Grabe-Heyne Kristin, Henne Christof, Odeyemi Isaac, Pöhlmann Johannes, Ahmed Waqas, Pollock Richard F
medac GmbH, Wedel, Germany.
Department of Health Professions, Health Economics and Outcomes Research, Manchester Metropolitan University, Manchester, UK.
J Med Econ. 2023 Jan-Dec;26(1):411-421. doi: 10.1080/13696998.2023.2189860.
Approximately 75% of bladder cancer (BC) cases present as non-muscle-invasive BC (NMIBC). In patients with high-risk NMIBC, the mainstay treatment is intravesical Bacillus Calmette-Guérin (BCG), with immediate radical cystectomy (RC) as an alternative treatment option. The aim of the present study was to evaluate the cost-utility of BCG versus RC in patients with high-risk NMIBC from the UK healthcare payer perspective.
A six-state Markov model was developed that covered controlled disease, recurrence, progression to muscle-invasive BC, metastatic disease, and death. The model included adverse events of BCG and RC and monitoring and palliative care. Drug costs were obtained from the British National Formulary. Intravesical delivery, RC, and monitoring costs were sourced from the National Tariff Payment System and the literature. Utility data were obtained from the literature. Analyses were run over a 30-year time horizon, with future costs and effects discounted at 3.5% . One-way and probabilistic sensitivity analyses were performed.
The base case analysis comparing BCG with RC showed that BCG would increase life expectancy by 0.88 years versus RC, from 7.74 to 8.62 years. BCG resulted in an increase of 0.76 quality-adjusted life years (QALYs) versus RC, from 5.63 to 6.39 QALYs. Patients incurred lower lifetime costs if treated with BCG (£47,753) than with RC (£64,264). Cost savings were mainly driven by the lower cost of BCG versus RC, and palliative care costs. Sensitivity analyses showed that results were robust to assumptions.
The evidence base informing efficacy estimates of BCG is heterogeneous as different BCG administration schedules were reported in the literature, while incidence and cost data on some BCG-associated adverse events were sparse.
Intravesical BCG led to increased QALYs and reduced costs versus RC for patients with high-risk NMIBC from the UK healthcare payer perspective.
约75%的膀胱癌(BC)病例表现为非肌层浸润性膀胱癌(NMIBC)。在高危NMIBC患者中,主要治疗方法是膀胱内灌注卡介苗(BCG),即时根治性膀胱切除术(RC)作为替代治疗选择。本研究的目的是从英国医疗保健支付者的角度评估BCG与RC治疗高危NMIBC患者的成本效益。
建立了一个六状态马尔可夫模型,涵盖疾病控制、复发、进展为肌层浸润性膀胱癌、转移性疾病和死亡。该模型包括BCG和RC的不良事件以及监测和姑息治疗。药物成本来自《英国国家处方集》。膀胱内给药、RC和监测成本来自国家收费支付系统和文献。效用数据来自文献。分析在30年的时间范围内进行,未来成本和效果按3.5%进行贴现。进行了单因素和概率敏感性分析。
比较BCG与RC的基础病例分析表明,与RC相比,BCG可使预期寿命延长0.88年,从7.74年增至8.62年。与RC相比,BCG使质量调整生命年(QALY)增加0.76,从5.63 QALY增至6.39 QALY。接受BCG治疗的患者终身成本(47,753英镑)低于接受RC治疗的患者(64,264英镑)。成本节约主要是由于BCG相对于RC的成本较低以及姑息治疗成本。敏感性分析表明,结果对假设具有稳健性。
由于文献中报道了不同的BCG给药方案,为BCG疗效估计提供依据的证据基础存在异质性,而一些与BCG相关的不良事件的发生率和成本数据稀少。
从英国医疗保健支付者的角度来看,对于高危NMIBC患者,膀胱内灌注BCG相对于RC可提高QALY并降低成本。