Muthukumar Madhusubramanian, Desai Kamal, Abogunrin Seye, Harrower Timothy, Gabriel Sylvie, Dinet Jerome
Modelling and Simulation.
Meta Research, Evidera, London.
Clinicoecon Outcomes Res. 2017 Apr 15;9:211-229. doi: 10.2147/CEOR.S112254. eCollection 2017.
Cervical dystonia (CD) involves painful involuntary contraction of the neck and shoulder muscles and abnormal posture in middle-aged adults. Botulinum neurotoxin type A (BoNT-A) is effective in treating CD but little is known about its associated cost-effectiveness.
To evaluate the cost-effectiveness of abobotulinumtoxinA for treating CD from the UK payer perspective.
A Markov model was developed to evaluate the cost-effectiveness of abobotulinum-toxinA versus best supportive care (BSC) in CD, with a lifetime horizon and health states for response, nonresponse, secondary nonresponse, and BSC in patients with CD (mean age: 53 years; 37% male). Clinical improvement measured using Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) was mapped to utility using data from a randomized trial of abobotulinumtoxinA. Health care resource use, costs, and other inputs were from the British National Formulary, Personal Social Services Research Unit, published literature, or expert opinion. Costs and outcomes were discounted at 3.5% per annum.
In the base case, the incremental lifetime quality-adjusted life-years (QALYs) gained from abobotulinumtoxinA arm versus BSC was 0.253 per patient, whereas the incremental cost was £7,160, leading to an incremental cost-effectiveness ratio (ICER) of £30,468 per QALY. One-way sensitivity analyses showed that these results were sensitive to the proportion of responders to abobotulinumtoxinA at first injection, duration between injections, the number of reinjections allowed among primary nonresponders, and any difference in baseline TWSTRS value between the BSC and abobotulinumtoxinA arms. Probabilistic sensitivity analysis showed that abobotulinumtoxinA was cost-effective 46% and 49% of times at thresholds of £20,000 and £30,000 per QALY, respectively. Scenarios are considered including vial-sharing, productivity losses, secondary response/nonresponse at subsequent injections, 5-year time horizon, and alternative reinjection intervals for BoNT-As produced ICERs ranging from cost-saving to £40,777 per QALY, versus BSC.
AbobotulinumtoxinA was found to be cost-effective in treating adults with CD, at acceptable willingness-to-pay thresholds in the UK.
颈部肌张力障碍(CD)表现为中年成年人颈部和肩部肌肉疼痛性不自主收缩以及异常姿势。A型肉毒杆菌神经毒素(BoNT-A)对治疗CD有效,但对其相关成本效益了解甚少。
从英国支付方的角度评估阿柏西普肉毒素A治疗CD的成本效益。
建立马尔可夫模型,以评估阿柏西普肉毒素A与最佳支持治疗(BSC)相比在CD治疗中的成本效益,模型涵盖患者一生,并设定了CD患者(平均年龄53岁;男性占37%)的反应、无反应、继发性无反应和BSC等健康状态。使用多伦多西部痉挛性斜颈评定量表(TWSTRS)测量的临床改善情况通过阿柏西普肉毒素A随机试验的数据转换为效用值。医疗保健资源使用、成本及其他投入数据来自《英国国家处方集》、个人社会服务研究单位、已发表文献或专家意见。成本和结果按每年3.5%进行贴现。
在基础案例中,与BSC相比,阿柏西普肉毒素A组每例患者获得的增量终身质量调整生命年(QALY)为0.253,而增量成本为7160英镑,导致每QALY的增量成本效益比(ICER)为30468英镑。单向敏感性分析表明,这些结果对首次注射时阿柏西普肉毒素A的反应者比例、注射间隔时间、原发性无反应者允许的再次注射次数以及BSC组和阿柏西普肉毒素A组之间基线TWSTRS值的任何差异敏感。概率敏感性分析表明,在每QALY阈值为20000英镑和30000英镑时,阿柏西普肉毒素A分别有46%和49%的概率具有成本效益。所考虑的情景包括药瓶共享、生产力损失、后续注射时的继发性反应/无反应、5年时间范围以及不同的肉毒杆菌神经毒素再次注射间隔,与BSC相比,产生的ICER范围从节省成本到每QALY 40777英镑不等。
在英国可接受的支付意愿阈值下,发现阿柏西普肉毒素A治疗成年CD患者具有成本效益。