Ethical and economic considerations of rare diseases in ethnic minorities: the case of mucopolysaccharidosis VI in Colombia

J Med Ethics. 2012 Nov;38(11):699-700. doi: 10.1136/medethics-2011-100204. Epub 2012 May 1.

Abstract

Mucopolysaccharidosis VI is an autosomal recessive lysosomal storage disorder associated with severe disability and premature death. The presence of a mucopolysaccharidosis-like disease in indigenous ethnic groups in Colombia can be inferred from archaeological findings. There are several indigenous patients with mucopolysaccharidosis VI currently receiving enzyme replacement therapy. We discuss the ethical and economic considerations, regarding both direct and indirect costs, of a high-cost orphan disease in a marginalised minority population in a developing country.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Colombia
  • Disabled Persons
  • Enzyme Replacement Therapy* / economics
  • Ethnicity
  • Humans
  • Minority Groups
  • Mucopolysaccharidosis VI / drug therapy
  • Mucopolysaccharidosis VI / economics*
  • Mucopolysaccharidosis VI / enzymology
  • Mucopolysaccharidosis VI / ethnology
  • N-Acetylgalactosamine-4-Sulfatase / administration & dosage*
  • Rare Diseases / economics*
  • Rare Diseases / ethnology

Substances

  • N-Acetylgalactosamine-4-Sulfatase