Ethical Issues Related To The Access To Orphan Drugs In Brazil: The Case Of Mucopolysaccharidosis Type I

Background/Aims: Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disorder treated with bone marrow transplantation or enzyme replacement therapy with laronidase, a high-cost orphan drug. Laronidase was approved by the US Food and Drug Administration and the European Medicines Agency in 2003 and by the Brazilian National Health Surveillance Agency in 2005. Many Brazilian MPS I patients have been receiving laronidase despite the absence of a governmental policy regulating access to the drug. Epidemiological and treatment data concerning MPS I are scarce. This study aims to present a demographic profile of Brazilian patients with MPS I, describe the routes of access to laronidase in Brazil, and discuss associated ethical issues relating to public funding of orphan drugs. Methods: In this cross-sectional observational study, data were collected nationwide between January and September 2008 from physicians, public institutions and non-governmental organisations involved with diagnosis and treatment of MPS I, using two data collection instruments specifically designed for this purpose. Results: The minimum prevalence of MPS I in Brazil was estimated at 1/2 700 000. Most patients (69.8%) were younger than 15 years; 60 (88.2%) received laronidase. The most common route of access to the drug was through lawsuits (86.6%). Conclusions: In Brazil, MPS I is predominantly a paediatric illness. Even though the cost of laronidase treatment is not officially covered by the Brazilian government, most MPS I patients receive the drug, usually through litigation. This gives rise to major ethical conflicts concerning drug access in a low-resource context. 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Judicialização da saúde, acesso à justiça e a efetividade do direito à saúde Judicialization of the right to health, access to justice and the effectiveness of the right to health

Este artigo busca analisar os vínculos entre acesso à justiça e a efetividade de um dos aspectos do direito à saúde: o acesso aos medicamentos. Inicialmente, apresenta os desafios e as dificuldades na conceituação de saúde e do direito à saúde. Em seguida, analisa processos judiciais individuais que reivindicam dos entes públicos o fornecimento de medicamentos, no período de junho de 2007 a julho de 2008, no Tribunal de Justiça do Estado do Rio de Janeiro. Constata-se que a prescrição médica individual, a hipossuficiência econômica e a urgência dos demandantes ao acesso aos medicamentos são os principais respaldos das decisões judiciais analisadas, que determinam o fornecimento de medicamento conforme requerido pelos autores. Conclui-se que a efetividade do direito à saúde requer um conjunto de respostas políticas e ações governamentais mais amplas, e não meramente formais e restritas às ordens judiciais. As demandas judiciais não podem ser consideradas como principal instrumento deliberativo na gestão da assistência farmacêutica no SUS, mas admitidas como um elemento importante na tomada de decisão dos gestores e, muitas vezes, na melhoria do acesso aos medicamentos no âmbito do SUS. No contexto democrático brasileiro, a judicialização pode expressar reivindicações e modos de atuação legítimos de cidadãos e de instituições. O principal desafio é formular estratégias políticas e sociais orquestradas com outros mecanismos e instrumentos de garantia democrática, que aperfeiçoem os sistemas de saúde e de justiça com vistas à efetividade do direito à saúde.<br>This paper aims to analyze the links between access to justice and the effectiveness of one aspect of the right to health: access to medicines. It initially presents the challenges and difficulties in the conceptualization of health and right to health, and then analyzes individual lawsuits demanding medicines against public services, from June 2007 to July 2008, entered at Rio de Janeiro State Court of Appeals. It appears that the medical drug prescription, the economic conditions of the applicants and the urgency of access to medicines are the main factual basis of judicial sentences examined, which determine the supply of medicines as required by the authors. Finally, it concludes that the effectiveness of the right to health requires a set of policy and broader government actions, and not merely formal and restrictive court orders. The individual's claims cannot be considered as the main deliberative instrument in the management of pharmaceutical care in the Brazilian Health System, but accepted as an important element in the decision making of managers and, often, in the improvement of the access to medicines under National Health System. In the Brazilian democratic context, the judicialization can express demands and modes of action of citizens and legitimate institutions. Thus, the main challenge is to make policy and social strategies orchestrated with other mechanisms and instruments of democratic security, to improve health and justice systems in order to give effectiveness to the right to health