Broadstreet Health Economics and Outcomes Research, Vancouver, Canada.
Ipsen Global, Boulogne-Billancourt, France.
J Med Econ. 2020 Jun;23(6):631-640. doi: 10.1080/13696998.2020.1722138. Epub 2020 Feb 7.
Injectable botulinum neurotoxins are a mainstay of treatment for pediatric spasticity. AbobotulinumtoxinA and onabotulinumtoxinA are both injectable toxin therapies used to treat pediatric lower limb (PLL) spasticity in Canada. The objective of this study was to assess the cost-effectiveness of abobotulinumtoxinA vs. onabotulinumtoxinA in the treatment of PLL spasticity in Canada. A probabilistic Markov cohort model with a 2-year time horizon was developed, with health states defined by response to therapy, as characterized by the goal attainment scale (GAS). Based on randomized controlled trial evidence, response to therapy was similar or higher for abobotulinumtoxinA relative to onabotulinumtoxinA; uncertainty was incorporated into model parameters, however, as the two therapies have not been compared head-to-head. Canadian resource use and cost data were incorporated. In the base case, abobotulinumtoxinA generated 1.48 quality-adjusted life years over the model time horizon, compared to 1.47 for onabotulinumtoxinA. AbobotulinumtoxinA was associated with cost savings of $123 CAD, reflecting lower costs in both medication acquisition and health services. The estimated improvement to quality of life and reduced costs result in an estimate of economic dominance for abobotulinumtoxinA over onabotulinumtoxinA. This dominant result persisted across probabilistic and scenario analyses.Key points for decision makersBased on a review of available clinical evidence, abobotulinumtoxinA was found to have significant and/or numerical efficacy benefits to onabotulinumtoxinA on functional outcomes (Goal Attainment Scale) and tone (Modified Ashworth Scale) and in the treatment of pediatric lower limb spasticityIn this cost-effectiveness analysis, abobotulinumtoxinA was found to be associated with greater quality-adjusted life years and lower costs than onabotulinumtoxinA (economically dominant)A limitation of this analysis was the uncertainty around key parameters. Specifically, the lack of head-to-head comparison data for the two therapies, and variable data regarding likely onabotulinumtoxinA dosing in PLL in clinical practice. However, across a range of plausible scenarios, the economic dominant result remained.
注射用肉毒毒素是治疗小儿痉挛的主要方法。阿博特毒素和奥博特毒素都是用于治疗加拿大小儿下肢（PLL）痉挛的注射毒素疗法。本研究的目的是评估阿博特毒素相对于奥博特毒素治疗加拿大 PLL 痉挛的成本效益。采用具有 2 年时间范围的概率马尔可夫队列模型，根据治疗反应（由目标实现量表（GAS）确定）来定义健康状态。基于随机对照试验证据，阿博特毒素相对于奥博特毒素的治疗反应相似或更高；然而，由于这两种疗法尚未进行头对头比较，因此模型参数中包含了不确定性。纳入了加拿大资源利用和成本数据。在基础情况下，阿博特毒素在模型时间范围内产生了 1.48 个质量调整生命年，而奥博特毒素为 1.47。阿博特毒素与 123 加元的成本节约相关，这反映了药物获取和医疗服务成本的降低。生活质量的改善和成本的降低导致阿博特毒素相对于奥博特毒素具有经济优势的估计。这一主导结果在概率和情景分析中均得到了维持。决策者要点根据现有临床证据的审查，发现阿博特毒素在功能结果（目标实现量表）和张力（改良 Ashworth 量表）方面具有显著的或数值上优于奥博特毒素的疗效优势，并且在治疗小儿下肢痉挛方面具有优势。在这项成本效益分析中，发现阿博特毒素与奥博特毒素相比，具有更高的质量调整生命年和更低的成本（经济优势）。本分析的一个局限性是关键参数存在不确定性。具体来说，缺乏两种疗法的头对头比较数据，以及在临床实践中 PLL 中奥博特毒素可能的剂量数据存在变异性。然而，在一系列合理的情景中，经济优势的结果仍然存在。
