Norwich Medical School, Norwich Research Park, University of East Anglia, Norwich, Norfolk NR4 7TJ, UK.
Norwich Medical School, Norwich Research Park, University of East Anglia, Norwich, Norfolk NR4 7TJ, UK.
J Cyst Fibros. 2019 Jul;18(4):452-460. doi: 10.1016/j.jcf.2019.01.007. Epub 2019 Feb 7.
Cystic Fibrosis (CF) is a heritable chronic condition. Due to the genetic and progressive nature of CF, a number of interventions are available for the condition. In the United Kingdom (U.K.) average annual cost of CF treatment is between €49,000 to €76,000 (2012) per patient [1]. A review of health economic modelling studies is warranted to provide decision makers and researchers with an in depth understanding of modelling practices in CF and guidance for future research.
Online searches were performed in the 5 databases, studies were included if they were: a) Model based economic evaluation for management of Cystic Fibrosis. Articles were restricted to English language only, but no restriction was applied on publication year.
Nine studies were reviewed, most were Markov cohort models. Models evaluated pharmaceutical interventions and drug adherence. Modelling structure was consistent across most articles and a range of sources were used to populate the models. Cost and utility data were based on different sources and elicitation methods respectively. The majority of models failed to incorporate significant health events which impact both cost and disease progression.
In our review we observed a lack of, application of European Medicines Agency (EMA) guidelines for clinical trial endpoints, model structure justifications and lastly, health-related quality of life derived utility information around important clinical events. Future work around conceptual modelling of CF progression, utility valuation of significant health events and meeting EMA guidelines for trial reporting is encouraged.
囊性纤维化（CF）是一种遗传性慢性疾病。由于 CF 的遗传和进行性特征，有许多干预措施可用于治疗这种疾病。在英国（U.K.），每位患者 CF 的平均年治疗成本在 49000 至 76000 欧元之间（2012 年）[1]。需要对健康经济模型研究进行审查，以便为决策者和研究人员提供深入了解 CF 建模实践的机会，并为未来的研究提供指导。
在 5 个数据库中进行了在线搜索，符合以下条件的研究被纳入：a）用于管理囊性纤维化的基于模型的经济评估。文章仅限于英语，且对发表年份没有限制。
共综述了 9 项研究，大多数为马尔可夫队列模型。模型评估了药物干预和药物依从性。模型结构在大多数文章中是一致的，并且使用了各种来源来填充模型。成本和效用数据分别基于不同的来源和启发方法。大多数模型未能纳入影响成本和疾病进展的重大健康事件。
在我们的综述中，我们观察到缺乏对临床试验终点的欧洲药品管理局（EMA）指南的应用、模型结构的理由，以及对重要临床事件的健康相关生活质量的效用信息的缺乏。鼓励围绕 CF 进展的概念建模、重大健康事件的效用估值以及满足 EMA 试验报告指南开展未来的工作。
