Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA.
Clin Ther. 2013 Apr;35(4):414-24. doi: 10.1016/j.clinthera.2013.02.020. Epub 2013 Mar 20.
Urinary incontinence (UI) secondary to a neurogenic pathology, including spinal cord injury and multiple sclerosis, is termed neurogenic detrusor overactivity (NDO). Patients with NDO experience decreased quality of life and are at risk for upper urinary tract damage. Two recent trials demonstrated that onabotulinumtoxinA significantly reduced UI, improved urodynamic parameters, and improved quality of life relative to placebo. However, the economic impact of onabotulinumtoxinA treatment for UI due to NDO in the United States remains unknown.
The objective of this analysis was to evaluate whether the benefit observed in NDO patients receiving onabotulinumtoxinA provides good value for money.
We developed a Markov state transition model to estimate population outcomes and costs for anticholinergic-refractory NDO patients who received either onabotulinumtoxinA or best supportive care (use of incontinence pads with either an anticholinergic drug, clean intermittent self-catheterization, or both). Nonresponding patients (<50% reduction in UI episodes at 6 weeks) were eligible to receive invasive procedures, including augmentation cystoplasty or sacral neuromodulation. Patients could transition through 6 health states, 3 defined based on response to initial treatment, 2 capturing patients who underwent invasive procedures, and death. Time in each health state was adjusted for patient quality of life and summed to estimate quality-adjusted life-years (QALYs). The model included direct medical costs related to initial and subsequent drug and invasive treatments, physician visits, and catheterization. Outcomes and costs were summed and compared across intervention groups by using the incremental cost-effectiveness ratio (ICER; cost per QALY). The time horizon of the model was 3 years, and results were discounted at 3%. Scenario, 1-way, and probabilistic sensitivity analyses were performed to test the robustness of the model results.
In the base case, onabotulinumtoxinA increased QALYs by 0.059 and costs by $1466 compared with best supportive care, which resulted in an estimated ICER of $24,720/QALY. OnabotulinumtoxinA also decreased mean UI episodes per person-year by 398, resulting in a cost of $4 per UI episode avoided. Model results were most sensitive to the probability of treatment response. The probabilistic sensitivity analysis indicated that at a willingness to pay of $50,000/QALY, onabotulinumtoxinA has a 97% probability of being cost-effective. In subgroup analyses of each etiology, onabotulinumtoxinA yielded an ICER of $32,268/QALY in multiple sclerosis and $2182 in spinal cord injury.
OnabotulinumtoxinA seems to be a cost-effective intervention for UI due to NDO compared with best supportive care.
由神经病变引起的尿失禁(UI),包括脊髓损伤和多发性硬化症,被称为神经源性逼尿肌过度活动(NDO)。NDO 患者生活质量下降,有上尿路损伤的风险。两项最近的试验表明,与安慰剂相比,肉毒杆菌毒素 A 显著减少了 UI,改善了尿动力学参数,并提高了生活质量。然而,在美国,由于 NDO 导致的尿失禁使用肉毒杆菌毒素 A 治疗的经济影响仍不清楚。
本分析的目的是评估接受肉毒杆菌毒素 A 治疗的 NDO 患者观察到的益处是否具有良好的性价比。
我们开发了一个马尔可夫状态转移模型,以估计接受肉毒杆菌毒素 A 或最佳支持性护理(使用抗胆碱能药物的失禁垫、间歇性清洁自我导尿或两者兼有)的抗胆碱能药物难治性 NDO 患者的人群结局和成本。无反应的患者(6 周时 UI 发作减少<50%)有资格接受侵入性手术,包括膀胱扩大术或骶神经调节。患者可以通过 6 种健康状态进行转换,其中 3 种状态基于初始治疗的反应,2 种状态用于捕获接受侵入性手术的患者和死亡。每个健康状态的时间都根据患者的生活质量进行了调整,并加起来估计了质量调整生命年(QALY)。该模型包括与初始和后续药物及侵入性治疗、医生就诊和导尿相关的直接医疗费用。通过增量成本效益比(ICER;每 QALY 的成本)比较干预组之间的结果和成本。模型的时间范围为 3 年,结果贴现率为 3%。进行了情景、单向和概率敏感性分析,以测试模型结果的稳健性。
在基础情况下,与最佳支持性护理相比,肉毒杆菌毒素 A 增加了 0.059 个 QALY,增加了 1466 美元的成本,导致估计的 ICER 为每 QALY 24720 美元。肉毒杆菌毒素 A 还使每人每年的平均 UI 发作次数减少了 398 次,从而使每次避免 UI 的成本达到 4 美元。模型结果对治疗反应的概率最为敏感。概率敏感性分析表明,在支付意愿为 50000 美元/QALY 的情况下,肉毒杆菌毒素 A 具有 97%的成本效益概率。在多发性硬化症和脊髓损伤的每种病因亚组分析中,肉毒杆菌毒素 A 的 ICER 分别为 32268 美元/QALY 和 2182 美元。
与最佳支持性护理相比,肉毒杆菌毒素 A 似乎是一种治疗 NDO 引起的尿失禁的具有成本效益的干预措施。