Sculpher M J, Buxton M J
Health Economics Research Group, Brunel University, England.
Pharmacoeconomics. 1993 Nov;4(5):345-52. doi: 10.2165/00019053-199304050-00005.
The construction of a composite effectiveness measure was explored using clinical data collected routinely in trials of drug therapies for asthma. The measure is the episode-free day (EFD), where an 'episode' is either an asthma attack, the need for rescue medication, sleep disturbance caused by asthma, or an adverse event. The EFD measure was used in a retrospective cost-effectiveness analysis of a previous Phase III controlled clinical trial of formoterol versus salbutamol, in which 145 patients with bronchial asthma were randomised to receive maintenance therapy with either inhaled formoterol or inhaled salbutamol over a 12-week period. Average and incremental cost-effectiveness ratios were assessed for the 2 drugs in terms of the total expected cost of drug plus rescue therapy, and EFD rates. The analysis suggests that, with relatively little addition to clinical data collection, economically and clinically meaningful composite measures can be constructed to assist in making cost-effectiveness comparisons between alternative asthma therapies.
利用哮喘药物治疗试验中常规收集的临床数据，探讨了综合疗效指标的构建。该指标为无发作天数（EFD），其中“发作”是指哮喘发作、需要使用急救药物、由哮喘引起的睡眠障碍或不良事件。EFD指标用于对先前一项福莫特罗与沙丁胺醇对比的III期对照临床试验进行回顾性成本效益分析，在该试验中，145例支气管哮喘患者被随机分配，在12周内接受吸入福莫特罗或吸入沙丁胺醇的维持治疗。根据药物加急救治疗的总预期成本和EFD率，评估了这两种药物的平均成本效益比和增量成本效益比。分析表明，只需在临床数据收集方面增加相对较少的工作量，就可以构建具有经济和临床意义的综合指标，以协助对替代哮喘治疗方案进行成本效益比较。
