Augusto Afonso Guerra Júnior

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems

Medicines have made an appreciable contribution to improving health. However, even high-income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimize their use. The objective is to review case histories among health authorities to improve the utilization and expenditure on new medicines. Subsequently, use these to develop exemplar models and outline their implications. A number of issues and challenges were identified from the case histories. These included the low number of new medicines seen as innovative alongside increasing requested prices for their reimbursement, especially for oncology, orphan diseases, diabetes and HCV. Proposed models center on the three pillars of pre-, peri- and post-launch including critical drug evaluation, as well as multi-criteria models for valuing medicines for orphan diseases alongside potentially capping pharmaceutical expenditure. In conclusion, the proposed models involving all key stakeholder groups are critical for the sustainability of healthcare systems or enhancing universal access. The models should help stimulate debate as well as restore trust between key stakeholder groups.

Disponibilidade de medicamentos essenciais em duas regiões de Minas Gerais, Brasil

OBJETIVO: Avaliar a disponibilidade de medicamentos essenciais em localidades com indice de desenvolvimento humano < 0,699. METODOS: Foram pesquisados 69 estabelecimentos, entre almoxarifados municipais, unidades ambulatoriais publicas, unidades privadas, unidades filantropicas e farmacias comerciais. Foram entrevistados os profissionais de saude diretamente responsaveis pelo controle dos estoques e dispensacao dos medicamentos. Tambem foram consultados documentos e registros institucionais. Uma lista de 21 medicamentos essenciais tracadores, selecionados dentre aqueles mais utilizados no Programa Farmacia Basica do Estado de Minas Gerais, foi definida como parâmetro para as medidas de disponibilidade. Para cada medicamento tracador foi verificada a disponibilidade no momento da visita e ao longo dos 12 meses imediatamente anteriores a pesquisa de campo. Tambem foi calculada a disponibilidade de cada um dos medicamentos essenciais tracadores para cada tipo de estabelecimento incluido na pesquisa de campo. RESULTADOS: A disponibilidade de medicamentos essenciais nos almoxarifados municipais foi de 52,0%; nas unidades publicas de saude, 46,9%; e nas unidades ambulatoriais filantropicas e privadas, 41,0% e 38,1%, respectivamente. Nas farmacias privadas, a disponibilidade de medicamentos essenciais alcancou 81,2%. CONCLUSAO: Ha baixa disponibilidade e descontinuidade na oferta de medicamentos essenciais no setor publico, penalizando individuos vulneraveis. As farmacias privadas sao a principal fonte de medicamentos essenciais. Os resultados indicam a necessidade de divulgar e implementar o conceito de essencialidade no pais.

Perfil epidemiológico dos pacientes em terapia renal substitutiva no Brasil, 2000-2004

OBJECTIVE To describe the clinical and epidemiological profile of patients under renal replacement therapies, identifying risk factors for death. METHODS This is a non-concurrent cohort study of data for 90,356 patients in the National Renal Replacement Therapies Database. A deterministic-probabilistic linkage was performed using the Authorization System for High Complexity/Cost Procedures and the Mortality Information System databases. All patients who started dialysis between 1/1/2000 and 12/31/2004 were included and followed until death or the end of 2004. Age, sex, region of residence, primary renal disease and causes of death were analyzed. A proportional hazards model was used to identify factors associated with risk of death. RESULTS The prevalence of patients under renal replacement therapies increased an average of 5.5%, while incidence remained stable during the period. Hemodialysis was the predominant initial modality (89%). The patients were majority male with mean age 53 years, residents of the Southeast region and presented unknown causes as the main cause of chronic renal disease, followed by hypertension, diabetes and glomerulonephritis. Of these patients, 42% progressed to death and 7% underwent kidney transplantation. The patients on peritoneal dialysis were older and had higher prevalence of diabetes. The death rate varied from 7% among transplanted patients to 45% among non-transplanted patients. In the final Cox proportional hazards model, the risk of mortality was associated with increasing age, female sex, having diabetes, living in the North and Northeast region, peritoneal dialysis as a first modality and not having renal transplantation. CONCLUSIONS There was an increased prevalence of patients on renal therapy in Brazil. Increased risk of death was associated with advanced age, diabetes, the female sex, residents of the North and Northeast region and lack of renal transplant.OBJETIVO: Describir el perfil epidemiologico y clinico de pacientes en terapia renal substitutiva, identificando factores asociados al riesgo de muerte. METODOS: Estudio de observacion, prospectivo no concurrente, a partir de datos de 90.356 pacientes de la Base Nacional en Terapias Renales Substitutivas, en Brasil. Fue realizado reracionamiento deterministico-probabilistico del Sistema de Informacion de Mortalidad. Fueron incluidos todos los pacientes incidentes que iniciaron dialisis entre 1/1/2000 y 31/12/2004, acompanados hasta la muerte o final de 2004. Edad, sexo, region de residencia, enfermedad renal primaria, causa del obito fueron analizados. Se ajusto un modelo de riesgos proporcionales para identificar factores asociados al riesgo de muerte. RESULTADOS: Ocurrio un aumento promedio de 5,5% en la prevalencia de pacientes en terapia, con relacion a la incidencia se mantuvo estable en el periodo. Hemodialisis fue la modalidad inicial predominante (89%). La mayoria de los pacientes era del sexo masculino, con edad promedio de 53 anos, residente en la region Sureste y presentaba causa indeterminada como principal causa basica de la enfermedad renal cronica, seguida de la hipertension, diabetes y glomerulonefritis. De esos pacientes, 7% realizaron transplante renal y 42% evolucionaron a obito. Los pacientes en dialisis peritoneal eran mas ancianos y presentaban mayor prevalencia de diabetes. Entre los no transplantados, 45% fueron a obito y, entre los transplantadas 7%. En el modelo final de riesgos proporcionales de Cox, el riesgo de mortalidad estuvo asociado con el aumento de la edad, sexo femenino, tener diabetes, residir en la region Norte y Noreste, dialisis peritoneal como modalidad de entrada y no haber realizado transplante renal. CONCLUSIONES: Hubo aumento de la prevalencia de pacientes en terapia renal en Brasil. Pacientes con edad avanzada, diabetes, del sexo femenino, residentes en la region Norte y Noreste y sin transplante renal presentan mayor riesgo de muerte.

A construção da base de dados nacional em Terapia Renal Substitutiva (TRS) centrada no indivíduo: aplicação do método de linkage determinístico-probabilístico

Os sistemas de informação em saúde são ferramentas fundamentais para subsidiar a tomada de decisões e auxiliar a organização dos serviços, por meio do planejamento das ações e do acompanhamento e avaliação dos objetivos propostos. O sistema de saúde brasileiro tem larga experiência com a captura e o uso de informações. No entanto, Morais e Gómez (2007) fazem uma reflexão de que os atuais pressupostos, práticas e saberes de informação e informática em saúde não mais dão conta da complexidade do processo saúde/doença/cuidado, apontando algumas questões: a fragmentação dos indivíduos entre diversas bases de dados em saúde, o que acar-

Efficacy of Mobile Apps to Support the Care of Patients With Diabetes Mellitus: A Systematic Review and Meta-Analysis of Randomized Controlled Trials.

Background Diabetes Mellitus (DM) is a chronic disease that is considered a global public health problem. Education and self-monitoring by diabetic patients help to optimize and make possible a satisfactory metabolic control enabling improved management and reduced morbidity and mortality. The global growth in the use of mobile phones makes them a powerful platform to help provide tailored health, delivered conveniently to patients through health apps. Objective The aim of our study was to evaluate the efficacy of mobile apps through a systematic review and meta-analysis to assist DM patients in treatment. Methods We conducted searches in the electronic databases MEDLINE (Pubmed), Cochrane Register of Controlled Trials (CENTRAL), and LILACS (Latin American and Caribbean Health Sciences Literature), including manual search in references of publications that included systematic reviews, specialized journals, and gray literature. We considered eligible randomized controlled trials (RCTs) conducted after 2008 with participants of all ages, patients with DM, and users of apps to help manage the disease. The meta-analysis of glycated hemoglobin (HbA1c) was performed in Review Manager software version 5.3. Results The literature search identified 1236 publications. Of these, 13 studies were included that evaluated 1263 patients. In 6 RCTs, there were a statistical significant reduction (P<.05) of HbA1c at the end of studies in the intervention group. The HbA1c data were evaluated by meta-analysis with the following results (mean difference, MD −0.44; CI: −0.59 to −0.29; P<.001; I²=32%).The evaluation favored the treatment in patients who used apps without significant heterogeneity. Conclusions The use of apps by diabetic patients could help improve the control of HbA1c. In addition, the apps seem to strengthen the perception of self-care by contributing better information and health education to patients. Patients also become more self-confident to deal with their diabetes, mainly by reducing their fear of not knowing how to deal with potential hypoglycemic episodes that may occur.

Introduction and Utilization of High Priced HCV Medicines across Europe; Implications for the Future

Background: Infection with the Hepatitis C Virus (HCV) is a widespread transmittable disease with a diagnosed prevalence of 2.0%. Fortunately, it is now curable in most patients. Sales of medicines to treat HCV infection grew 2.7% per year between 2004 and 2011, enhanced by the launch of the protease inhibitors (PIs) boceprevir (BCV) and telaprevir (TVR) in addition to ribavirin and pegylated interferon (pegIFN). Costs will continue to rise with new treatments including sofosbuvir, which now include interferon free regimens. Objective: Assess the uptake of BCV and TVR across Europe from a health authority perspective to offer future guidance on dealing with new high cost medicines. Methods: Cross-sectional descriptive study of medicines to treat HCV (pegIFN, ribavirin, BCV and TVR) among European countries from 2008 to 2013. Utilization measured in defined daily doses (DDDs)/1000 patients/quarter (DIQs) and expenditure in Euros/DDD. Health authority activities to influence treatments categorized using the 4E methodology (Education, Engineering, Economics and Enforcement). Results: Similar uptake of BCV and TVR among European countries and regions, ranging from 0.5 DIQ in Denmark, Netherlands and Slovenia to 1.5 DIQ in Tayside and Catalonia in 2013. However, different utilization of the new PIs vs. ribavirin indicates differences in dual vs. triple therapy, which is down to factors including physician preference and genotypes. Reimbursed prices for BCV and TVR were comparable across countries. Conclusion: There was reasonable consistency in the utilization of BCV and TVR among European countries in comparison with other high priced medicines. This may reflect the social demand to limit the transmission of HCV. However, the situation is changing with new curative medicines for HCV genotype 1 (GT1) with potentially an appreciable budget impact. These concerns have resulted in different prices across countries, with their impact on budgets and patient outcomes monitored in the future to provide additional guidance.

Determinants of expenditures on dialysis in the Unified National Health System, Brazil, 2000 to 2004

The aim of this study was to compare total outpatient expenditures on hemodialysis and peritoneal dialysis from 2000 to 2004 in patients that began dialysis in 2000 under the Unified National Health System (SUS). A historical cohort was developed, consisting of patients that began dialysis in 2000, identified by probabilistic matching in the database of Authorizations for High-Complexity/High-Cost Procedures (APAC). A multiple linear regression model was used, including individual and clinical attributes and health services supply variables. The cohort included 10,899 patients, 88.5% of whom began hemodialysis and 11.5% peritoneal dialysis. The dialysis modality explains 12% of the variance in expenditures, and patients in peritoneal dialysis showed 20% higher mean annual expenditure. The differences in expenditures are explained according to the State of Brazil and health services supply level. Individual risk variables did not alter the models explanatory power, while age and diabetes mellitus were significant. The study showed the importance of the National Health Systems payment mechanism for explaining differences in expenditures on dialysis treatment in Brazil.

Ciclosporina versus tacrolimus no transplante renal no Brasil: uma comparação de custos

In Brazil, the Unified National Health System (SUS) is responsible for the majority of kidney transplants. To maintain these interventions, the guidelines recommend the use of cyclosporine or tacrolimus, associated with corticosteroids and azathioprine or mycophenolate. Taking the perspective of the National Health System, an economic analysis was performed on the outpatient and hospital resources and medicines used by patient and therapeutic group. A cohort was constructed from 2000 to 2004, with 5,174 kidney transplant patients in use of cyclosporine or tacrolimus, identified by probabilistic record linkage from the National Health System. The cohort included 4,015 patients in use of cyclosporine and 1,159 using tacrolimus. The majority were males, age < 38 years, with nephritis, cardiovascular diseases, and indeterminate causes as the most frequent primary diagnoses. After 48 months of follow-up, the expenditures were higher for kidney transplants in hospitals in the Northeast, cadaver donors, patients in dialysis > 24 months before the transplant, and in the tacrolimus group. Total hospital and outpatient costs and expenditure on medication were higher in patients on tacrolimus as compared to the cyclosporine group.

Pesquisa e produção científica em economia da saúde no Brasil

This article presents a diagnosis of the health economics (HE) in Brazil, based on a survey of HE research groups currently registered in the National Council for Scientific and Technological Development (CNPq), as well as an inventory of the Brazilian scientific production in HE, published between January 1999 and June 2004, available in the Bireme Virtual Health Library (Paho/WHO). It describes the data considering geographic regions, types of institution and topics of research. Of the research groups with works related to HE subject matters, 48 have very diversified activities, concentrated in the Southeastern region of the country. Only 14% (376) of the 2.617 publications that had been evaluated were related to HE. Most of the studies were published in 2002 and their main topics were management, financing, allocative efficiency and equity in the distribution of health resources. The diagnosis allows the identification of important aspects that help understand the development of the HE field in Brazil between 1999 and 2004.

CYD-TDV dengue vaccine: systematic review and meta-analysis of efficacy, immunogenicity and safety

INTRODUCTION Dengue virus (DENV) is a serious global health problem. CYD-TDC (Dengvaxia®) was the first vaccine to gain regulatory approval to try and address this problem. AIM Summarize all available evidence on the immunogenicity, efficacy and safety of the CYD-TDV dengue vaccine. METHOD Meta-analysis and systematic review. RESULTS The best and worst immunogenicity results were for DENV4 and DENV1, respectively. Vaccine efficacy of 60% was derived from studies with participants aged 2-16 years old, with DENV4 and DENV2 presenting the best and worst results, respectively. Erythema and swelling were more frequent with CYD-TDV. No differences were detected for systemic adverse events. CONCLUSION CYD-TDV showed moderate efficacy in children and adolescents. From the immunogenicity results in adults, we can expect satisfactory efficacy from vaccination in this population.