van Hout Ben A, Gagnon Dennis D, McNulty Pauline, O'Hagan Anthony
Julius Center for General Practice and Patient Oriented Research, Academic Medical Center Utrecht, Utrecht, The Netherlands.
Pharmacoeconomics. 2003;21(5):315-26. doi: 10.2165/00019053-200321050-00003.
A number of new antiepileptic agents have been introduced within a short period of time. Direct comparisons are not available, and information about the balance between costs and effects for these new therapies is lacking.
To introduce a first approximation of the cost effectiveness of the new therapeutic agents (topiramate and lamotrigine) for epilepsy that have been assessed in clinical trials against placebo.
Without head to head comparative data no formal methods are available to assess the relative cost effectiveness of two products; therefore, a Bayesian approach was developed. The approach starts with the 'proportionality assumption' saying that the differences in healthcare expenditure (less the direct cost of therapy) are directly proportional to the differences in effectiveness. Given this assumption, a therapy that is x times as expensive as an alternative therapy has an equivalent cost-effectiveness profile if the acquisition cost is x times as high. Moreover, simple formulas can be derived to calculate the probabilities that a therapy is dominant (more effective and less expensive) and that it is weakly dominant (more effective and a better cost-effectiveness profile). The approach is applied to data from published fixed dosage, parallel-design studies comparing both topiramate and lamotrigine with placebo.
Assuming that the 'proportionality assumption' holds for the medical treatment of epilepsy, and disregarding uncertainties, it is estimated that topiramate may be priced more than 2.2 times its current acquisition cost and still be more cost effective than lamotrigine. Taking uncertainties into account, it is estimated that lamotrigine 500 mg/day is dominated by topiramate 200 mg/day with a probability of 0.875 and by topiramate 400 mg/day with a probability of 0.986.
A simple method can be applied to assess the relative cost effectiveness of two therapies in the absence of direct comparative data. Applying this method to compare topiramate and lamotrigine leads to a strong preference for topiramate. However, to be able to draw this conclusion, some heroic assumptions need to be made. As such the method as developed here only reflects a first approximation. It needs to be used with care and is not intended to replace good comparative research.
在短时间内已引入多种新型抗癫痫药物。尚无直接比较,且缺乏这些新疗法成本与效果平衡的信息。
初步估算在临床试验中与安慰剂对照评估的新型抗癫痫治疗药物(托吡酯和拉莫三嗪)的成本效益。
由于没有直接对比数据,无法采用正式方法评估两种产品的相对成本效益;因此,开发了一种贝叶斯方法。该方法始于“比例假设”,即医疗保健支出差异(减去治疗直接成本)与效果差异成正比。基于此假设,如果一种疗法的购置成本是另一种替代疗法的x倍,那么其成本效益相当。此外,可推导出简单公式来计算一种疗法占优(更有效且成本更低)以及弱占优(更有效且成本效益更佳)的概率。该方法应用于已发表的固定剂量、平行设计研究的数据,这些研究比较了托吡酯和拉莫三嗪与安慰剂。
假设“比例假设”适用于癫痫的药物治疗,且不考虑不确定性因素,估计托吡酯的定价可以高于其当前购置成本2.2倍以上,且仍比拉莫三嗪更具成本效益。考虑到不确定性因素,估计拉莫三嗪500毫克/天被托吡酯200毫克/天占优的概率为0.875,被托吡酯400毫克/天占优的概率为0.986。
在缺乏直接对比数据的情况下,可应用一种简单方法评估两种疗法的相对成本效益。应用该方法比较托吡酯和拉莫三嗪,结果强烈倾向于托吡酯。然而,要得出这一结论,需要做出一些大胆假设。因此,此处开发的方法仅反映初步估算。使用时需谨慎,且无意取代良好的对比研究。