目的:评价重组因子IXFc融合蛋白(rFIXFc)和重组因子IX(rFIX)治疗中国血友病B(HB)的终生成本-效果。
方法:我们开发了一个决策分析马尔可夫模型,包括三个健康状态:活着,需要手术,死了.该模型估计了儿童预防的终生成本和质量调整寿命年(QALYs),其次是按需治疗在成年期从中国的医疗保健系统的角度来看,中重度到重度HB患者。来自关键临床试验的疗效数据,临床指南建议,和专家咨询适用于两种情况(全剂量和低剂量)。进行单向敏感性分析和概率敏感性分析(PSA)以评估模型的稳健性。
结果:寿命成本,QALYs,计算了增量成本效益比,并将结果与2021年中国人均国内生产总值(12,551-37,653美元)一到三倍的支付意愿(WTP)阈值进行比较。
结果:RFIXFc在两种情况下都比rFIX具有更低的成本和更多的QALY,这表明它是中国中重度至重度乙型肝炎的主要策略(更有效,更便宜)。在全剂量情况下,与低剂量方案相比,rFIXFc节省了更多的资金,产生了更多的QALY(低剂量是中国典型的临床现实)。PSA表明,rFIXFc在WTP阈值为$12,551-$37,653的全剂量和低剂量治疗具有超过90%的成本效益。
结论:与rFIX相比,rFIXFc似乎是中国中重度至重度HB患者的终身管理的一种具有成本效益的选择。
To evaluate the lifetime cost-effectiveness of recombinant factor IX Fc fusion protein (rFIXFc) and recombinant factor IX (rFIX) for the treatment of hemophilia B (HB) in
China.
We developed a decision-analytic Markov model including three health states: alive, requiring surgery, and dead. This model estimated the lifetime cost and quality-adjusted life-years (QALYs) of prophylaxis in childhood, followed by on-demand treatment in adulthood for moderate-severe to severe HB patients from
China\'s healthcare system perspective. Efficacy data derived from pivotal clinical trials, clinical guideline recommendations, and expert consultation were applied to two scenarios (full dose and low dose). One-way sensitivity analysis and probabilistic sensitivity analysis (PSA) were performed to assess the robustness of the model.
Lifetime cost, QALYs, and the incremental cost-effectiveness ratio were calculated, and the results were compared with willingness-to-pay (WTP) thresholds of one to three times the gross domestic product per capita of
China in 2021 ($12,551-$37,653).
RFIXFc was associated with lower cost and more QALYs than rFIX in both scenarios, which suggested that it is a dominant strategy (more effective and cheaper) for moderate-severe to severe HB in China. In the full-dose scenario, rFIXFc saved more money and yielded more QALYs than in the low-dose scenario (low doses are the typical clinical reality in
China). PSA demonstrated that rFIXFc had an over 90% probability of being cost-effective with full-dose and low-dose treatment at WTP thresholds of $12,551-$37,653.
Compared with rFIX, rFIXFc appears to be a cost-effective option for the lifetime management of moderate-severe to severe HB patients in
China.