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.

Statements about hemophilia A in Brazil: an expert Delphi panel / Consensos em hemofilia A no Brasil: painel Delphi de especialistas

Objective: Understanding unmet needs related to hemophilia A management in Brazil is critical for supporting decision-making. Methods: A modified Delphi consensus panel was conducted. Hematologists with extensive experience treating hemophilia in the Brazilian Public Health System were invited to answer questions regarding indicators of severe hemophilia prophylaxis effectiveness, emicizumab treatment indications, and bypassing agents used to reduce bleeding in patients with inhibitors, immune tolerance induction (ITI) use, and adherence. The consensus was defined as ≥75% of votes in Round 1 or using a 5-point Likert-type scale (1 = strongly disagree, 2 = disagree, 3 = neither agree nor disagree, 4 = agree, and 5 = strongly agree) in Round 2, which included questions not reaching minimum cut-off in the first step. Results: Nine expert panelists with extensive experience in the Brazilian Public Health System participated. The panel reached an agreement on recommendations about prophylaxis, bleeding treatment patterns, and bleeding sites. From patients' perspectives, venous access and infusion frequency were the most significant barriers to improving patient treatment. According to most experts, emicizumab will not replace ITI or long-term factor VIII therapy. Still, emicizumab was thought to be a good therapeutic option for patients with difficult venous access, patients requiring central venous access, in the presence of inhibitors, or patients experiencing infusion-related pain. Conclusion: The information gleaned from this study may be helpful to both decision-makers and those in charge of developing healthcare economic models for the treatment of hemophilia A in Brazil.

Objetivo: É fundamental entender as necessidades não atendidas relacionadas ao manejo da hemofilia A no Brasil. Métodos: Foi conduzido um painel Delphi modificado. Foram convidados hematologistas com vasta experiência no tratamento de hemofilia no SUS para responder a perguntas sobre indicadores de eficácia da profilaxia, indicações de tratamento com emicizumabe, uso de agentes de bypass, uso de indução de tolerância imunológica (ITI) e adesão. O consenso foi definido como ≥75% dos votos na rodada 1 ou usando uma escala do tipo Likert de 5 pontos (1 = discordo totalmente, 2 = discordo, 3 = não concordo nem discordo, 4 = concordo e 5 = concordo totalmente) na segunda rodada, que incluiu questões que não atingiram o corte mínimo na primeira etapa. Resultados: Nove especialistas participaram e houve consenso sobre recomendações para profilaxia, padrões de tratamento de sangramento e locais de sangramento. O acesso venoso e a frequência da infusão foram identificados como as barreiras mais significativas para melhorar o tratamento do paciente. De acordo com a maioria, emicizumabe não substituirá a ITI ou tratamento com fator VIII de longo prazo. Emicizumabe foi considerado uma boa opção terapêutica para

Vaccines are different: A systematic review of budget impact analyses of vaccines.

INTRODUCTION: Several countries require manufacturers to present a budget impact analysis (BIA), together with a cost-effectiveness analysis, to support national funding requests. However, guidelines for conducting BIA of vaccines are scarce. OBJECTIVES: To analyze the methodological approaches used in published budget impact analysis (BIA) of vaccines, discussing specific methodological issues related to vaccines. MATERIAL AND METHODS: This systematic review of the literature on BIA of vaccines was carried out in accordance with the Centre for Reviews and Dissemination - CRD guidelines. We searched multiple databases: MedLine, Embase, Biblioteca Virtual de Saúde (BVS), Cochrane Library, DARE Database, NHS Economic Evaluation Database (NHS EED), HTA Database (via Centre for Reviews and Dissemination - CRD), and grey literature. Two researchers, working independently, selected the studies and extracted the data. The methodology quality of individual studies was assessed using the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force. A qualitative narrative synthesis was conducted. RESULTS: Twenty-two studies were reviewed. The most frequently evaluated vaccines were pneumococcal (41%), influenza (23%) and rotavirus (18%). The target population was stated in 21 studies (95%) and the perspective was clear in 20 (91%). Only 36% reported the calculations used to complete the BIA, 27% informed the total and disaggregated costs for each time period, and 9% showed the change in resource use for each time period. More than half of the studies (55%, n=12) reported less than 50% of the items recommended in the checklist. CONCLUSIONS: The production of BIA of vaccines has increased from 2009. The report of the methodological steps was unsatisfactory, making it difficult to assess the validity of the results presented. Vaccines specific issues should be discussed in international guidelines for BIA of vaccines, to improve the quality of the studies.