Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, PO Box 1738, 3000 DR, Rotterdam, The Netherlands,
Pharmacoeconomics. 2013 Oct;31(10):863-76. doi: 10.1007/s40273-013-0084-z.
National regulatory agencies often have to use cost-effectiveness (CE) data from multinational randomized controlled trials (RCTs) for national decision making on reimbursement of new drugs. We need to make the best use of these patient-level data to obtain estimates of country-specific CE. Several methods, ranging from simple to statistically complex, have existed for years. We investigated which of these methods are used to estimate CE ratios in economic evaluations performed alongside recent, multinational RCTs that enrolled at least 500 patients.
In this systematic literature review, studies were classified based on whether resource use, unit costs, health outcomes and utility value sets were obtained from all countries, a subset of countries or one country. We recorded if the study presented trial-wide and country-specific CE results and reported the statistical analyses that were used to estimate them.
We included 21 studies, of which the majority used measurements of health care utilization and health outcomes from all countries to estimate CE. Thirteen studies used a one-country valuation of health care utilization; six used a multi-country valuation. Despite the availability of country-specific utility value sets, none of the studies that presented quality-adjusted life-years (QALYs) used multi-country valuation. Valuation of health care utilization and health outcomes was not always consistent within a study: three studies combined a multi-country valuation of health care utilization, with a one-country valuation of health outcomes. Most studies calculated trial-wide CE estimates, while 11 studies calculated country- or region-specific estimates. Thirteen studies used relatively simple methods, which do not take the possible interaction between the country and treatment effect on health care utilization and health outcomes into account. Eight studies used more advanced statistical methods. Three of them used a fixed-effects modeling approach. Five studies explicitly took the hierarchical structure of the data into account, which leads to more appropriate estimates of population average results and associated standard errors. In this way, they help improve transferability of the published results.
Based on this systematic review, we concluded that the uptake of more advanced statistical methods has been relatively slow, while simpler naïve methods are still routinely employed.
国家监管机构在为新药报销做出国家决策时，经常需要使用来自跨国随机对照试验（RCT）的成本效益（CE）数据。我们需要充分利用这些患者水平数据，以获得针对特定国家的 CE 估计值。多年来，已经存在从简单到复杂统计的多种方法。我们研究了在最近的、至少有 500 名患者入组的跨国 RCT 中进行的经济评估中，使用了哪些方法来估计 CE 比。
在这项系统文献综述中，根据资源使用、单位成本、健康结果和效用值集是否来自所有国家、部分国家或一个国家，对研究进行了分类。我们记录了研究是否呈现了试验范围和国家特定的 CE 结果，并报告了用于估计它们的统计分析。
我们纳入了 21 项研究，其中大多数研究使用所有国家的卫生保健利用和健康结果测量来估计 CE。13 项研究使用一国卫生保健利用估值；6 项研究使用多国估值。尽管有国家特定的效用值集，但没有一项呈现质量调整生命年（QALY）的研究使用多国估值。一项研究内的卫生保健利用和健康结果的估值并不总是一致的：三项研究结合了多国卫生保健利用估值和一国健康结果估值。大多数研究计算了试验范围的 CE 估计值，而 11 项研究计算了国家或地区特定的估计值。13 项研究使用了相对简单的方法，这些方法没有考虑到国家和治疗效果对卫生保健利用和健康结果的可能相互作用。八项研究使用了更先进的统计方法。其中三项研究使用了固定效应建模方法。五项研究明确考虑了数据的层次结构，这可以更恰当地估计人群平均结果和相关标准误差。通过这种方式，他们有助于提高已发表结果的可转移性。
根据这项系统综述，我们得出结论，虽然仍经常使用简单的天真方法，但更先进的统计方法的采用相对较慢。
