Gherardi Alexandre, Roze Stéphane, Kuijvenhoven Johan, Ghatnekar Ola, Yip Sonderegger Yum Lina
a HEVA HEOR , Lyon , France.
b Spaarne Gasthuis, Haarlem Zuid , Haarlem , the Netherlands.
J Med Econ. 2018 Sep;21(9):869-877. doi: 10.1080/13696998.2018.1484371. Epub 2018 Jun 22.
Budesonide with multi-matrix technology (MMX) is an oral corticosteroid, shown to have high topical activity against ulcerative colitis (UC) while maintaining low systemic bioavailability with few adverse events. The aim of this study was to evaluate the cost-effectiveness of budesonide MMX versus commonly used corticosteroids, in the second-line treatment of active mild-to-moderate UC in the Netherlands.
An eight-state Markov model with an 8 week cycle length captured remission, four distinct therapy stages, hospitalization, possible colectomy and mortality. Remission probability for budesonide MMX was based on the CORE-II study. Population characteristics were derived from the Dutch Inflammatory Bowel Disease South Limburg cohort (n = 598) and included patients with proctitis (39%), left-sided (42%) and extensive disease (19%). Comparators (topical budesonide foam and enema, oral budesonide and prednisolone) were selected based on current Dutch clinical practice. Treatment effects were evaluated by network meta-analysis using a Bayesian framework. Cost-effectiveness analysis was performed over a 5 year time horizon from a societal perspective, with costs, health-state and adverse event utilities derived from published sources. Outcomes were weighted by disease extent distribution and corresponding comparators.
Budesonide MMX was associated with comparable quality-adjusted life year (QALY) gain versus foam and oral formulations (+0.01 QALYs) in the total UC population, whilst being cost-saving (EUR 366 per patient). Probabilistic sensitivity analysis evaluated an 86.6% probability of budesonide MMX being dominant (cost-saving with QALY gain) versus these comparators. Exploratory analysis showed similar findings versus prednisolone.
Differing definitions of trial end-points and remission across trials meant indirect comparison was not ideal. However, in the absence of head-to-head clinical data, these comparisons are reasonable alternatives and currently offer the only comparison of second-line UC treatments.
In the present analysis, budesonide MMX was shown to be cost-effective versus comparators in the total UC population, for the second-line treatment of active mild-to-moderate UC in the Netherlands.
布地奈德多基质技术（MMX）是一种口服皮质类固醇，已显示出对溃疡性结肠炎（UC）具有高局部活性，同时保持低全身生物利用度且不良事件较少。本研究的目的是评估在荷兰对活动性轻至中度UC进行二线治疗时，布地奈德MMX与常用皮质类固醇相比的成本效益。
一个具有8周周期长度的八状态马尔可夫模型涵盖缓解、四个不同的治疗阶段、住院、可能的结肠切除术和死亡率。布地奈德MMX的缓解概率基于CORE-II研究。人群特征来自荷兰南林堡炎症性肠病队列（n = 598），包括直肠炎患者（39%）、左侧疾病患者（42%）和广泛性疾病患者（19%）。根据荷兰当前的临床实践选择对照药物（局部用布地奈德泡沫剂和灌肠剂、口服布地奈德和泼尼松龙）。使用贝叶斯框架通过网络荟萃分析评估治疗效果。从社会角度在5年时间范围内进行成本效益分析，成本、健康状态和不良事件效用值来自已发表的资料。结果根据疾病范围分布和相应的对照药物进行加权。
在整个UC人群中，与泡沫剂和口服制剂相比，布地奈德MMX的质量调整生命年（QALY）增益相当（+0.01 QALYs），同时节省成本（每位患者366欧元）。概率敏感性分析评估布地奈德MMX相对于这些对照药物具有主导性（节省成本且QALY增益）的概率为86.6%。探索性分析显示与泼尼松龙相比有类似结果。
不同试验中试验终点和缓解的定义不同，这意味着间接比较并不理想。然而，在缺乏直接头对头临床数据的情况下，这些比较是合理的替代方法，目前是二线UC治疗的唯一比较方式。
在本分析中，对于荷兰活动性轻至中度UC的二线治疗，布地奈德MMX在整个UC人群中与对照药物相比显示出成本效益。
