Choon-Quinones Mimi, Zelei Tamás, Németh Bertalan, Tóth Manna, Jia Xiao Yang, Barnett Mike, Keown Paul, Durie Brian, Harousseau Jean-Luc, Hose Dirk, Kaló Zoltán
International Myeloma Foundation, North Hollywood, CA, USA.
Syreon Research Institute, Budapest, Hungary.
J Med Econ. 2023 Jan-Dec;26(1):110-119. doi: 10.1080/13696998.2022.2144056.
The goal of this study was to review the economic evaluations of health technologies in multiple myeloma (MM) and provide guidance and recommendations for future health economic analyses.
A systemic literature review (SLR) was conducted on original economic assessment studies and structured review papers focusing on the studies in MM. The search was limited to English language papers published from 1 January 2000 onwards. Publications not applying any type of modelling methodology to describe disease progression and patient pathways over a specific time horizon were excluded.
A total of 2,643 publications were initially identified, of which 148 were eligible to be included in the full-text review phase. From these, 49 publications were included in the final analysis. Most published health economic analyses supported by models came from high-income countries. Evaluations from middle-income countries were rarely published. Diagnostic technologies were rarely modelled and integrated care had not been modelled. Very few models investigated MM treatments from a societal perspective and there was a relative lack of evaluations regarding minimal residual disease (MRD).
Limitations of the publications included differences between trial populations and modelled populations, justification of methods, lack of confounder analyses, and small trial populations. Limitations of our study included the infeasibility of comparing MM economic evaluations due to the significant variance in modelled therapeutic lines and indications, and the relative scarcity of published economic evaluations from non-high-income countries.
As published economic models lacked many of the elements of the complex and heterogeneous patient pathways in MM and they focused on single decision problems, a thorough, open-source economic whole disease modelling framework is needed to assess the economic value of a wide range of technologies across countries with various income levels with a more detailed view on MM, by including patient-centric and societal aspects.
本研究的目的是回顾多发性骨髓瘤(MM)健康技术的经济评估,并为未来的健康经济分析提供指导和建议。
对专注于MM研究的原始经济评估研究和结构化综述论文进行了系统文献综述(SLR)。检索限于2000年1月1日起发表的英文论文。未应用任何类型建模方法来描述特定时间范围内疾病进展和患者路径的出版物被排除。
初步确定了2643篇出版物,其中148篇有资格纳入全文综述阶段。其中,49篇出版物纳入最终分析。大多数由模型支持的已发表健康经济分析来自高收入国家。来自中等收入国家的评估很少发表。诊断技术很少被建模,综合护理也未被建模。很少有模型从社会角度研究MM治疗,并且关于微小残留病(MRD)的评估相对较少。
出版物的局限性包括试验人群与建模人群之间的差异、方法的合理性、缺乏混杂因素分析以及试验人群规模小。我们研究的局限性包括由于建模治疗线和适应症的显著差异而无法比较MM经济评估,以及非高收入国家已发表经济评估相对较少。
由于已发表的经济模型缺乏MM复杂且异质患者路径的许多要素,并且它们侧重于单一决策问题,因此需要一个全面的、开源的经济全疾病建模框架,以更详细地从MM角度评估不同收入水平国家各种技术的经济价值,包括以患者为中心和社会方面。