Introduction: Mucopolysaccharidosis VI is a rare disease characterized by the arylsulfatase B enzyme deficiency, which is responsible for different clinical manifestations. The treatment consists of enzyme replacement therapy with intravenous administration of galsulfase. Objective: Evaluate the effectiveness of the enzyme replacement therapy with galsulfase for the mucopolysaccharidosis VI treatment. Method: Systematic review of observational studies. The databases of PubMed, Cochrane Library, Lilacs, and Journal of Inherited Metabolic Disease were reviewed. The selection of studies, data mining, and methodological quality assessment were independently conducted by two authors. Results: Eighteen studies fulfilled the inclusion criteria. Two studies were cohorts, one was longitudinal study, one was cross-sectional, one was a case-control, eight were case series, and five were case reports. A total of 362 participants with mucopolysaccharidosis type VI were evaluated, and 14 different outcomes related to the treatment effect were identified. Seven outcomes showed positive results, characterized by the patient survival, quality of life, respiratory function, joint mobility, physical resistance, reduction of urinary glycosaminoglycans, and growth. The hearing function and the cognitive development were stable after the treatment. Other outcomes related to the cardiac function, visual acuity, sleep apnea, and the size of the liver and spleen presented inconclusive outcomes. Concerning safety, light adverse reactions of hypersensitivity were reported. Conclusion: This review provided a broader panoramic view of the outcomes related to mucopolysaccharidosis type VI. Regardless of the inherent limitations of observational studies, the outcomes indicate that the enzyme replacement therapy has a positive effect on most of the outcomes associated to the disease.

This review provided a broader panoramic view of the outcomes related to mucopolysaccharidosis type VI. Regardless of the inherent limitations of observational studies, the outcomes indicate that the enzyme replacement therapy has a positive effect on most of the outcomes associated to the disease.

Contexto: O gasto elevado com medicamentos biológicos ameaça a sustentabilidade dos serviços de saúde. O objetivo da presente re-visão rápida foi embasar a discussão da Política Nacional de Medi-camentos Biológicos no âmbito do Sistema Único de Saúde (SUS), por meio da identificação de barreiras de acesso a esses medica-mentos. Metodologia: Revisão rápida da literatura nas fontes de infor-mação Medline via PubMed, EMBASE, Biblioteca Cochrane e Center for Review and Dissemination (CRD). Resultados: Foram incluídos nove estudos com delineamento transversal. No contexto do usuário, as barreiras foram a falta de conhecimento sobre o medicamento, a dis-tância entre a moradia e o serviço de saúde, os longos períodos de espera por atendimento e a passividade sobre decisão de tratamen-to. Para os profissionais de saúde, as barreiras referem-se a aceitabi-lidade em relação ao medicamento, intercambialidade e substituição, percepção de ausência de dados clínicos que favoreçam eficácia e segurança. No contexto do gestor, as barreiras foram alto custo do medicamento, problemas de reembolso e de assistência. Não foram encontradas barreiras no âmbito governamental do sistema de saúde nos estudos incluídos, apenas no âmbito privado, isto é, a proprieda-de industrial. Conclusão: Para que políticas de saúde sejam efetivas, é essencial o emprego de evidências científicas. São necessárias soluções para as barreiras de acesso de medicamentos biológicos.

IntroductionThe elevated costs with biologic products threaten the sustainability of health services, and, therefore, the access to these medicines in the perspectives of user, health professional, health manager and system. The entry of biosimilar products in the market could be an option to subsidize the search for solutions to those problems.MethodsWe conducted a rapid review using the databases Medline (via PubMed), EMBASE, Cochrane Library and CRD. The eligibility criteria were HTAs, systematic reviews and cross-sectional studies.ResultsLiterature search retrieved 640 registries and, after duplicate removal, screening of titles and abstracts and full text reading, nine cross-sectional studies were selected. From a user's point of view, the following barriers were identified: lack of knowledge about the medicine, distance between the place of living and the health service (especially in the rural area), long waiting periods for service, passivity in regard to treatment. From a health professional's point of view the barriers were: acceptability of the expert in regard to treatment, interchangeability and substitution, the perception of lack of data showing efficacy and safety. Finally, from the payer's (or health manager) point of view, the barriers were: high cost of medicine, problems with reimbursement and bureaucracy. We did not retrieve any barriers from the health system's perspective from the selected studies.ConclusionsThe entry of biosimilar medicines in the market can induce competition and, therefore, reduce prices of biologic treatments. It is necessary to search for potential solutions to the access barriers identified in this rapid review.