METHODS: The analysis was based on data from 172 participants of the KOPAL multi-centre, cluster randomised controlled trial. Patients with advanced congestive heart failure (CHF), chronic obstructive pulmonary disease (COPD), or dementia were randomised into intervention group (IG) and control group (CG, usual care). Cost-effectiveness was examined over 48 weeks from a societal and healthcare payer\'s perspective. Effects were quantified as quality-adjusted life years (QALYs, EQ-5D-5L). Incremental cost-effectiveness ratios were calculated and cost-effectiveness acceptability curves were constructed.
RESULTS: Baseline imbalances in costs and effects could be observed between IG and CG. After adjusting for these imbalances and compared to the CG, mean costs in the IG were non-significantly higher from a societal and lower from a payer\'s perspective. On the effect side, the IG had marginally lower mean QALYs. The results were characterized by high statistical uncertainty, indicated by large confidence intervals for the cost and effect differences between groups and probabilities of cost-effectiveness between 18% and 65%, depending on the perspective and willingness-to-pay.
CONCLUSIONS: Based on the results of this study, the cost-effectiveness of the KOPAL intervention was uncertain. The results highlighted (methodological) challenges of economic evaluations in patients with chronic, non-malignant diseases related to sample size, heterogeneity of participants, and the way the intervention effectiveness is typically captured in economic evaluations.
方法:分析基于KOPAL多中心的172名参与者的数据,整群随机对照试验。晚期充血性心力衰竭(CHF)患者,慢性阻塞性肺疾病(COPD),或痴呆被随机分为干预组(IG)和对照组(CG,常规护理)。从社会和医疗保健支付者的角度,在48周内检查了成本效益。效果量化为质量调整生命年(QALYs,EQ-5D-5L)。计算了增量成本效益比,并构建了成本效益可接受性曲线。
结果:可以观察到IG和CG之间成本和效果的基线不平衡。在调整这些不平衡并与CG进行比较后,从社会角度来看,IG的平均成本并不显著较高,而从付款人的角度来看则较低。在效果方面,IG的平均QALY略低。结果具有较高的统计不确定性,由群体之间的成本和效果差异的大置信区间以及18%至65%的成本效益概率表明,取决于视角和支付意愿。
结论:根据本研究的结果,KOPAL干预的成本-效果不确定.结果强调了慢性病患者经济评估的(方法论)挑战,与样本量相关的非恶性疾病,参与者的异质性,以及干预有效性通常在经济评估中被捕获的方式。