A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis.

@article{Soohoo1997ACA,
  title={A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis.},
  author={Nelson Fong Soohoo and Jennifer A. Schneider and Robert M. Kaplan},
  journal={Medical decision making : an international journal of the Society for Medical Decision Making},
  year={1997},
  volume={17 2},
  pages={193-8}
}
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). METHOD Decision-tree analysis and cost-effectiveness analysis. Cost data were estimated from current clinical charges… CONTINUE READING

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