Puig-Peiro Ruth, Mason Anne, Mestre-Ferrandiz Jorge, Towse Adrian, McGrath Clare, Jonsson Bengt
Servei Català de la Salut (CatSalut)
University of York.
Int J Technol Assess Health Care. 2015;31(6):371-9. doi: 10.1017/S0266462315000720. Epub 2016 Jan 20.
Pharmaceuticals' relative effectiveness has come to the fore in the policy arena, reflecting the need to understand how relative efficacy (what can work) translates into added benefit in routine clinical use (what does work). European payers and licensing authorities assess value for money and post-launch benefit-risk profiles, and efforts to standardize assessments of relative effectiveness across the European Union (EU) are under way. However, the ways that relative effectiveness differs across EU healthcare settings are poorly understood.
To understand which factors influence differences in relative effectiveness, we developed an analytical framework that treats the healthcare system as a health production function. Using evidence on breast cancer from England, Spain, and Sweden as a case study, we investigated the reasons why the relative effectiveness of a new drug might vary across healthcare systems. Evidence was identified from a literature review and national clinical guidance.
The review included thirteen international studies and thirty country-specific studies. Cross-country differences in population age structure, deprivation, and educational attainment were consistently associated with variation in outcomes. Screening intensity appeared to drive differences in survival, although the impact on mortality was unclear.
The way efficacy translates into relative effectiveness across health systems is likely to be influenced by a range of complex and interrelated factors. These factors could inform government and payer policy decisions on ways to optimize relative effectiveness, and help increase understanding of the potential transferability of data on relative effectiveness from one health system to another.
药物的相对疗效在政策领域已成为焦点，这反映出需要了解相对疗效（可能有效的情况）如何转化为常规临床使用中的额外益处（实际有效的情况）。欧洲的支付方和许可当局会评估性价比以及上市后效益风险状况，并且正在努力在欧盟范围内对相对疗效评估进行标准化。然而，人们对欧盟不同医疗环境下相对疗效的差异方式了解甚少。
为了了解哪些因素会影响相对疗效的差异，我们构建了一个将医疗系统视为健康生产函数的分析框架。以英国、西班牙和瑞典关于乳腺癌的证据作为案例研究，我们调查了一种新药的相对疗效在不同医疗系统中可能存在差异的原因。证据来自文献综述和国家临床指南。
该综述纳入了13项国际研究和30项针对特定国家的研究。人口年龄结构、贫困程度和教育水平的跨国差异始终与结果的差异相关。筛查强度似乎推动了生存率的差异，尽管对死亡率的影响尚不清楚。
疗效在不同卫生系统中转化为相对疗效的方式可能受到一系列复杂且相互关联的因素影响。这些因素可为政府和支付方关于优化相对疗效方法的政策决策提供参考，并有助于增进对相对疗效数据从一个卫生系统向另一个卫生系统潜在可转移性的理解。
