Estimated costs for Duchenne muscular dystrophy care in Brazil.

BACKGROUND: The economic burden of rare diseases on health systems is still not widely measured, with the generation of accurate information about the costs with medical care for subjects with rare diseases being crucial when defining health policies. Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy, with new technologies recently being studied for its management. Information about the costs related to the disease in Latin America is scarce, and the objective of this study is to evaluate the annual hospital, home care and transportation costs per patient with DMD treatment in Brazil. RESULTS: Data from 27 patients were included, the median annual cost per patient was R$ 17,121 (IQR R$ 6,786; 25,621). Home care expenditures accounted for 92% of the total costs, followed by hospital costs (6%) and transportation costs (2%). Medications and loss of family, and patient's productivity are among the most representative consumption items. When disease worsening due to loss of the ability to walk was incorporated to the analysis, it was shown that wheelchair users account for an incremental cost of 23% compared with non-wheelchair users. CONCLUSIONS: This is an original study in Latin America to measure DMD costs using the micro-costing technique. Generating accurate information about costs is crucial to provide health managers with information that could help establish more sustainable policies when deciding upon rare diseases in emerging countries.

Custo-efetividade e limiar de custo para o uso do sistema intrauterino liberador de levonorgestrel para o tratamento do sangramento uterino anormal sob a perspectiva do Sistema de Saúde Suplementar no Brasil / Cost-effectiveness and cost threshold for the use of levonorgestrel-releasing intrauterine system for treatment of heavy menstrual bleeding from the Supplementary Health System perspective in Brazil

Objetivo: Definir um limiar de custo para o sistema intrauterino liberador de levonorgestrel 52 mg (SIU-LNG 52 mg) para o tratamento do sangramento uterino anormal (SUA) sob a perspectiva do Sistema de Saúde Suplementar brasileiro. Métodos: Foi elaborado um modelo de custo-efetividade para definir o limiar de custo de inserção de SIU-LNG 52 mg em mulheres com SUA em comparação à histerectomia. Um modelo de Markov foi estruturado com ciclos anuais para reproduzir o tratamento do SUA em um e cinco anos, considerando custos médicos diretos e o percentual de histerectomias evitadas como desfecho. O custo da histerectomia foi variado até o valor de 10.000 reais brasileiros (BRL) com incrementos de 500 BRL a cada nova simulação para avaliar o limiar de custo do SIU-LNG 52 mg para igualar o custo total de tratamento de ambas as estratégias analisadas. Resultados: O SIU-LNG 52 mg demonstrou ser uma opção de tratamento dominante quando comparada à histerectomia, levando à redução da frequência de realização do procedimento cirúrgico em 59,62% das mulheres e redução de 2.557,91 BRL no custo total de tratamento em cinco anos. Ao considerar a histerectomia a 6.000 BRL, o custo do procedimento com SIU-LNG 52 mg poderia ser de até 6.150,35 BRL e 3.994,60 BRL para igualar o custo total de tratamento, em horizontes temporais de um e cinco anos, respectivamente. Conclusão: SIU-LNG 52 mg demonstrou ser uma opção dominante para o manejo do SUA, sendo capaz de atrelar economia para o sistema de saúde a benefícios para a mulher perante a cirurgia.

Objective: To define a cost threshold for the use of levonorgestrel-releasing intrauterine system 52 mg (LNG-IUS 52 mg) for the treatment of heavy menstrual bleeding (HMB) in the Brazilian Supplementary Health System perspective. Methods: A cost-effectiveness model was built to define the cost threshold for insertion of LNG-IUS 52 mg considering women with diagnosis of HMB as the target population and hysterectomy as the comparator. A Markov model was structured with annual cycles to reproduce HMB treatment in 1 and 5 years, considering direct medical costs and the percentage of avoided hysterectomies as the outcome. Hysterectomy cost was varied up to 10,000 Brazilian real (BRL) with increments of 500 BRL at each new simulation to define LNG-IUS 52 mg cost threshold, to provide equal total treatment costs for both strategies. Results: LNG-IUS 52 mg proved to be a cost-saving option when compared to hysterectomy, leading to a reduction in the frequency of the surgical procedure by 59.62% and a total treatment cost reduction of 2,557.91 BRL in 5 years. When considering hysterectomy at 6,000 BRL, the cost of the procedure with LNG-IUS 52 mg could be up to 6,150.35 BRL and 3,994.60 BRL to provide equal total treatment cost in 1 and 5 years time horizon, respectively. Conclusion: LNG-IUS 52 mg has proven to be a cost-saving option for the health system in the management of HMB, in addition to the known benefits for women against surgery.