The commonest prescribed group of drugs is antimicrobials. Various studies have shown that they are overused globally. Since Primary health care represents the first tier of the health care system, evaluation of antimicrobial use in primary health w e settings is a necessity to ensure rational and cost-effective use of these agents in the treatment of infectious diseases. It has been reported by Hooton and Levy (2001 : 1088) that 20% to 50% of antimicrobials are inappropriately used in developing countries. According to Rebana et al. (1998: 175) the increasing overuse of antimicrobials has resulted in an enormous escalation in the total costs of drugs contributing to 15% to 30 % of the total health budget. Hooton and Levy (2001: 1087) reported in a study that inappropriate use and overuse of antimicrobials are risk factors for the emergence of antibiotic resistant bacteria. There is a high incidence of infectious diseases in developing countries that are due to the rapid spread of resistant strains through over-crowding, poor sanitation and unsafe sexual practices (Liu et al., 1999: 540). The general objective of the study was the analysis and interpretation of the usage and related costs of antimicrobial prescriptions in a private primary health w e setting in South Africa. The study is a non-experimental, quantitative, retrospective drug utilisation review of antimicrobial usage in a private primary health care setting. Data were obtained from the central database of a private primary health care service provider. Data of nine randomly selected clinics, situated in different geographical areas of South Africa, were extracted for the period 1st January to 31st December 2001. The study population was made of the total patient population of patients using antimicrobials during this one year period. Antimicrobial usage was analysed according to: number of patients, age and gender distribution, diagnosis, pharmacological groups. The total number of patients who visited the nine clinics during the year was 83 655 of which 59.50% were females and 40.22% males. In 0.28% of the cases gender was not indicated. Patients in age groups 6 (20-40 years) and 7 (40-60 years) accounted for the highest number of patients (66.31%, n = 54 964). A total of 515 976 medicine items costing R1 716 318.90 were prescribed, of these, 18.69%, (N=96 423) were antimicrobials costing 60.89%, (R1 045 108.00). Of the total number of patients that visited the nine clinics, 65.34% (N=54 663) were prescribed antimicrobials. The total number of diagnoses (140 723) where antimicrobials were prescribed accounted for 68.52% (N46 42 1). The highest number of antimicrobial prescriptions according to pharmacological and age groups were: penicillins followed by sulphonamides and tetracyclines. The diagnoses with the highest number of antimicrobial prescriptions were the respiratory tract infections (viral influenza, acute bronchitis and upper respiratory tract infection) and pelvic inflammatory disease The prescribing of antimicrobials in respiratory tract infections could indicate overuse and inappropriate use of these drugs. Because most of these infections are caused by viruses or other non-bacterial agents, are self limiting. Therefore, the use of antibiotics courses is neither necessary nor appropriate in these conditions. The overuse and inappropriate use of such drugs have an effect on the health of the patients needing cure, and the general budget on health care service. It is recommended that further studies are conducted on antimicrobial prescribing and use. / Thesis (M. Pharm. (Pharmacy Practice))--North-West University, Potchefstroom Campus, 2005.

South African prescribers have a large choice of combination analgesic preparations available for prescribing. According to Desmeules et al. (2003:8) the advantages of combining analgesics include increasing the duration of analgesia, widening the spectrum of efficacy, improved patient compliance and reduced parenteral abuse potential. According to McMahon (1975:13) one of the principle arguments against fixed-dose combinations is that the physician surrenders flexibility in managing his patient. Combination analgesics may expose patients to ingredients not necessary for pain relief in their particular condition (Beaver, 1984). Rigas (1997:454) explains that the value of pharmaco-economics in providing cost-effective pharmacologic treatment for pain must not only be seen as a containment effort, but rather as a valuation effort. Meaningful economic analyses based on empiric information about cost and a range of subjective and objective outcomes are needed to minimise cost without compromising care. The objective of this study was to review and interpret the prescribing patterns of combination analgesics and the cost associated with their usage for the period 2001-2006 in a section of the private healthcare sector in South Africa. This research can be classified as a quantitative, retrospective drug utilisation review study. Data were obtained from a medicine claims database, and the study population consisted of all combination analgesic prescriptions (Mims® category 3.3) for the period 1 January 2001 to 31 December 2002 and 1 January 2004 to 31 December 2006. Prescribing Patterns of Combination Analgesics in the Private Health Care Sector. Firstly pain and the treatment thereof with combination analgesics were investigated from the literature to understand the disease and to determine the prevalence and treatment thereof. Secondly, managed health care, drug utilisation review, pharmacoeconomics and pharmaco-epidemiology were investigated from the literature to understand these concepts. The influence of the South African government on the medicine pricing regulations was discussed. Thirdly, through the empirical investigation the utilisation patterns of combination analgesics were reviewed, analysed and interpreted. It was determined that combination analgesic drugs represented 8.87% (n=261 907) of all medicine claimed during 2001 (N=2 951 326), decreased to 7.20% (n=381 809) during 2004 (N=5 305 846) after which it increased to 7.92% (n=187 745) in 2006 (N=2 370 572). Between 2001 (N=R379 708 489.00) and 2006 (N=R279 160 832.00) the cost percentage of the combination analgesic drugs decreased from 4.95% (n=R18 798 202.42) to 3.15% (n=R8 791 228.57). The average cost per combination analgesic drugs decreased from R71.77 ± 61.67 to R46.83 ± 43.41 between 2001 and 2006. This decrease was of no practical significance (d<0.8). The average number of combination analgesics per prescription stayed relatively constant varying between 1.01 ± 0.11 in 2001 and 1.02 ± 0.13 in 2006. The percentage generic combination analgesic drugs claimed increased from 29.63% (n=77 608) in 2001 to 66.37% (n=124 600) in 2006 (N=261 907 for 2001 and N=187 745 for 2006) even though generic medicine items claimed by the total database only increased from 26.79% (n=790 548) in 2001 to 40.27% (n=954 561) during 2006 (N=2 951 326 for 2001 and N=2 370 572 for 2006). The combination of ibuprofen 200mg, paracetamol 250mg and codeine phosphate 10mg (e.g. Myprodol® capsules, Mybulen® capsules, Gen-payne® capsules and Ibupain Forte® capsules) represented the active ingredient combination with the highest prevalence for the entire study period, increasing from 28.44% (n=74 483) in 2001 to 33.08% (n=62 100) in 2006 of all combination analgesics prescribed (N=261 907 for 2001 and N=187 745 for 2006). Generic substitution influenced the prevalence of the innovator medicine item, Myprodol® Capsules dramatically, causing a decrease from 23.16% (n=60 631) in 2001 to 3.77% (n=7 084) in 2006 representation of all combination analgesic prescribed. In 2006, the generics of Myprodol® Capsules e.g. Dentopain Forte®, Mybulen® Capsules, Gen-payne® and Ibupain Forte® represented 23.79% (n=44651) of all combination analgesics claimed. Recommendations were derived regarding certain aspects of the clinical and economical management of pain e.g. the implication of generic substitution with regard to cost and prescribing patterns, and the decreasing cost of combination analgesics which might encourage abuse, needs further investigation. South African prescribers have a large choice of combination analgesic preparations available for prescribing. According to Desmeules et al. (2003:8) the advantages of combining analgesics include increasing the duration of analgesia, widening the spectrum of efficacy, improved patient compliance and reduced parenteral abuse potential. According to McMahon (1975:13) one of the principle arguments against fixed-dose combinations is that the physician surrenders flexibility in managing his patient. Combination analgesics may expose patients to ingredients not necessary for pain relief in their particular condition (Beaver, 1984). Rigas (1997:454) explains that the value of pharmaco-economics in providing cost-effective pharmacologic treatment for pain must not only be seen as a containment effort, but rather as a valuation effort. Meaningful economic analyses based on empiric information about cost and a range of subjective and objective outcomes are needed to minimise cost without compromising care. The objective of this study was to review and interpret the prescribing patterns of combination analgesics and the cost associated with their usage for the period 2001-2006 in a section of the private healthcare sector in South Africa. This research can be classified as a quantitative, retrospective drug utilisation review study. Data were obtained from a medicine claims database, and the study population consisted of all combination analgesic prescriptions (Mims® category 3.3) for the period 1 January 2001 to 31 December 2002 and 1 January 2004 to 31 December 2006. Prescribing Patterns of Combination Analgesics in the Private Health Care Sector. Firstly pain and the treatment thereof with combination analgesics were investigated from the literature to understand the disease and to determine the prevalence and treatment thereof. Secondly, managed health care, drug utilisation review, pharmacoeconomics and pharmaco-epidemiology were investigated from the literature to understand these concepts. The influence of the South African government on the medicine pricing regulations was discussed. Thirdly, through the empirical investigation the utilisation patterns of combination analgesics were reviewed, analysed and interpreted. It was determined that combination analgesic drugs represented 8.87% (n=261 907) of all medicine claimed during 2001 (N=2 951 326), decreased to 7.20% (n=381 809) during 2004 (N=5 305 846) after which it increased to 7.92% (n=187 745) in 2006 (N=2 370 572). Between 2001 (N=R379 708 489.00) and 2006 (N=R279 160 832.00) the cost percentage of the combination analgesic drugs decreased from 4.95% (n=R18 798 202.42) to 3.15% (n=R8 791 228.57). The average cost per combination analgesic drugs decreased from R71.77 ± 61.67 to R46.83 ± 43.41 between 2001 and 2006. This decrease was of no practical significance (d<0.8). The average number of combination analgesics per prescription stayed relatively constant varying between 1.01 ± 0.11 in 2001 and 1.02 ± 0.13 in 2006. The percentage generic combination analgesic drugs claimed increased from 29.63% (n=77 608) in 2001 to 66.37% (n=124 600) in 2006 (N=261 907 for 2001 and N=187 745 for 2006) even though generic medicine items claimed by the total database only increased from 26.79% (n=790 548) in 2001 to 40.27% (n=954 561) during 2006 (N=2 951 326 for 2001 and N=2 370 572 for 2006). The combination of ibuprofen 200mg, paracetamol 250mg and codeine phosphate 10mg (e.g. Myprodol® capsules, Mybulen® capsules, Gen-payne® capsules and Ibupain Forte® capsules) represented the active ingredient combination with the highest prevalence for the entire study period, increasing from 28.44% (n=74 483) in 2001 to 33.08% (n=62 100) in 2006 of all combination analgesics prescribed (N=261 907 for 2001 and N=187 745 for 2006). Generic substitution influenced the prevalence of the innovator medicine item, Myprodol® Capsules dramatically, causing a decrease from 23.16% (n=60 631) in 2001 to 3.77% (n=7 084) in 2006 representation of all combination analgesic prescribed. In 2006, the generics of Myprodol® Capsules e.g. Dentopain Forte®, Mybulen® Capsules, Gen-payne® and Ibupain Forte® represented 23.79% (n=44651) of all combination analgesics claimed. Recommendations were derived regarding certain aspects of the clinical and economical management of pain e.g. the implication of generic substitution with regard to cost and prescribing patterns, and the decreasing cost of combination analgesics which might encourage abuse, needs further investigation. / Thesis (M.Pharm. (Pharmacy Practice))--North-West University, Potchefstroom Campus, 2008.

Thesis (M. Pharm.)--North-West University, Potchefstroom Campus, 2008.

South African prescribers have a large choice of combination analgesic preparations available for prescribing. According to Desmeules et al. (2003:8) the advantages of combining analgesics include increasing the duration of analgesia, widening the spectrum of efficacy, improved patient compliance and reduced parenteral abuse potential. According to McMahon (1975:13) one of the principle arguments against fixed-dose combinations is that the physician surrenders flexibility in managing his patient. Combination analgesics may expose patients to ingredients not necessary for pain relief in their particular condition (Beaver, 1984). Rigas (1997:454) explains that the value of pharmaco-economics in providing cost-effective pharmacologic treatment for pain must not only be seen as a containment effort, but rather as a valuation effort. Meaningful economic analyses based on empiric information about cost and a range of subjective and objective outcomes are needed to minimise cost without compromising care. The objective of this study was to review and interpret the prescribing patterns of combination analgesics and the cost associated with their usage for the period 2001-2006 in a section of the private healthcare sector in South Africa. This research can be classified as a quantitative, retrospective drug utilisation review study. Data were obtained from a medicine claims database, and the study population consisted of all combination analgesic prescriptions (Mims® category 3.3) for the period 1 January 2001 to 31 December 2002 and 1 January 2004 to 31 December 2006. Prescribing Patterns of Combination Analgesics in the Private Health Care Sector. Firstly pain and the treatment thereof with combination analgesics were investigated from the literature to understand the disease and to determine the prevalence and treatment thereof. Secondly, managed health care, drug utilisation review, pharmacoeconomics and pharmaco-epidemiology were investigated from the literature to understand these concepts. The influence of the South African government on the medicine pricing regulations was discussed. Thirdly, through the empirical investigation the utilisation patterns of combination analgesics were reviewed, analysed and interpreted. It was determined that combination analgesic drugs represented 8.87% (n=261 907) of all medicine claimed during 2001 (N=2 951 326), decreased to 7.20% (n=381 809) during 2004 (N=5 305 846) after which it increased to 7.92% (n=187 745) in 2006 (N=2 370 572). Between 2001 (N=R379 708 489.00) and 2006 (N=R279 160 832.00) the cost percentage of the combination analgesic drugs decreased from 4.95% (n=R18 798 202.42) to 3.15% (n=R8 791 228.57). The average cost per combination analgesic drugs decreased from R71.77 ± 61.67 to R46.83 ± 43.41 between 2001 and 2006. This decrease was of no practical significance (d<0.8). The average number of combination analgesics per prescription stayed relatively constant varying between 1.01 ± 0.11 in 2001 and 1.02 ± 0.13 in 2006. The percentage generic combination analgesic drugs claimed increased from 29.63% (n=77 608) in 2001 to 66.37% (n=124 600) in 2006 (N=261 907 for 2001 and N=187 745 for 2006) even though generic medicine items claimed by the total database only increased from 26.79% (n=790 548) in 2001 to 40.27% (n=954 561) during 2006 (N=2 951 326 for 2001 and N=2 370 572 for 2006). The combination of ibuprofen 200mg, paracetamol 250mg and codeine phosphate 10mg (e.g. Myprodol® capsules, Mybulen® capsules, Gen-payne® capsules and Ibupain Forte® capsules) represented the active ingredient combination with the highest prevalence for the entire study period, increasing from 28.44% (n=74 483) in 2001 to 33.08% (n=62 100) in 2006 of all combination analgesics prescribed (N=261 907 for 2001 and N=187 745 for 2006). Generic substitution influenced the prevalence of the innovator medicine item, Myprodol® Capsules dramatically, causing a decrease from 23.16% (n=60 631) in 2001 to 3.77% (n=7 084) in 2006 representation of all combination analgesic prescribed. In 2006, the generics of Myprodol® Capsules e.g. Dentopain Forte®, Mybulen® Capsules, Gen-payne® and Ibupain Forte® represented 23.79% (n=44651) of all combination analgesics claimed. Recommendations were derived regarding certain aspects of the clinical and economical management of pain e.g. the implication of generic substitution with regard to cost and prescribing patterns, and the decreasing cost of combination analgesics which might encourage abuse, needs further investigation. South African prescribers have a large choice of combination analgesic preparations available for prescribing. According to Desmeules et al. (2003:8) the advantages of combining analgesics include increasing the duration of analgesia, widening the spectrum of efficacy, improved patient compliance and reduced parenteral abuse potential. According to McMahon (1975:13) one of the principle arguments against fixed-dose combinations is that the physician surrenders flexibility in managing his patient. Combination analgesics may expose patients to ingredients not necessary for pain relief in their particular condition (Beaver, 1984). Rigas (1997:454) explains that the value of pharmaco-economics in providing cost-effective pharmacologic treatment for pain must not only be seen as a containment effort, but rather as a valuation effort. Meaningful economic analyses based on empiric information about cost and a range of subjective and objective outcomes are needed to minimise cost without compromising care. The objective of this study was to review and interpret the prescribing patterns of combination analgesics and the cost associated with their usage for the period 2001-2006 in a section of the private healthcare sector in South Africa. This research can be classified as a quantitative, retrospective drug utilisation review study. Data were obtained from a medicine claims database, and the study population consisted of all combination analgesic prescriptions (Mims® category 3.3) for the period 1 January 2001 to 31 December 2002 and 1 January 2004 to 31 December 2006. Prescribing Patterns of Combination Analgesics in the Private Health Care Sector. Firstly pain and the treatment thereof with combination analgesics were investigated from the literature to understand the disease and to determine the prevalence and treatment thereof. Secondly, managed health care, drug utilisation review, pharmacoeconomics and pharmaco-epidemiology were investigated from the literature to understand these concepts. The influence of the South African government on the medicine pricing regulations was discussed. Thirdly, through the empirical investigation the utilisation patterns of combination analgesics were reviewed, analysed and interpreted. It was determined that combination analgesic drugs represented 8.87% (n=261 907) of all medicine claimed during 2001 (N=2 951 326), decreased to 7.20% (n=381 809) during 2004 (N=5 305 846) after which it increased to 7.92% (n=187 745) in 2006 (N=2 370 572). Between 2001 (N=R379 708 489.00) and 2006 (N=R279 160 832.00) the cost percentage of the combination analgesic drugs decreased from 4.95% (n=R18 798 202.42) to 3.15% (n=R8 791 228.57). The average cost per combination analgesic drugs decreased from R71.77 ± 61.67 to R46.83 ± 43.41 between 2001 and 2006. This decrease was of no practical significance (d<0.8). The average number of combination analgesics per prescription stayed relatively constant varying between 1.01 ± 0.11 in 2001 and 1.02 ± 0.13 in 2006. The percentage generic combination analgesic drugs claimed increased from 29.63% (n=77 608) in 2001 to 66.37% (n=124 600) in 2006 (N=261 907 for 2001 and N=187 745 for 2006) even though generic medicine items claimed by the total database only increased from 26.79% (n=790 548) in 2001 to 40.27% (n=954 561) during 2006 (N=2 951 326 for 2001 and N=2 370 572 for 2006). The combination of ibuprofen 200mg, paracetamol 250mg and codeine phosphate 10mg (e.g. Myprodol® capsules, Mybulen® capsules, Gen-payne® capsules and Ibupain Forte® capsules) represented the active ingredient combination with the highest prevalence for the entire study period, increasing from 28.44% (n=74 483) in 2001 to 33.08% (n=62 100) in 2006 of all combination analgesics prescribed (N=261 907 for 2001 and N=187 745 for 2006). Generic substitution influenced the prevalence of the innovator medicine item, Myprodol® Capsules dramatically, causing a decrease from 23.16% (n=60 631) in 2001 to 3.77% (n=7 084) in 2006 representation of all combination analgesic prescribed. In 2006, the generics of Myprodol® Capsules e.g. Dentopain Forte®, Mybulen® Capsules, Gen-payne® and Ibupain Forte® represented 23.79% (n=44651) of all combination analgesics claimed. Recommendations were derived regarding certain aspects of the clinical and economical management of pain e.g. the implication of generic substitution with regard to cost and prescribing patterns, and the decreasing cost of combination analgesics which might encourage abuse, needs further investigation. / Thesis (M.Pharm. (Pharmacy Practice))--North-West University, Potchefstroom Campus, 2008.

Thesis (M. Pharm.)--North-West University, Potchefstroom Campus, 2008.

Introdução: As mucopolisaccaridoses tipo I (MPS I), tipo II (MPS II) e tipo VI (MPS VI) são doenças lisossômicas (DL) para as quais está disponível a terapia de reposição enzimática (TRE) com laronidase, idursulfase e galsufase, respectivamente. Objetivo Primário: Analisar a frequência anual de intervenções médicas (número de consultas, internações, cirurgias, exames solicitados, medicamentos prescritos, equipamentos de uso crônico e outras formas de terapia) em uma amostra de pacientes brasileiros com MPS I, II e VI e, desta forma, contribuir para o conhecimento dos aspectos farmacoeconômicos relacionados a essas doenças. Metodologia: Estudo exploratório, retrospectivo, de base hospitalar, baseado em revisão de prontuário, com amostragem por conveniência, e que foi realizado em duas etapas (etapas 1 e 2). Um instrumento específico para a coleta de dados de ambas as etapas foi construído pela equipe do estudo, que é multidisciplinar. Os desfechos de interesse foram as frequências anuais de intervenções médicas (consultas, exames, cirurgias, internações, medicamentos utilizados, outras formas de terapia). A etapa 1 consistiu em estudo pré-experimental, realizado no Serviço de Genética Médica do Hospital de Clínicas de Porto Alegre (SGM-HCPA), e que comparou as variáveis de interesse, para o mesmo grupo de pacientes, entre o período pré e pós-TRE. Os critérios de inclusão dessa etapa foram: ter diagnóstico confirmado de MPS I; estar em acompanhamento regular no SGM-HCPA desde o diagnóstico; estar em TRE por pelo menos um ano; e não ter participado de ensaio clínico envolvendo TRE ou ter realizado transplante de células-tronco hematopoiéticas. A etapa 2 foi transversal, multicêntrica (centros incluídos: SGMHCPA, Departamento de Genética Médica da Universidade Estadual de Campinas - UNICAMP, Pontifícia Universidade Católica de Campinas – PUC-Campinas, e Departamento de Pediatria da Universidade Estadual do Rio de Janeiro - UERJ), e comparou as variáveis de interesse entre grupos diferentes de pacientes (aqueles recebendo TRE e aqueles não recebendo TRE). Para essa etapa, foram considerados somente os dados relativos a 2010, sendo os seguintes os critérios de inclusão dos pacientes: ter diagnóstico confirmado de MPS I, II e VI; não estar participando de nenhum ensaio clínico envolvendo TRE ou ter realizado transplante de células-tronco hematopoiéticas; estar em TRE por pelo menos 12 meses antes do início da coleta, ou em acompanhamento por pelo 12 meses antes do início da coleta. Resultados: Etapa 1 - Nove pacientes (graves=3; atenuados=6) com MPS I foram incluídos no estudo, com mediana de idade de diagnóstico de 4,4 anos. Somente o número de cirurgias/ano/paciente foi dependente do tempo de doença (p=0,0004) e da gravidade do fenótipo (p=0,014). Com relação às comparações pré e pós-TRE, as variáveis que apresentaram diferença significativa (média do número/ano/paciente) foram: exames (pré-TRE=10,2+2,7; pós-TRE=22,5+2,1; p=0,005) e internações (pré-TRE=0,05+0,04; pós-TRE=0,30+0,11; p=0,013). Para as demais variáveis, não foi encontrada associação. Etapa 2 - Trinta e quatro pacientes com MPS I (n=12), II (n=17) e VI (n=5) foram incluídos no estudo. Desses, sete não utilizavam TRE (grupo “sem TRE") e 27 faziam uso de tratamento específico (grupo “com TRE"). Não foi encontrada correlação significativa entre tempo de doença e as variáveis estudadas. Considerando a amostra total, foi encontrada diferença entre o grupo “sem TRE” e o grupo “com TRE” em relação à mediana de internações hospitalares e de cirurgias realizadas [1(0-2) vs. 0 (0-1), p=0,015; e 0 (0-2) vs. 0 (0-0), p=0,040, respectivamente]. Para as crianças/adolescentes (<18 anos), não foi encontrada diferença estatística entre os grupos. Os pacientes com comprometimento cognitivo utilizavam mais medicamentos que os demais (p=0,024). Encontrou-se correlação negativa entre as variáveis duração da TRE e número anual de internações (r= -0,504; p=0,007). Discussão/ Conclusões: Este é um dos primeiros estudos a avaliar aspectos relacionados à farmaconomia da TRE para as MPS. De acordo com os resultados obtidos na etapa 2, verifica-se que, desconsiderando-se o custo associado às infusões, o custo do tratamento de pacientes com MPS parece ser menor para aqueles pacientes que utilizam a TRE do que para os pacientes que fazem somente tratamento sintomático. Entretanto, de acordo com a etapa 1 do estudo, a TRE parece não impedir a evolução da doença, pelo menos em relação à MPS I, e, assim, a cada ano de vida do paciente ocorreria um incremento do custo associado ao tratamento. Estudos adicionais, com maior tamanho amostral, deverão ser realizados para confirmar nossos achados. / Introduction: The mucopolysaccharidoses type I (MPS I), II (MPS II) and VI (MPS VI) are lysosomal disorders (LSD) for which enzyme replacement therapy (ERT) with laronidase, idursulfase and galsulfase, respectively, are available. Principal objective: To analyze the annual frequency of medical interventions (number of medical appointments, hospital admissions, surgical procedures, exams performed, medications prescribed, ancillary therapies and the use of medical devices) in a sample of Brazilian patients with MPS I, II and VI, and thus, contribute to the understanding of some pharmacoeconomic aspects related to these diseases. Methodology: Retrospective, exploratory, hospital-based study, based on medical records review, with convenience sampling, which was conducted in two steps (steps 1 and 2). A specific data collection instrument for both steps was designed by the study team, which is multidisciplinary. The chosen outcomes were: annual frequencies of medical interventions (medical appointments, exams, surgical procedures, hospital admissions, medications used and ancillary therapies). Step 1 was a pre-experimental study conducted at the Medical Genetics Service of Hospital de Clínicas de Porto Alegre (SGM-HCPA), and compared the variables of interest between the pre and post-ERT periods for the same group of patients. The patient inclusion criteria were: a biochemical diagnosis of MPS I and regular follow-up at SGM-HCPA since diagnosis; ERT for at least 1 year; no enrollment in any clinical trials involving ERT, and no history of hematopoietic stem cell transplantation. Step 2 was a cross-sectional and multicentric estudy (Centers included: SGM-HCPA), the Department of Medical Genetics of Universidade Estadual de Campinas - UNICAMP, Pontifícia Universidade Católica de Campinas - PUC-Campinas, and the Department of Pediatrics at Universidade Estadual do Rio de Janeiro – UERJ, which compared the variables of interest between different groups of patients (those receiving and those not receiving ERT). For this step only data from 2010 were considered. The inclusion patient criteria were: a biochemical diagnosis of MPS I, II or VI; no enrollment in any clinical trials involving ERT, and no history of hematopoietic stem cell transplantation, to be on ERT for at least 12 months before the start of data collection or to undergo regular follow-up for at least 12 months before the start of data collection. Results: Step 1 – Nine MPS I patients (severe=3; attenuated phenotype=6) were included in the study with median age at diagnosis was 4.4 years. Only the number/year/patient of surgeries was found to be dependent on length of disease (p=0.0004) and on severity of phenotype (p=0.014). Regarding pre- and post-ERT comparisons, the variables for which a significant difference was detected (mean number/year/patient) were exams (pre-ERT, 10.2±2.7; post-ERT, 22.5±2.1; p=0.005) and hospital admissions (pre-ERT, 0.05±0.04; post-ERT, 0.30±0.11; p=0.013). For the other variables, no association was found. Step 2: Thirty-four patients with MPS I, II and VI were included (I=12, II=17, VI=5). From them, 27 on ERT (“ERT group”) and 7 receiving supportive care only (“non-ERT group”). There were no significant correlation between length of disease and any of the variables of interest. There were significant between-group differences in the median number of hospital admissions and surgical procedures, both of which were higher in the non-ERT group [1(0-2) vs. 0 (0-1), p=0,015; e 0 (0-2) vs. 0 (0-0), p=0,040, respectively]. There were no significant between-group differences when only children and adolescents (<18 years) were taken into account. Patients with cognitive involvement used more medications than the others (p=0.024). A correlation was detected between time on ERT and the hospital admissions variable (r= -0.504; p=0.007). Discussion/conclusions: This was one of the first studies to evaluate aspects related to pharmacoeconomics of ERT for MPS. According to the results of step 2, and not acknowledging the costs associated with recombinant enzyme infusions, patients with MPS who undergo ERT generate less cost to SUS than patients on symptomatic treatment. On the other hand, according to the results of step 1, ERT seems not to stop the disease progress, at least in respect to MPS I, and thus, for each year of a patient life occurred an increase in cost associated with treatment. Additional studies with larger sample size are needed to confirm our findings.

A albumina humana (AH) é um medicamento de ampla utilização e de custo elevado que integra o elenco de procedimentos especiais do Ministério da Saúde no Brasil. Em 2004, a ANVISA publicou uma resolução que estabelece diretrizes para o uso terapêutico deste medicamento. Apesar dos dados da literatura sugerirem conclusões não definitivas sobre os reais benefícios do uso de albumina no tratamento de pacientes com síndrome nefrótica (SN), a diretriz recomenda o uso nos casos de edemas refratários ao uso de diuréticos. O objetivo deste estudo foi avaliar através de análises econômicas a indicação clinica da diretriz para uso de Albumina Humana da ANVISA, em pacientes com Síndrome Nefrótica. A partir de uma coorte concorrente foram realizadas análises econômicas dos tipos custo-efetividade e custoutilidade. A população do estudo foi constituída por pacientes adultos e pediátricos internados com síndrome nefrótica em quatro grandes hospitais do SUS do Estado da Bahia. Os pacientes incluídos foram divididos em dois grupos comparativos conforme o seguimento ou não das orientações de uso de albumina humana da diretriz da ANVISA. A perspectiva da análise econômica foi sobre o provedor de atenção à saúde, o SUS. Os dados de qualidade de vida dos pacientes foram obtidos através de aplicação dos questionários genéricos SF36 e CHQ-PF50. O índice QALY (anos de vida ajustados à qualidade de vida) foi utilizado para a avaliação de custo-utilidade da comparação. Ao final do estudo foram selecionados 109 pacientes, com 60% de adultos, 56% do gênero feminino, mais de 90% de negros e pardos tendo 41,3% seguido as diretrizes da ANVISA. O custo médio global por internação (média de 22 dias) para o SUS foi de R$ 2.360/paciente. Os parâmetros de efetividade analisados, peso, diurese e balanço hídrico foram mais efetivos em termos de custo para os pacientes que seguiram a diretriz. A RCEI para dias de internação evitados evidenciou um custo adicional de R$55,33/dia evitado. A análise de qualidade de vida demonstrou que seguir a diretriz dominou o não seguimento propiciando um acréscimo de 8% de qualidade de vida e gerando uma economia de R$ 3.458,13 por QALY ganho. As análises de sensibilidade das RCEI e RCUI evidenciaram a robustez dos resultados, principalmente na variação do QALY, que mesmo após 60% de variabilidade garantiu economia para o sistema. Os resultados deste estudo forneceram informações sobre o uso de albumina humana em pacientes com síndrome nefrótica que podem juntamente com outros dados nortear as práticas de saúde no SUS do ponto de vista clinico e econômico. As análises econômicas indicaram que apresentam melhores relações de custo-efetividade e custo utilidade os tratamentos dos pacientes que seguiram as orientações da diretriz da ANVISA. / Human albumin (HA) is a drug widely used and of high cost that integrates the cast of special procedures of the Ministry of Health in Brazil. In 2004, ANVISA issued a resolution establishing guidelines for the therapeutic use of this drug. Although data from the literature suggest no definite conclusions about the real benefits of the use of albumin in the treatment of patients with nephrotic syndrome (NS), the guideline recommends the use in cases of edema refractory to diuretics. The aim of this study was to evaluate economic analyzes through the clinical indication of guideline for use of Human Albumin ANVISA, in patients with nephrotic syndrome. Economic analyzes of both cost-effectiveness and cost-utility types were performed from a concurrent cohort. The study population was composed of adult and pediatric patients hospitalized with nephrotic syndrome in four major public hospitals in the State of Bahia. The included patients were divided into two distinct groups according to whether ANVISA’s guidelines for use of human albumin were followed by them or not. The economic perspective of the analysis regarded the health care provider, the NHS. The quality of life data of the patients were obtained through application of generic questionnaires SF36 and CHQ - PF50. The QALY index (quality-adjusted life year) was used to assess cost-utility comparison. At the end of the study 109 patients were selected, consisting of 60% adults, 56 % females, over 80 % blacks and dark-skinned, where 41.3 % followed the guidelines of ANVISA. The overall average cost per hospitalization (22 days average) to the NHS was R$ 2.360/patient. The analyzed parameters, weight, urine volume, and fluid balance were more effective in terms of cost for patients who followed the guidelines. The ICER for days of hospitalization avoided showed an additional cost of R$55.33/avoided day. The quality of life analysis showed that the majority followed the guidelines providing an increase of 8 % for quality of life and generating savings of R$ 3,458.13 per QALY gained. Sensitivity analyzes of the ICER and ICUR showed the robustness of the results, especially the variation of the QALY, which even after 60% of variability guaranteed savings for the system. The results of this study provided information on the use of human albumin in patients with nephrotic syndrome that can, along with other data, guide health care practices in the NHS from both clinical and financial standpoints. The economic analyzes that show better the costeffectiveness and cost utility of treatment patients who followed the recommendations the guideline of ANVISA.

Introdução: As mucopolisaccaridoses tipo I (MPS I), tipo II (MPS II) e tipo VI (MPS VI) são doenças lisossômicas (DL) para as quais está disponível a terapia de reposição enzimática (TRE) com laronidase, idursulfase e galsufase, respectivamente. Objetivo Primário: Analisar a frequência anual de intervenções médicas (número de consultas, internações, cirurgias, exames solicitados, medicamentos prescritos, equipamentos de uso crônico e outras formas de terapia) em uma amostra de pacientes brasileiros com MPS I, II e VI e, desta forma, contribuir para o conhecimento dos aspectos farmacoeconômicos relacionados a essas doenças. Metodologia: Estudo exploratório, retrospectivo, de base hospitalar, baseado em revisão de prontuário, com amostragem por conveniência, e que foi realizado em duas etapas (etapas 1 e 2). Um instrumento específico para a coleta de dados de ambas as etapas foi construído pela equipe do estudo, que é multidisciplinar. Os desfechos de interesse foram as frequências anuais de intervenções médicas (consultas, exames, cirurgias, internações, medicamentos utilizados, outras formas de terapia). A etapa 1 consistiu em estudo pré-experimental, realizado no Serviço de Genética Médica do Hospital de Clínicas de Porto Alegre (SGM-HCPA), e que comparou as variáveis de interesse, para o mesmo grupo de pacientes, entre o período pré e pós-TRE. Os critérios de inclusão dessa etapa foram: ter diagnóstico confirmado de MPS I; estar em acompanhamento regular no SGM-HCPA desde o diagnóstico; estar em TRE por pelo menos um ano; e não ter participado de ensaio clínico envolvendo TRE ou ter realizado transplante de células-tronco hematopoiéticas. A etapa 2 foi transversal, multicêntrica (centros incluídos: SGMHCPA, Departamento de Genética Médica da Universidade Estadual de Campinas - UNICAMP, Pontifícia Universidade Católica de Campinas – PUC-Campinas, e Departamento de Pediatria da Universidade Estadual do Rio de Janeiro - UERJ), e comparou as variáveis de interesse entre grupos diferentes de pacientes (aqueles recebendo TRE e aqueles não recebendo TRE). Para essa etapa, foram considerados somente os dados relativos a 2010, sendo os seguintes os critérios de inclusão dos pacientes: ter diagnóstico confirmado de MPS I, II e VI; não estar participando de nenhum ensaio clínico envolvendo TRE ou ter realizado transplante de células-tronco hematopoiéticas; estar em TRE por pelo menos 12 meses antes do início da coleta, ou em acompanhamento por pelo 12 meses antes do início da coleta. Resultados: Etapa 1 - Nove pacientes (graves=3; atenuados=6) com MPS I foram incluídos no estudo, com mediana de idade de diagnóstico de 4,4 anos. Somente o número de cirurgias/ano/paciente foi dependente do tempo de doença (p=0,0004) e da gravidade do fenótipo (p=0,014). Com relação às comparações pré e pós-TRE, as variáveis que apresentaram diferença significativa (média do número/ano/paciente) foram: exames (pré-TRE=10,2+2,7; pós-TRE=22,5+2,1; p=0,005) e internações (pré-TRE=0,05+0,04; pós-TRE=0,30+0,11; p=0,013). Para as demais variáveis, não foi encontrada associação. Etapa 2 - Trinta e quatro pacientes com MPS I (n=12), II (n=17) e VI (n=5) foram incluídos no estudo. Desses, sete não utilizavam TRE (grupo “sem TRE") e 27 faziam uso de tratamento específico (grupo “com TRE"). Não foi encontrada correlação significativa entre tempo de doença e as variáveis estudadas. Considerando a amostra total, foi encontrada diferença entre o grupo “sem TRE” e o grupo “com TRE” em relação à mediana de internações hospitalares e de cirurgias realizadas [1(0-2) vs. 0 (0-1), p=0,015; e 0 (0-2) vs. 0 (0-0), p=0,040, respectivamente]. Para as crianças/adolescentes (<18 anos), não foi encontrada diferença estatística entre os grupos. Os pacientes com comprometimento cognitivo utilizavam mais medicamentos que os demais (p=0,024). Encontrou-se correlação negativa entre as variáveis duração da TRE e número anual de internações (r= -0,504; p=0,007). Discussão/ Conclusões: Este é um dos primeiros estudos a avaliar aspectos relacionados à farmaconomia da TRE para as MPS. De acordo com os resultados obtidos na etapa 2, verifica-se que, desconsiderando-se o custo associado às infusões, o custo do tratamento de pacientes com MPS parece ser menor para aqueles pacientes que utilizam a TRE do que para os pacientes que fazem somente tratamento sintomático. Entretanto, de acordo com a etapa 1 do estudo, a TRE parece não impedir a evolução da doença, pelo menos em relação à MPS I, e, assim, a cada ano de vida do paciente ocorreria um incremento do custo associado ao tratamento. Estudos adicionais, com maior tamanho amostral, deverão ser realizados para confirmar nossos achados. / Introduction: The mucopolysaccharidoses type I (MPS I), II (MPS II) and VI (MPS VI) are lysosomal disorders (LSD) for which enzyme replacement therapy (ERT) with laronidase, idursulfase and galsulfase, respectively, are available. Principal objective: To analyze the annual frequency of medical interventions (number of medical appointments, hospital admissions, surgical procedures, exams performed, medications prescribed, ancillary therapies and the use of medical devices) in a sample of Brazilian patients with MPS I, II and VI, and thus, contribute to the understanding of some pharmacoeconomic aspects related to these diseases. Methodology: Retrospective, exploratory, hospital-based study, based on medical records review, with convenience sampling, which was conducted in two steps (steps 1 and 2). A specific data collection instrument for both steps was designed by the study team, which is multidisciplinary. The chosen outcomes were: annual frequencies of medical interventions (medical appointments, exams, surgical procedures, hospital admissions, medications used and ancillary therapies). Step 1 was a pre-experimental study conducted at the Medical Genetics Service of Hospital de Clínicas de Porto Alegre (SGM-HCPA), and compared the variables of interest between the pre and post-ERT periods for the same group of patients. The patient inclusion criteria were: a biochemical diagnosis of MPS I and regular follow-up at SGM-HCPA since diagnosis; ERT for at least 1 year; no enrollment in any clinical trials involving ERT, and no history of hematopoietic stem cell transplantation. Step 2 was a cross-sectional and multicentric estudy (Centers included: SGM-HCPA), the Department of Medical Genetics of Universidade Estadual de Campinas - UNICAMP, Pontifícia Universidade Católica de Campinas - PUC-Campinas, and the Department of Pediatrics at Universidade Estadual do Rio de Janeiro – UERJ, which compared the variables of interest between different groups of patients (those receiving and those not receiving ERT). For this step only data from 2010 were considered. The inclusion patient criteria were: a biochemical diagnosis of MPS I, II or VI; no enrollment in any clinical trials involving ERT, and no history of hematopoietic stem cell transplantation, to be on ERT for at least 12 months before the start of data collection or to undergo regular follow-up for at least 12 months before the start of data collection. Results: Step 1 – Nine MPS I patients (severe=3; attenuated phenotype=6) were included in the study with median age at diagnosis was 4.4 years. Only the number/year/patient of surgeries was found to be dependent on length of disease (p=0.0004) and on severity of phenotype (p=0.014). Regarding pre- and post-ERT comparisons, the variables for which a significant difference was detected (mean number/year/patient) were exams (pre-ERT, 10.2±2.7; post-ERT, 22.5±2.1; p=0.005) and hospital admissions (pre-ERT, 0.05±0.04; post-ERT, 0.30±0.11; p=0.013). For the other variables, no association was found. Step 2: Thirty-four patients with MPS I, II and VI were included (I=12, II=17, VI=5). From them, 27 on ERT (“ERT group”) and 7 receiving supportive care only (“non-ERT group”). There were no significant correlation between length of disease and any of the variables of interest. There were significant between-group differences in the median number of hospital admissions and surgical procedures, both of which were higher in the non-ERT group [1(0-2) vs. 0 (0-1), p=0,015; e 0 (0-2) vs. 0 (0-0), p=0,040, respectively]. There were no significant between-group differences when only children and adolescents (<18 years) were taken into account. Patients with cognitive involvement used more medications than the others (p=0.024). A correlation was detected between time on ERT and the hospital admissions variable (r= -0.504; p=0.007). Discussion/conclusions: This was one of the first studies to evaluate aspects related to pharmacoeconomics of ERT for MPS. According to the results of step 2, and not acknowledging the costs associated with recombinant enzyme infusions, patients with MPS who undergo ERT generate less cost to SUS than patients on symptomatic treatment. On the other hand, according to the results of step 1, ERT seems not to stop the disease progress, at least in respect to MPS I, and thus, for each year of a patient life occurred an increase in cost associated with treatment. Additional studies with larger sample size are needed to confirm our findings.

A albumina humana (AH) é um medicamento de ampla utilização e de custo elevado que integra o elenco de procedimentos especiais do Ministério da Saúde no Brasil. Em 2004, a ANVISA publicou uma resolução que estabelece diretrizes para o uso terapêutico deste medicamento. Apesar dos dados da literatura sugerirem conclusões não definitivas sobre os reais benefícios do uso de albumina no tratamento de pacientes com síndrome nefrótica (SN), a diretriz recomenda o uso nos casos de edemas refratários ao uso de diuréticos. O objetivo deste estudo foi avaliar através de análises econômicas a indicação clinica da diretriz para uso de Albumina Humana da ANVISA, em pacientes com Síndrome Nefrótica. A partir de uma coorte concorrente foram realizadas análises econômicas dos tipos custo-efetividade e custoutilidade. A população do estudo foi constituída por pacientes adultos e pediátricos internados com síndrome nefrótica em quatro grandes hospitais do SUS do Estado da Bahia. Os pacientes incluídos foram divididos em dois grupos comparativos conforme o seguimento ou não das orientações de uso de albumina humana da diretriz da ANVISA. A perspectiva da análise econômica foi sobre o provedor de atenção à saúde, o SUS. Os dados de qualidade de vida dos pacientes foram obtidos através de aplicação dos questionários genéricos SF36 e CHQ-PF50. O índice QALY (anos de vida ajustados à qualidade de vida) foi utilizado para a avaliação de custo-utilidade da comparação. Ao final do estudo foram selecionados 109 pacientes, com 60% de adultos, 56% do gênero feminino, mais de 90% de negros e pardos tendo 41,3% seguido as diretrizes da ANVISA. O custo médio global por internação (média de 22 dias) para o SUS foi de R$ 2.360/paciente. Os parâmetros de efetividade analisados, peso, diurese e balanço hídrico foram mais efetivos em termos de custo para os pacientes que seguiram a diretriz. A RCEI para dias de internação evitados evidenciou um custo adicional de R$55,33/dia evitado. A análise de qualidade de vida demonstrou que seguir a diretriz dominou o não seguimento propiciando um acréscimo de 8% de qualidade de vida e gerando uma economia de R$ 3.458,13 por QALY ganho. As análises de sensibilidade das RCEI e RCUI evidenciaram a robustez dos resultados, principalmente na variação do QALY, que mesmo após 60% de variabilidade garantiu economia para o sistema. Os resultados deste estudo forneceram informações sobre o uso de albumina humana em pacientes com síndrome nefrótica que podem juntamente com outros dados nortear as práticas de saúde no SUS do ponto de vista clinico e econômico. As análises econômicas indicaram que apresentam melhores relações de custo-efetividade e custo utilidade os tratamentos dos pacientes que seguiram as orientações da diretriz da ANVISA. / Human albumin (HA) is a drug widely used and of high cost that integrates the cast of special procedures of the Ministry of Health in Brazil. In 2004, ANVISA issued a resolution establishing guidelines for the therapeutic use of this drug. Although data from the literature suggest no definite conclusions about the real benefits of the use of albumin in the treatment of patients with nephrotic syndrome (NS), the guideline recommends the use in cases of edema refractory to diuretics. The aim of this study was to evaluate economic analyzes through the clinical indication of guideline for use of Human Albumin ANVISA, in patients with nephrotic syndrome. Economic analyzes of both cost-effectiveness and cost-utility types were performed from a concurrent cohort. The study population was composed of adult and pediatric patients hospitalized with nephrotic syndrome in four major public hospitals in the State of Bahia. The included patients were divided into two distinct groups according to whether ANVISA’s guidelines for use of human albumin were followed by them or not. The economic perspective of the analysis regarded the health care provider, the NHS. The quality of life data of the patients were obtained through application of generic questionnaires SF36 and CHQ - PF50. The QALY index (quality-adjusted life year) was used to assess cost-utility comparison. At the end of the study 109 patients were selected, consisting of 60% adults, 56 % females, over 80 % blacks and dark-skinned, where 41.3 % followed the guidelines of ANVISA. The overall average cost per hospitalization (22 days average) to the NHS was R$ 2.360/patient. The analyzed parameters, weight, urine volume, and fluid balance were more effective in terms of cost for patients who followed the guidelines. The ICER for days of hospitalization avoided showed an additional cost of R$55.33/avoided day. The quality of life analysis showed that the majority followed the guidelines providing an increase of 8 % for quality of life and generating savings of R$ 3,458.13 per QALY gained. Sensitivity analyzes of the ICER and ICUR showed the robustness of the results, especially the variation of the QALY, which even after 60% of variability guaranteed savings for the system. The results of this study provided information on the use of human albumin in patients with nephrotic syndrome that can, along with other data, guide health care practices in the NHS from both clinical and financial standpoints. The economic analyzes that show better the costeffectiveness and cost utility of treatment patients who followed the recommendations the guideline of ANVISA.

A albumina humana (AH) é um medicamento de ampla utilização e de custo elevado que integra o elenco de procedimentos especiais do Ministério da Saúde no Brasil. Em 2004, a ANVISA publicou uma resolução que estabelece diretrizes para o uso terapêutico deste medicamento. Apesar dos dados da literatura sugerirem conclusões não definitivas sobre os reais benefícios do uso de albumina no tratamento de pacientes com síndrome nefrótica (SN), a diretriz recomenda o uso nos casos de edemas refratários ao uso de diuréticos. O objetivo deste estudo foi avaliar através de análises econômicas a indicação clinica da diretriz para uso de Albumina Humana da ANVISA, em pacientes com Síndrome Nefrótica. A partir de uma coorte concorrente foram realizadas análises econômicas dos tipos custo-efetividade e custoutilidade. A população do estudo foi constituída por pacientes adultos e pediátricos internados com síndrome nefrótica em quatro grandes hospitais do SUS do Estado da Bahia. Os pacientes incluídos foram divididos em dois grupos comparativos conforme o seguimento ou não das orientações de uso de albumina humana da diretriz da ANVISA. A perspectiva da análise econômica foi sobre o provedor de atenção à saúde, o SUS. Os dados de qualidade de vida dos pacientes foram obtidos através de aplicação dos questionários genéricos SF36 e CHQ-PF50. O índice QALY (anos de vida ajustados à qualidade de vida) foi utilizado para a avaliação de custo-utilidade da comparação. Ao final do estudo foram selecionados 109 pacientes, com 60% de adultos, 56% do gênero feminino, mais de 90% de negros e pardos tendo 41,3% seguido as diretrizes da ANVISA. O custo médio global por internação (média de 22 dias) para o SUS foi de R$ 2.360/paciente. Os parâmetros de efetividade analisados, peso, diurese e balanço hídrico foram mais efetivos em termos de custo para os pacientes que seguiram a diretriz. A RCEI para dias de internação evitados evidenciou um custo adicional de R$55,33/dia evitado. A análise de qualidade de vida demonstrou que seguir a diretriz dominou o não seguimento propiciando um acréscimo de 8% de qualidade de vida e gerando uma economia de R$ 3.458,13 por QALY ganho. As análises de sensibilidade das RCEI e RCUI evidenciaram a robustez dos resultados, principalmente na variação do QALY, que mesmo após 60% de variabilidade garantiu economia para o sistema. Os resultados deste estudo forneceram informações sobre o uso de albumina humana em pacientes com síndrome nefrótica que podem juntamente com outros dados nortear as práticas de saúde no SUS do ponto de vista clinico e econômico. As análises econômicas indicaram que apresentam melhores relações de custo-efetividade e custo utilidade os tratamentos dos pacientes que seguiram as orientações da diretriz da ANVISA. / Human albumin (HA) is a drug widely used and of high cost that integrates the cast of special procedures of the Ministry of Health in Brazil. In 2004, ANVISA issued a resolution establishing guidelines for the therapeutic use of this drug. Although data from the literature suggest no definite conclusions about the real benefits of the use of albumin in the treatment of patients with nephrotic syndrome (NS), the guideline recommends the use in cases of edema refractory to diuretics. The aim of this study was to evaluate economic analyzes through the clinical indication of guideline for use of Human Albumin ANVISA, in patients with nephrotic syndrome. Economic analyzes of both cost-effectiveness and cost-utility types were performed from a concurrent cohort. The study population was composed of adult and pediatric patients hospitalized with nephrotic syndrome in four major public hospitals in the State of Bahia. The included patients were divided into two distinct groups according to whether ANVISA’s guidelines for use of human albumin were followed by them or not. The economic perspective of the analysis regarded the health care provider, the NHS. The quality of life data of the patients were obtained through application of generic questionnaires SF36 and CHQ - PF50. The QALY index (quality-adjusted life year) was used to assess cost-utility comparison. At the end of the study 109 patients were selected, consisting of 60% adults, 56 % females, over 80 % blacks and dark-skinned, where 41.3 % followed the guidelines of ANVISA. The overall average cost per hospitalization (22 days average) to the NHS was R$ 2.360/patient. The analyzed parameters, weight, urine volume, and fluid balance were more effective in terms of cost for patients who followed the guidelines. The ICER for days of hospitalization avoided showed an additional cost of R$55.33/avoided day. The quality of life analysis showed that the majority followed the guidelines providing an increase of 8 % for quality of life and generating savings of R$ 3,458.13 per QALY gained. Sensitivity analyzes of the ICER and ICUR showed the robustness of the results, especially the variation of the QALY, which even after 60% of variability guaranteed savings for the system. The results of this study provided information on the use of human albumin in patients with nephrotic syndrome that can, along with other data, guide health care practices in the NHS from both clinical and financial standpoints. The economic analyzes that show better the costeffectiveness and cost utility of treatment patients who followed the recommendations the guideline of ANVISA.