title: A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis.
abstract: OBJECTIVE
  Cysteamine is a recently licensed orphan drug used to treat the inherited metabolic disease cystinosis. The drug delays the onset of renal failure in cystinotic patients and may provide many other significant health benefits. This study examined the cost-effectiveness of the administration of cysteamine to cystinotic patients prior to end-stage renal disease (ESRD).
  
  
  METHODS
  Decision-tree analysis and cost-effectiveness analysis. Cost data were estimated from current clinical charges and Medicare public-access reports. Life expectancy outcomes were derived from both published and unpublished clinical studies and from the U.S. Renal Data System.
  
  
  RESULTS
  Cysteamine therapy can extend the life of kidneys and delay renal transplantation, thereby increasing life expectancy for patients with cystinosis. Patients receiving cysteamine therapy prior to renal failure have lifetime-treatment drug costs of $234,000, in comparison with $238,000 for those who are not medicated. Costs of cysteamine therapy are offset by savings associated with delaying transplantation and costs of dialysis.
  
  
  CONCLUSIONS
  Use of the orphan drug cysteamine both improves health outcomes and reduces health care costs for patients with cystinosis.
authors.affiliation: University of California, San Diego 92093-0622, USA.
authors.forename: N
authors.initials: N
authors.lastname: Soohoo
authors.forename: J A
authors.initials: JA
authors.lastname: Schneider
authors.forename: R M
authors.initials: RM
authors.lastname: Kaplan
mesh_terms: D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D003543:Cysteamine / Q000191:economics* / Q000627:therapeutic use; D003554:Cystinosis / Q000188:drug therapy / Q000191:economics* / Q000401:mortality; D003661:Decision Support Techniques; D003663:Decision Trees; D005260:Female; D006801:Humans; D007223:Infant; D007676:Kidney Failure, Chronic / Q000191:economics / Q000401:mortality / Q000517:prevention & control; D016030:Kidney Transplantation / Q000191:economics; D015993:Life Tables; D008297:Male; D009965:Orphan Drug Production / Q000191:economics*; D006435:Renal Dialysis / Q000191:economics; D016019:Survival Analysis
chemical_list: D003543:Cysteamine