A COST-EFFECTIVENESS ANALYSIS OF rhDNase IN CHILDREN WITH CYSTIC FIBROSIS

Abstract

Objectives: This study compared the relative cost-effectiveness of daily recombinant human deoxyribonuclease (rhDNase), with alternate day rhDNase and hypertonic saline (HS) for treating children with cystic fibrosis (CF). Methods: A randomized controlled trial with a crossover design allocated 40 CF children consecutively to 12 weeks of daily rhDNase, alternate day rhDNase, or HS. The primary outcome measure was forced expiratory volume in 1 second (FEV 1 ), a measure of lung function. All health resource use was prospectively documented for each patient and multiplied by unit costs to give a total health service cost for each 12-week treatment period. The nonparametric bootstrap method was used to present costeffectiveness acceptability curves and net benefit statistics for each treatment comparison, for various hypothetical levels of the decision maker's ceiling ratio. Results: Compared with HS, there was a 14% improvement in FEV 1 for daily rhDNase (95% CI, 5% to 23%), and a 12% improvement (95% CI, 2% to 22%) for alternate day rhDNase. For a ceiling ratio of £200 per 1% gain in FEV 1 , the mean net benefits of daily and alternate day rhDNase compared with HS were £1,158 (95% CI, −£621 to 2,842) and £1,188 (95% CI, −847 to 3,343), respectively; the mean net benefit of daily compared with alternate day rhDNase was −£30 (95% CI, −£2,091 to 1,576). Conclusions: If decision makers are prepared to pay £200 for a 1% gain in FEV 1 over a 12-week period, then on average either rhDNase strategy is cost-effective.

Cost‐effectiveness acceptability curves – facts, fallacies and frequently asked questions

“…CEACs can now be found in major medical journals such as BMJ [1][2][3][4][5], New England Journal of Medicine [6,7], Lancet [8], Circulation [9], Chest [10] as well as health economics and health technology assessment journals [11][12][13][14][15][16][17]. Given the widespread dissemination of this technique it is important to ensure that both analysts and potential users of the information understand the nature and interpretation of these curves.…”

Nebulised dornase alfa versus placebo or hypertonic saline in adult critically ill patients: a systematic review of randomised clinical trials with meta-analysis and trial sequential analysis

“…Moreover, when two interventions are (presumed) equivalent, it is of clinical and financial interest to assess costs. In 2001, Grieve and colleagues performed a cost-effectiveness analysis of dornase alfa use in children with CF [ 28 ]. The drug cost per day was increased by a factor of 50 (£0.38 vs. £20.39) by using dornase alfa as compared to hypertonic saline.…”

Colistimethate sodium powder and tobramycin powder for inhalation for the treatment of chronic Pseudomonas aeruginosa lung infection in cystic fibrosis: systematic review and economic model

“…The Novartis Pharmaceuticals submission presents the details of a NMA, a discussion of the difficulties of undertaking economic analyses of treatments for CF, and a brief discussion of three previously published economic analyses of CF treatments. 5,92,94 The submission makes particular note that these three economic analyses have deviated considerably from NICE's reference case 95 with respect to the primary health economic outcome measure adopted, which in each case relates to short-term FEV 1 improvements rather than QALYs gained. The Novartis Pharmaceuticals submission does not include any form of de novo economic evaluation.…”