LeLorier J, Perreault S, Birnbaum H, Greenberg P, Sheehy O
Research Center, Centre Hospitalier de l'Université de Montréal-Hôtel-Dieu, Québec, Canada.
Clin Ther. 2000 Jan;22(1):140-51. doi: 10.1016/s0149-2918(00)87985-0.
Two identical 24-week, double-blind, placebo-controlled trials of tobramycin solution for inhalation (TOBI [PathoGenesis Corporation, Seattle, Washington]) in cystic fibrosis patients with chronic Pseudomonas aeruginosa infection were conducted in the United States. The aim of the present study was to extrapolate the US trial data to a Canadian setting, using Canadian costs to estimate the savings in direct medical costs that might result from use of a similar 24-week TOBI regimen versus usual care in 2 Canadian provinces.
Cystic fibrosis is a genetic disease in which persistent respiratory infection, usually due to P. aeruginosa infection, is the major cause of morbidity and mortality.
The US trials demonstrated that TOBI produced significant improvements in pulmonary function test results, reduced sputum levels of P. aeruginosa, and resulted in a 26% reduction in the probability of hospitalization (95% CI, 2%-43% vs placebo in the clinical trials). Individual patient data from the US trials were used to calculate the mean number of days in hospital as well as the mean number of days of home intravenous or oral antibiotic therapy. To adjust for Canadian pricing, pertinent economic data were obtained from Statistics Canada and the Ontario and Quebec health ministries. Demographic and baseline data were obtained from health surveys conducted by the Canadian Cystic Fibrosis Foundation.
Economic analysis showed that the use of TOBI for 24 weeks would result in estimated mean per-patient savings in direct medical costs (in Canadian dollars) of $4055 in Ontario and $4916 in Quebec, which would substantially offset the Canadian acquisition price of $8602 for the same 24-week period.
Assuming that the percentage of reduction in hospital days observed in the US trials would also occur in the Canadian clinical setting, use of TOBI would reduce the use of health care services, particularly hospital days, and lead to substantial savings in direct medical costs that would offset its acquisition price. Whether this reduction actually occurs after TOBI enters the Canadian market is a subject for future investigation.
在美国开展了两项针对患有慢性铜绿假单胞菌感染的囊性纤维化患者的吸入用妥布霉素溶液（TOBI[PathoGenesis公司，华盛顿州西雅图]）的24周、双盲、安慰剂对照试验。本研究的目的是将美国试验数据外推至加拿大情况，使用加拿大成本来估计在加拿大两个省份使用类似的24周TOBI治疗方案与常规治疗相比可能节省的直接医疗成本。
囊性纤维化是一种遗传性疾病，其中持续性呼吸道感染（通常由铜绿假单胞菌感染引起）是发病和死亡的主要原因。
美国试验表明，TOBI可显著改善肺功能测试结果，降低痰液中铜绿假单胞菌水平，并使住院概率降低26%（临床试验中与安慰剂相比，95%CI为2%-43%）。美国试验的个体患者数据用于计算住院天数以及家庭静脉或口服抗生素治疗天数的平均值。为根据加拿大定价进行调整，从加拿大统计局以及安大略省和魁北克省卫生部获取了相关经济数据。人口统计学和基线数据来自加拿大囊性纤维化基金会进行的健康调查。
经济分析表明，使用TOBI治疗24周将使安大略省每位患者的直接医疗成本估计平均节省4055加元，魁北克省为4916加元，这将大幅抵消同一24周期间8602加元的加拿大采购价格。
假设在美国试验中观察到的住院天数减少百分比在加拿大临床环境中也会出现，使用TOBI将减少医疗服务的使用，特别是住院天数，并导致直接医疗成本大幅节省，从而抵消其采购价格。TOBI进入加拿大市场后这种减少是否实际发生是未来研究的主题。
