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A cost-effectiveness analysis of rhDNase in children with cystic fibrosis
Richard Grieve1, Simon Thompson, Charles Normand
1Department of Public Health and Policy, London School of Hygiene and Tropical Medicine, UK. richard.grieve@lshtm.ac.uk
Insights
Daily or alternate day recombinant human deoxyribonuclease (rhDNase) treatments are cost-effective for children with cystic fibrosis (CF), offering significant lung function improvements. Both rhDNase regimens provide comparable benefits when compared to hypertonic saline (HS).
Area of Science:
- Pulmonary Medicine
- Health Economics
- Pediatric Respiratory Diseases
Background:
- Cystic Fibrosis (CF) management requires effective therapies to improve lung function.
- Recombinant human deoxyribonuclease (rhDNase) and hypertonic saline (HS) are common treatments.
- Evaluating the cost-effectiveness of different rhDNase dosing strategies is crucial for resource allocation.
Purpose of the Study:
- To compare the cost-effectiveness of daily rhDNase, alternate-day rhDNase, and HS in pediatric CF patients.
- To assess the impact of these treatments on lung function, measured by FEV1.
- To determine the economic value of rhDNase therapies based on willingness-to-pay thresholds.
Main Methods:
- A randomized controlled trial with a crossover design involving 40 children with CF.
- Treatments administered over 12-week periods: daily rhDNase, alternate-day rhDNase, or HS.
- Primary outcome: Forced Expiratory Volume in 1 second (FEV1). Health resource utilization was tracked to calculate total costs.
Main Results:
- Daily rhDNase showed a 14% FEV1 improvement versus HS; alternate-day rhDNase showed a 12% improvement.
- At a ceiling ratio of £200 per 1% FEV1 gain, both daily and alternate-day rhDNase demonstrated positive net benefits compared to HS.
- No significant cost-effectiveness difference was observed between daily and alternate-day rhDNase regimens.
Conclusions:
- Both daily and alternate-day rhDNase are cost-effective treatment options for pediatric CF patients.
- The choice between daily and alternate-day rhDNase may not significantly impact cost-effectiveness at a willingness-to-pay of £200/1% FEV1 gain.
- These findings support the use of rhDNase therapies in CF management, considering both clinical and economic factors.
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 (FEV1), 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 cost-effectiveness 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 FEV1 for daily rhDNase (95% Cl, 5% to 23%), and a 12% improvement (95% Cl, 2% to 22%) for alternate day rhDNase. For a ceiling ratio of 200 pounds sterling per 1% gain in FEV1, the mean net benefits of daily and alternate day rhDNase compared with HS were 1,158 pounds sterling (95% Cl, -621pounds sterling to 2,842) and 1,188 pounds sterling (95% Cl, -847 to 3,343), respectively; the mean net benefit of daily compared with alternate day rhDNase was -30 pounds sterling (95% Cl, -2,091 pounds sterling to 1,576).
Conclusions:
If decision makers are prepared to pay 200 pounds sterling for a 1% gain in FEV1 over a 12-week period, then on average either rhDNase strategy is cost-effective.
