EFFECTS OF RECOMBINANT HUMAN DNASE THERAPY ON HEALTH-CARE USE AND COSTS IN PATIENTS WITH CYSTIC-FIBROSIS

OBJECTIVE: To assess the effects of recombinant human DNase (rhDNase) therapy on the cost of treating respiratory tract infections (RTIs) in patients with cystic fibrosis. DESIGN: We prospectively documented th e use of healthcare services among 968 patients with cystic fibrosis w ho participated in a recent Phase III double-blind, multicenter, clini cal trial in which patients were assigned randomly to receive either r hDNase 2.5 mg once daily, rhDNase 2.5 mg twice daily, or placebo. All patients were followed for 24 weeks. Data from secondary sources were used to estimate a total cost of RTI-related care (excluding the cost of study therapy) for each trial participant, based on observed levels of resource use. MAIN OUTCOME MEASURES: Number of RTI-related hospita l admissions, days of RTI-related outpatient antibiotic therapy (intra venous and oral), and total costs of RTI-related care (excluding the c ost of study therapy). RESULTS: Patients randomized to receive rhDNase once daily averaged 0.15 fewer RTI-related hospital admissions (0.41 vs 0.56 for placebo; p < 0.05) and 1.5 fewer days of RTI-related outpa tient intravenous antibiotic therapy (2.9 vs 4.4; p < 0.05). Patients randomized to receive rhDNase twice daily had 0.14 fewer hospital admi ssions (p < 0.01), but no reduction in outpatient intravenous antibiot ic therapy. Compared with placebo, the cost of treating RTIs over 24 w eeks was $814-1682 less among patients receiving rhDNase. CONCLUSIONS: rhDNase therapy reduced the costs of treating RTIs in patients with c ystic fibrosis; assuming once-daily dosing, these savings would offset about one-third of the cost of such therapy.