United BioSource Corporation, London, UK.
J Med Econ. 2013;16(3):342-8. doi: 10.3111/13696998.2012.756398. Epub 2012 Dec 18.
Omalizumab, licensed for patients with uncontrolled persistent allergic (IgE mediated) asthma, was found to be cost-effective based upon its clinical trial data. Observational studies have been undertaken to determine the real life outcomes of using omalizumab in the community.
To determine the cost-effectiveness of omalizumab based upon observational data from the Netherlands and compare to its cost-effectiveness using clinical trial data.
An observational study (eXpeRience) recruited allergic asthma patients eligible for Omalizumab therapy and followed them while on treatment. At 1 year, data from the Dutch patients enrolled in eXpeRience were examined to estimate the number of exacerbations and resource use while on omalizumab therapy compared to the year prior to omalizumab use. Observational data were used in a Markov model to calculate the lifetime cost-effectiveness ratios.
In the 1 year prior to omalizumab therapy the per-person rate of exacerbations was 3.39 compared to 1.07 in the year taking omalizumab. The discounted incremental lifetime additional costs for omalizumab were €55,865 for 1.46 additional quality-adjusted life years (QALY), resulting in €38,371/QALY. Using the INNOVATE clinical trial outcomes and current resource use, the prior ratio was €34,911/QALY, similar to the observational ratio. As in all observational studies, the main limitation is obtaining complete and accurate data. Patients with missing exacerbation or response data were excluded from this analysis.
Non-clinical trial experience with omalizumab supported the finding of fewer exacerbations in the allergic asthma population while treated with omalizumab, and therapy was found to continue to have an attractive cost-effectiveness ratio.
奥马珠单抗获批用于治疗未得到控制的持续性过敏性（IgE 介导）哮喘患者，其基于临床试验数据被证明具有成本效益。为了确定奥马珠单抗在社区中的实际应用效果，已经开展了观察性研究。
根据荷兰的观察性数据确定奥马珠单抗的成本效益，并将其与临床试验数据进行比较。
一项观察性研究（eXpeRience）招募了符合奥马珠单抗治疗条件的过敏性哮喘患者，并在治疗期间对他们进行随访。在第 1 年，对 eXpeRience 中纳入的荷兰患者的数据进行了评估，以估算在接受奥马珠单抗治疗期间与治疗前一年相比，发作次数和资源使用情况。观察性数据被用于马尔可夫模型来计算终生成本效益比。
在接受奥马珠单抗治疗前的 1 年中，每人每年的发作率为 3.39 次，而在接受奥马珠单抗治疗的那一年为 1.07 次。奥马珠单抗的增量终生额外成本为 55865 欧元，额外获得 1.46 个质量调整生命年（QALY），即 38371 欧元/QALY。使用 INNOVATE 临床试验结果和当前资源使用情况，之前的比值为 34911 欧元/QALY，与观察性比值相似。与所有观察性研究一样，主要限制是获得完整和准确的数据。在这项分析中，对那些没有发作或反应数据的患者进行了排除。
奥马珠单抗的非临床试验经验支持了在接受奥马珠单抗治疗的过敏性哮喘人群中发作次数减少的发现，并且治疗继续具有有吸引力的成本效益比。
