McGirr Ashleigh A, Schwartz Kevin L, Allen Upton, Solomon Melinda, Sander Beate
a Dalla Lana School of Public Health , University of Toronto , Toronto , ON , Canada.
b Division of Infectious Disease , The Hospital for Sick Children , Toronto , ON , Canada.
Hum Vaccin Immunother. 2017 Mar 4;13(3):599-606. doi: 10.1080/21645515.2016.1235670. Epub 2016 Oct 21.
Children with cystic fibrosis (CF) are at higher risk of severe respiratory syncytial virus (RSV) infection, which can lead to a decline in lung function. A monoclonal antibody, palivizumab (PMB), effectively prevents RSV hospitalizations; however, the high cost of PMB, approximately C$10,000 per patient per RSV season, limits its widespread use. We assess the cost-effectiveness of PMB prophylaxis in CF children less than 2 y of age from the Canadian healthcare payer's perspective.
In 2014, a Markov cohort model of CF disease and infant RSV infections in the Canadian setting was developed based on literature data. Infants were treated with monthly PMB injections over the 5-month RSV season. Lifetime health outcomes, quality-adjusted life years (QALYs) and 2013 $CAD costs, discounted at 5%, were estimated. Findings are summarized as incremental cost-effectiveness ratios (ICERs) and budget impact. Deterministic sensitivity analysis was conducted to assess parameter uncertainty.
Implementation of a hypothetical Canadian RSV prophylaxis program resulted in ICERs of C$652,560 (all CF infants) and C$157,332 (high-risk CF infants) per QALY gained and an annual budget impact of C$1,400,000 (all CF infants) and C$285,000 (high-risk CF infants). The analysis was highly sensitive to the probability of severe RSV, the degree of lung deterioration following infection, and the cost of PMB.
Our results suggest PMB is not cost-effective in Canada by commonly used thresholds. However, given the rarity of CF and relatively small budget impact, consideration may be given for the selective use of PMB for immunoprophylaxis of RSV in high-risk CF infants on a case-by-case scenario basis.
囊性纤维化(CF)患儿发生严重呼吸道合胞病毒(RSV)感染的风险更高,这可能导致肺功能下降。单克隆抗体帕利珠单抗(PMB)可有效预防RSV住院;然而,PMB成本高昂,每个RSV季节每位患者约10,000加元,限制了其广泛使用。我们从加拿大医疗保健支付方的角度评估PMB预防在2岁以下CF患儿中的成本效益。
2014年,基于文献数据建立了加拿大环境下CF疾病和婴儿RSV感染的马尔可夫队列模型。婴儿在5个月的RSV季节每月接受PMB注射治疗。估计了终身健康结局、质量调整生命年(QALY)和2013年加元成本,按5%进行贴现。研究结果总结为增量成本效益比(ICER)和预算影响。进行了确定性敏感性分析以评估参数不确定性。
实施一个假设的加拿大RSV预防项目,每获得一个QALY的ICER为652,560加元(所有CF婴儿)和157,332加元(高危CF婴儿),年度预算影响为1,400,000加元(所有CF婴儿)和285,000加元(高危CF婴儿)。该分析对严重RSV的概率、感染后肺功能恶化程度以及PMB成本高度敏感。
我们的结果表明,按照常用阈值,PMB在加拿大不具有成本效益。然而,鉴于CF的罕见性和相对较小的预算影响,可考虑在逐案基础上对高危CF婴儿选择性使用PMB进行RSV免疫预防。