Syreon Research Romania, Targu Mures, Romania.
George Emil Palade University of Medicine, Pharmacy, Science and Technology, Targu Mures, Romania.
BioDrugs. 2023 Mar;37(2):219-233. doi: 10.1007/s40259-023-00583-9. Epub 2023 Feb 16.
Economic evaluations are widely used to predict the economic impact of new treatment alternatives. Comprehensive economic reviews in the field of chronic lymphocytic leukemia (CLL) are warranted to supplement the existing analyses focused on specific therapeutic areas.
A systematic literature review was conducted based on literature searches in Medline and EMBASE to summarize the published health economics models related to all types of CLL therapies. Narrative synthesis of relevant studies was performed focusing on compared treatments, patient populations, modelling approaches and key findings.
We included 29 studies, the majority of which were published between 2016 and 2018, when data from large clinical trials in CLL became available. Treatment regimens were compared in 25 cases, while the remaining four studies considered treatment strategies with more complex patient pathways. Based on the review results, Markov modelling with a simple structure of three health states (progression-free, progressed, death) can be considered as the traditional basis to simulate cost effectiveness. However, more recent studies added further complexity, including additional health states for different therapies (e.g. best supportive care or stem cell transplantation), for progression-free state (e.g. by differentiating between with or without treatment), or for response status (i.e. partial response and complete response).
As personalized medicine is increasingly gaining recognition, we expect that future economic evaluations will also incorporate new solutions, which are necessary to capture a larger number of genetic and molecular markers and more complex patient pathways with individual patient-level allocation of treatment options and thus economic assessments.
经济评估被广泛用于预测新治疗方案的经济影响。有必要对慢性淋巴细胞白血病(CLL)领域进行全面的经济评估,以补充针对特定治疗领域的现有分析。
根据 Medline 和 EMBASE 的文献检索,进行了系统的文献综述,以总结与所有类型 CLL 治疗相关的已发表的健康经济学模型。重点关注比较治疗、患者人群、建模方法和主要发现,对相关研究进行叙述性综合。
我们共纳入 29 项研究,其中大多数发表于 2016 年至 2018 年,当时 CLL 的大型临床试验数据可用。在 25 项研究中比较了治疗方案,而其余 4 项研究考虑了具有更复杂患者路径的治疗策略。根据综述结果,具有简单三种健康状态(无进展、进展、死亡)的 Markov 模型可以被视为模拟成本效益的传统基础。然而,最近的研究增加了更多的复杂性,包括不同疗法的其他健康状态(例如,最佳支持治疗或干细胞移植)、无进展状态(例如,区分是否治疗)或反应状态(即部分缓解和完全缓解)。
随着个体化医学的认可度越来越高,我们预计未来的经济评估也将纳入新的解决方案,这对于捕捉更多的遗传和分子标志物以及更复杂的患者路径,包括根据个体患者的治疗选择进行分配,以及经济评估,是必要的。