Thongsri Watsamon, Bussabawalai Thanaporn, Leelahavarong Pattara, Wanitkun Suthep, Durongpisitkul Kritvikrom, Chaikledkaew Usa, Teerawattananon Yot
a Health Intervention and Technology Assessment Program (HITAP) , Ministry of Public Health , Nonthaburi , Thailand.
b Social and Administrative Pharmacy Excellence Research (SAPER) Unit, Department of Pharmacy, Faculty of Pharmacy , Mahidol University , Bangkok , Thailand.
Expert Rev Pharmacoecon Outcomes Res. 2016 Aug;16(4):525-36. doi: 10.1586/14737167.2016.1120672. Epub 2015 Dec 19.
This study aims to compare the lifetime costs and health outcomes of both first-line and sequential combination treatments with standard treatment for pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD) (PAH-CHD) patients.
A cost-utility analysis was performed using a Markov model based on a societal perspective. One-way and probabilistic sensitivity analyses were performed to investigate the effect of parameter uncertainty.
As first-line treatments, both beraprost (incremental cost-effectiveness ratio (ICER) = 192,752 and 201,308 Thai baht (THB) per quality-adjusted life year (QALY) gained) and sildenafil (ICER = 249,770 and 226,802 THB per QALY gained) seemed cost-effective for PAH-CHD patients aged ≤30 years in functional classes II and III, respectively, while no treatment was cost-effective for the sequential combination therapy.
Sildenafil should be included in the National Drug List of Essential Medicines as the first-line treatment for PAH-CHD, and its price per dose should be negotiated to be reduced by 43-57%.
本研究旨在比较一线治疗和序贯联合治疗与标准治疗对先天性心脏病（CHD）相关肺动脉高压（PAH）（PAH-CHD）患者的终生成本和健康结局。
基于社会视角，使用马尔可夫模型进行成本效用分析。进行单向和概率敏感性分析以研究参数不确定性的影响。
作为一线治疗，对于功能分级为II级和III级、年龄≤30岁的PAH-CHD患者，贝拉普罗斯（每获得一个质量调整生命年（QALY）的增量成本效益比（ICER）分别为192,752和201,308泰铢（THB））和西地那非（每获得一个QALY的ICER为249,770和226,802泰铢）似乎都具有成本效益，而序贯联合治疗没有一种治疗方案具有成本效益。
西地那非应列入国家基本药物清单，作为PAH-CHD的一线治疗药物，其每剂价格应协商降低43%-57%。
