Briggs Andrew H, Lozano-Ortega Greta, Spencer Sally, Bale Geraldine, Spencer Michael D, Burge P Sherwood
Public Health & health Policy, University of Glasgow, Glasgow, UK.
Value Health. 2006 Jul-Aug;9(4):227-35. doi: 10.1111/j.1524-4733.2006.00106.x.
To explore the cost-effectiveness of fluticasone propionate (FP) for the treatment of chronic obstructive pulmonary disease (COPD), we estimated costs and quality-adjusted life-years (QALYs) over 3 years, based on an economic appraisal of a previously reported clinical trial (Inhaled Steroids in Obstructive Lung Disease in Europe [ISOLDE]).
Seven hundred forty-two patients enrolled in the ISOLDE trial who received either FP or placebo had data available on health-care costs and quality of life over the period of the study. The SF-36-based utility scores for quality of life were used to calculate QALYs. A combined imputation and bootstrapping procedure was employed to handle missing data and to estimate statistical uncertainty in the estimated cumulative costs and QALYs over the study period. The imputation approach was based on propensity scoring and nesting this approach within the bootstrap ensured that multiple imputations were performed such that statistical estimates included imputation uncertainty.
Complete data were available on mortality within the follow-up period of the study and a nonsignificant trend toward improved survival of 0.06 (95% confidence interval [CI]-0.01 to 0.15) life-years was observed. In an analysis based on a propensity scoring approach to missing data we estimated the incremental costs of FP versus placebo to be 1021 sterling pound(95% CI 619-1338 sterling pound) with an additional effect of 0.11 QALYs (CI 0.04-0.20). Cost-effectiveness estimates for the within-trial period of 17,700 sterling pound per life-year gained (6900 sterling pound to infinity) and 9500 sterling pound per QALY gained (CI 4300-26,500 sterling pound) were generated that include uncertainty due to the imputation process. An alternative imputation approach did not materially affect these estimates.
Previous analyses of the ISOLDE study showed significant improvement on disease-specific health status measures and a trend toward a survival advantage for treatment with FP. This analysis shows that joint considerations of quality of life and survival result in a substantial increase in QALYs favoring treatment with FP. Based on these data, the inhaled corticosteroid FP appears cost-effective for the treatment of COPD. Confirmation or refutation of this result may be achieved once the Towards a Revolution in COPD Health (TORCH) study reports, a large randomized controlled trial powered to detect mortality changes associated with the use of FP alone, or in combination with salmeterol, which is also collecting resource use and utility data suitable for estimating cost-effectiveness.
为探讨丙酸氟替卡松（FP）治疗慢性阻塞性肺疾病（COPD）的成本效益，我们基于对先前一项临床试验（欧洲阻塞性肺病吸入类固醇[ISOLDE]）的经济评估，估算了3年期间的成本和质量调整生命年（QALY）。
ISOLDE试验中742名接受FP或安慰剂治疗的患者在研究期间有医疗保健成本和生活质量数据。基于SF - 36的生活质量效用评分用于计算QALY。采用合并插补和自举程序来处理缺失数据，并估计研究期间累计成本和QALY估计值中的统计不确定性。插补方法基于倾向评分，将此方法嵌套在自举法中可确保进行多次插补，使统计估计值包含插补不确定性。
在研究的随访期内可获取完整的死亡率数据，观察到生存改善有不显著趋势，增加0.06（95%置信区间[CI] - 0.01至0.15）生命年。在基于倾向评分法处理缺失数据的分析中，我们估计FP相对于安慰剂的增量成本为1021英镑（95% CI 619 - 1338英镑），额外效果为0.11 QALY（CI 0.04 - 0.20）。得出试验期内每获得1个生命年的成本效益估计值为17700英镑（6900英镑至无穷大），每获得1个QALY的成本效益估计值为9500英镑（CI 4300 - 26500英镑），其中包括因插补过程产生的不确定性。另一种插补方法对这些估计值影响不大。
先前对ISOLDE研究的分析表明，在疾病特异性健康状况指标上有显著改善，且FP治疗有生存优势趋势。本分析表明，综合考虑生活质量和生存情况会使支持FP治疗的QALY大幅增加。基于这些数据，吸入性糖皮质激素FP治疗COPD似乎具有成本效益。一旦“慢性阻塞性肺疾病健康革命（TORCH）”研究报告结果，可能会证实或反驳这一结果。该研究是一项大型随机对照试验，旨在检测单独使用FP或与沙美特罗联合使用时与死亡率变化相关的情况，同时也在收集适合估算成本效益的资源使用和效用数据。
