obtaining that gain.The cost-effectiveness will vary with the goal of treatment and type of population that received the intervention. The higher the cardiovascular risk of the treated population, the greater the benefit from intervention in terms of reducing cardiovascular events.

Estudo prospectivo não-randomizado e multicêntrico comparando stents farmacológicos com stents convencionais em pacientes multiarteriais / Prospective non-randomized multicenter study comparing pharmacological stents with conventional stents in multi-arterial patients

Fundamentos: A angioplastia em multiarteriais é segura no tratamento das coronariopatias. Objetivo: Comparar stents farmacológicos (SF) e convencionais (SC) em pacientes multiarteriais. Método: Avaliação prospectiva: 109 pacientes, outubro/2002 a setembro/2005: 72 do grupo SF e 37 do grupo SC. Desfechos: óbito, reestenose clínica e a combinação de ambos. Resultados: Seguimento: 24,6 maior ou menor que 9,5 meses. Havia 52 (72,2 por cento) homens no SF e 24 (64,9 por cento) no SC. Idade média: 66,2 maior ou menor que 10,5 (42 a 90) anos.Comorbidades foram mais presentes no SF...

Estudo prospectivo da angioplastia percutânea com stents farmacológicos em pacientes diabéticos / Prospective study of percutaneous angioplasty with pharmacological stents in diabetic patients

Objetivo: Avaliar os resultados tardios da angioplastia com os stents farmacológicos (SF) Paclitaxel (Taxus) e Rapamicina (Cypher) em pacientes diabéticos. Métodos: Estudo prospectivo de amostra não-selecionada de pacientes consecutivos, compreendendo 107 indivíduos tratados eletivamente por angioplastia com SF, no período de maio de 2003 a dezembro de 2005. Em 74 pacientes (70,5 por cento) foi utilizado stent Taxus, e em 31 (29,5 por cento), Cypher. Os desfechos no período foram: óbito, restenose, revascularização da lesão-alvo (RLA) e do vaso-alvo (RVA). Resultados: A idade média dos pacientes foi de 65,3 mais ou menos 9,5 amos (46-86 anos), sendo 63,8 por cento homens (IC 95 por cento igual 53,8 por cento a 72,8 por cento). O seguimento foi de 24,9 mais ou menos 9,0 meses (12,-54,6 meses). Os fatores de risco...

Pharmacoeconomics applied to chronic hepatitis C.

Life expectancy has increased over the last century as it had never been before. This is the result of a combination of many favorable variables such as level of education, improved socio-economic environment and development of medicine. However, new improvements demand heavy investment. Thus, the incorporation of medical technology became a health and economic issue. The pharmacoeconomic knowledge field is being developed to help in the analysis of medical costs and patient needs. The applies to hepatitic C, a common and chronic worldwide disease. In this article, the authors describe the rational behind this type of health economic analysis and review a hepatitis C model. Overall, in a non-Brazilian scenario, it was demonstrated that peginterferon alfa-2a (40KD) is cost effective in the treatment of HCV disease.

Pharmacoeconomics applied to chronic hepatitis C

Life expectancy has increased over the last century as it had never been before. This is the result of a combination of many favorable variables such as level of education, improved socio-economic environment and development of medicine. However, new improvements demand heavy investment. Thus, the incorporation of medical technology became a health and economic issue. The pharmacoeconomic knowledge field is being developed to help in the analysis of medical costs and patient needs. The applies to hepatitic C, a common and chronic worldwide disease. In this article, the authors describe the rational behind this type of health economic analysis and review a hepatitis C model. Overall, in a non-Brazilian scenario, it was demonstrated that peginterferon alfa-2a (40KD) is cost effective in the treatment of HCV disease.