Applied Health Research Centre, Li Ka Shing Knowledge Institute, St Michael's Hospital, Toronto, Ontario, Canada; Institute for Clinical Evaluative Sciences, Toronto, Ontario, Canada; Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Institute for Clinical Evaluative Sciences, Toronto, Ontario, Canada.
Ann Allergy Asthma Immunol. 2018 Jan;120(1):59-65.e2. doi: 10.1016/j.anai.2017.08.016. Epub 2017 Oct 3.
Omalizumab is indicated for the treatment of moderate to severe asthma. There is limited observational evidence on the costs and effectiveness of omalizumab.
To examine the costs and effectiveness of omalizumab for treatment of severe asthma relative to nonusers.
We conducted a within-person repeated-measures matched cohort study in Ontario, Canada from April 1, 2012 to March 31, 2014. Continuous users of omalizumab were matched with up to 4 nonusers according to age, sex, recent specialist visits, oral corticosteroid use, asthma severity, and Charlson comorbidity score. The primary outcome was direct health care costs. Secondary outcomes were asthma-related hospitalizations or emergency department visits and oral corticosteroid use. The association between omalizumab use and each outcome was assessed using mixed-effects models adjusting for confounders.
Ninety-five omalizumab users and 352 nonusers were matched. Among users, there was a significant increase in health care costs of $1,796 per person owing to the cost of the medication at treatment initiation (P < .0001). Costs did not change significantly among nonusers ($85 increase in average monthly costs per person; P = .59). We found no significant changes in the rates of asthma-related hospitalizations or emergency department visits among omalizumab users (P = .44) or nonusers (P = .99) between pre- and postintervention periods.
The use of omalizumab was associated with increased costs but no evidence of lower rates of clinically important outcomes. These results suggest omalizumab had limited effectiveness in our study population. Future studies should further explore subsets of patients most likely to benefit from omalizumab therapy.
奥马珠单抗被批准用于治疗中重度哮喘。关于奥马珠单抗的成本和疗效，仅有有限的观察性证据。
评估奥马珠单抗治疗重度哮喘的成本和疗效与非使用者相比。
我们在加拿大安大略省进行了一项基于个体的重复测量匹配队列研究，时间为 2012 年 4 月 1 日至 2014 年 3 月 31 日。根据年龄、性别、最近专科就诊、口服皮质类固醇使用、哮喘严重程度和 Charlson 合并症评分，将连续使用奥马珠单抗的患者与至多 4 名非使用者进行匹配。主要结局是直接医疗保健成本。次要结局是哮喘相关住院或急诊就诊和口服皮质类固醇使用。使用混合效应模型调整混杂因素后，评估奥马珠单抗使用与每种结局的关联。
匹配了 95 名奥马珠单抗使用者和 352 名非使用者。在使用者中，由于治疗开始时药物的成本，每人的医疗保健成本显著增加了 1796 美元（P<0.0001）。在非使用者中，成本没有显著变化（每人每月平均成本增加 85 美元；P=0.59）。我们没有发现奥马珠单抗使用者（P=0.44）或非使用者（P=0.99）在干预前后期间哮喘相关住院或急诊就诊率有显著变化。
奥马珠单抗的使用与成本增加有关，但没有证据表明其临床重要结局的发生率降低。这些结果表明奥马珠单抗在我们的研究人群中疗效有限。未来的研究应进一步探索最有可能从奥马珠单抗治疗中获益的患者亚组。
