Ethical Issues Related to the Access to Orphan Drugs in Brazil: The Case of Mucopolysaccharidosis Type I
Schwartz, Ida V D
Krug, Bárbara C
Santana-da-Silva, Luiz C
Steiner, Carlos E
Acosta, Angelina X
Ribeiro, Erlane M
Galera, Marcial F
Leivas, Paulo G C
Journal of medical ethics 2011 Apr; 37(4): 233-9
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.
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