A Cost-Effectiveness Analysis of a Program to Control Rheumatic Fever and Rheumatic Heart Disease in Pinar del Rio, Cuba

Background
Acute rheumatic fever (ARF) and rheumatic heart disease (RHD) persist in many low- and middle-income countries. To date, the cost-effectiveness of population-based, combined primary and secondary prevention strategies has not been assessed. In the Pinar del Rio province of Cuba, a comprehensive ARF/RHD control program was undertaken over 1986 – 1996. The present study analyzes the cost-effectiveness of this Cuban program.
Methods and Findings
We developed a decision tree model based on the natural history of ARF/RHD, comparing the costs and effectiveness of the 10-year Cuban program to a “do nothing” approach. Our population of interest was the cohort of children aged 5 – 24 years resident in Pinar del Rio in 1986. We assessed costs and health outcomes over a lifetime horizon, and we took the healthcare system perspective on costs but did not apply a discount rate. We used epidemiologic, clinical, and direct medical cost inputs that were previously collected for publications on the Cuban program. We estimated health gains as disability-adjusted life years (DALYs) averted using standard approaches developed for the Global Burden of Disease studies. Cost-effectiveness acceptability thresholds were defined by one and three times per capita gross domestic product per DALY averted. We also conducted an uncertainty analysis using Monte Carlo simulations and several scenario analyses exploring the impact of alternative assumptions about the program’s effects and costs. We found that, compared to doing nothing, the Cuban program averted 5051 DALYs (1844 per 100,000 school-aged children) and saved $7,848,590 (2010 USD) despite a total program cost of $202,890 over 10 years. In the scenario analyses, the program remained cost saving when a lower level of effectiveness and a reduction in averted years of life lost were assumed. In a worst-case scenario including 20-fold higher costs, the program still had a 100% of being cost-effective and an 85% chance of being cost saving.
Conclusions
A 10-year program to control ARF/RHD in Pinar del Rio, Cuba dramatically reduced morbidity and premature mortality in children and young adults and was cost saving. The results of our analysis were robust to higher program costs and more conservative assumptions about the program’s effectiveness. It is possible that the program’s effectiveness resulted from synergies between primary and secondary prevention strategies. The findings of this study have implications for non-communicable disease policymaking in other resource-limited settings.

## 研究背景
急性风湿热（Acute rheumatic fever, ARF）与风湿性心脏病（rheumatic heart disease, RHD）仍在众多中低收入国家持续流行。迄今为止，基于人群的联合一级与二级预防策略的成本效益尚未得到评估。古巴比那尔德里奥省于1986年至1996年间实施了一项综合性ARF/RHD防控项目，本研究旨在分析该古巴项目的成本效益。

## 研究方法与结果
我们基于ARF/RHD的自然病史构建了决策树模型，将该为期10年的古巴项目与“不予干预”方案的成本与效益进行对比。本研究的目标人群为1986年居住在比那尔德里奥省的5至24岁儿童队列。我们以终生随访为时限评估了成本与健康结局，并采用医疗卫生系统视角核算成本，但未设置贴现率。研究使用的流行病学、临床及直接医疗成本参数均取自此前针对该古巴项目发表的研究数据。我们采用全球疾病负担研究（Global Burden of Disease studies）制定的标准方法，以避免的伤残调整生命年（disability-adjusted life years, DALYs）量化健康获益。成本效益可接受性阈值以每避免1个DALY对应的人均国内生产总值的1倍和3倍进行定义。我们还通过蒙特卡洛模拟（Monte Carlo simulations）开展了不确定性分析，并设置多项情景分析以探究不同防控效果与成本假设对结果的影响。
结果显示，与不予干预方案相比，该古巴项目虽在10年间总计投入202,890美元，却避免了5051个DALY的损失（每10万名学龄儿童对应避免1844个DALY），并节省了7,848,590美元（按2010年美元计价）。在情景分析中，即使假设项目效益较低、避免的生命损失年限减少，该项目仍可实现成本节约。在成本增加20倍的最差情景下，该项目仍具有100%的成本效益比，且有85%的概率实现成本节约。

## 研究结论
在古巴比那尔德里奥省开展的为期10年的ARF/RHD防控项目，大幅降低了儿童与青年群体的发病率和过早死亡率，且实现了成本节约。本研究的分析结果对项目成本升高及效益评估更为保守的假设均具有稳健性。该项目的防控效益可能源于一级与二级预防策略间的协同效应。本研究结果可为其他资源有限地区的非传染性疾病政策制定提供参考。