RT Journal Article
SR Electronic
T1 Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
JF Journal of Medical Ethics
JO J Med Ethics
FD BMJ Publishing Group Ltd and Institute of Medical Ethics
SP 233
OP 239
DO 10.1136/jme.2010.037150
VO 37
IS 4
A1 Raquel Boy
A1 Ida V D Schwartz
A1 Bárbara C Krug
A1 Luiz C Santana-da-Silva
A1 Carlos E Steiner
A1 Angelina X Acosta
A1 Erlane M Ribeiro
A1 Marcial F Galera
A1 Paulo G C Leivas
A1 Marlene Braz
YR 2011
UL http://jme.bmj.com/content/37/4/233.abstract
AB 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. The Brazilian health policy framework lacks evidence-based clinical protocols for the distribution of orphan drugs.