Medical Outcomes and Research in Economics (MORE)® Research Group, Sunnybrook Research Institute, Toronto, Ontario, Canada.
J Med Econ. 2013;16(1):115-24. doi: 10.3111/13696998.2012.734886. Epub 2012 Oct 10.
To review and summarize the literature concerning the cost-effectiveness of palivizumab compared to no prophylaxis in infants and young children with congenital heart disease (CHD).
A systematic literature search (MEDLINE to March 2012, limited to English language) identified studies that examined the cost-effectiveness of palivizumab in CHD populations. The quality of each study was assigned a quality score of 1-100 based on the Quality of Health Economic Studies (QHES) instrument.
Ten studies were identified through the search strategy, of which four principally addressed the research question and six additional articles examined CHD in conjunction with other high-risk indications for palivizumab in their economic analyses. QHES for the studies ranged from 58-100, with a median score of 93 (76 for principal articles, 94 for secondary analyses). Cost-utility analyses, which evaluated costs per quality-adjusted life year (QALY), showed favorable results in five analyses (range $10,329-$16,648 per QALY), while the other two suggested no cost-effectiveness ($146,061 and $169,971 per QALY). Of three cost-effectiveness analyses, which assessed costs per hospital admission prevented (HAP), two concluded that the drug was not cost-effective ($16,216/day of hospitalization prevented and $868,296/HAP), while one did not interpret the final result ($43,561/HAP).
Significant variance exists across study characteristics, analytic models utilized, duration of RSV seasons assessed, primary outcome measures evaluated, sensitivity analyses conducted, and other model assumptions. Further, it was difficult to obtain true CHD-based quality scores for the studies that analyzed more than one indication.
The findings of this review currently remain inconclusive. Although a favorable trend was identified in the cost-utility analyses, additional rigorously conducted studies are necessary to better estimate the cost-effectiveness of palivizumab for CHD infants in clinical practice.
综述并总结关于帕利珠单抗（palivizumab）与无预防措施相比用于患有先天性心脏病（CHD）的婴儿和幼儿的成本效益的文献。
系统文献检索（MEDLINE 至 2012 年 3 月，仅限于英文）确定了研究帕利珠单抗在 CHD 人群中的成本效益的研究。根据健康经济研究质量（QHES）工具，为每项研究分配了 1-100 的质量评分。
通过搜索策略确定了 10 项研究，其中 4 项主要解决了研究问题，6 项额外的文章在其经济分析中结合其他高风险帕利珠单抗适应症研究了 CHD。研究的 QHES 范围为 58-100，中位数为 93（主要文章为 76，次要分析为 94）。评估每质量调整生命年（QALY）成本效益的成本效用分析显示，五项分析结果有利（每 QALY 成本为 10329-16648 美元），而另外两项则表明无成本效益（每 QALY 成本为 146061 美元和 169971 美元）。在三项评估预防住院人次（HAP）成本效益的分析中，两项结论认为该药物不具有成本效益（预防 16216 美元/天的住院费用和预防 868296 美元/HAP），而一项未解释最终结果（预防 43561 美元/HAP）。
研究特征、分析模型使用、评估的 RSV 季节持续时间、主要结果评估、进行的敏感性分析和其他模型假设存在显著差异。此外，对于分析了多个适应症的研究，很难获得真正基于 CHD 的质量评分。
本综述的研究结果目前尚无定论。尽管在成本效用分析中发现了有利趋势，但需要进行更多严格的研究，以更好地估计帕利珠单抗在临床实践中用于 CHD 婴儿的成本效益。
