A COST-EFFECTIVENESS ANALYSIS OF THE ORPHAN DRUG CYSTEAMINE IN THE TREATMENT OF INFANTILE CYSTINOSIS

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 si gnificant health benefits. This study examined the cost-effectiveness of the administration of cysteamine to cystinotic patients prior to en d-stage renal disease (ESRD). Method. 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 Life expectancy outcomes were derived from both published and unpublished clinical studies and from the U.S. Renal Data System.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 exten d the life of kidneys and delay renal transplantation, thereby increas ing life expectancy for patients with cystinosis. Patients receiving c ysteamine therapy prior to renal failure have lifetime-treatment drug costs of $234,000, in comparison with $238,000 for those who are not m edicated. 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 reduc es health care costs for patients with cystinosis.