Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
Background/AimsMucopolysaccharidosis 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 i...
Saved in:
Published in | Journal of medical ethics Vol. 37; no. 4; pp. 233 - 239 |
---|---|
Main Authors | , , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
England
BMJ Publishing Group Ltd and Institute of Medical Ethics
01.04.2011
BMJ Publishing Group BMJ Publishing Group Ltd BMJ Publishing Group LTD |
Subjects | |
Online Access | Get full text |
Cover
Loading…
Summary: | Background/AimsMucopolysaccharidosis 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.MethodsIn 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.ResultsThe 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%).ConclusionsIn 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. |
---|---|
Bibliography: | istex:6261D250642F9353CC7E239E3717376003D1876A ark:/67375/NVC-RK3GN58H-0 local:medethics;37/4/233 PMID:21266385 ArticleID:medethics37150 href:medethics-37-233.pdf ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 |
ISSN: | 0306-6800 1473-4257 |
DOI: | 10.1136/jme.2010.037150 |