Background

Cost-utility analysis typically relies on preference-based measures (PBMs). While generic PBMs are widely used, disease-specific PBMs can capture aspects relevant for certain patient populations. Here the EORTC QLU-C10D, a cancer-specific PBM based on the QLQ-C30, is validated using Dutch trial data with the EQ-5D-3L as a generic comparator measure.

Methods

We retrospectively analysed data from four Dutch randomised controlled trials (RCTs) comprising the EORTC QLQ-C30 and the EQ-5D-3L. Respective Dutch value sets were applied. Correlations between the instruments were calculated for domains and index scores. Bland–Altman plots and intra-class correlations (ICC) displayed agreement between the measures. Independent and paired t-tests, effect sizes and relative validity indices were used to determine the instruments’ performance in detecting clinically known-group differences and health changes over time.

Results

We analysed data from 602 cancer patients from four different trials. In overall, the EORTC QLU-C10D showed good relative validity with the EQ-5D-3L as a comparator (correlations of index scores r = 0.53–0.75, ICCs 0.686–0.808, conceptually similar domains showed higher correlations than dissimilar domains). Most importantly, it detected 63% of expected clinical group differences and 50% of changes over time in patients undergoing treatment. Both instruments showed poor performance in survivors. Detection rate and measurement efficiency were clearly higher for the QLU-C10D than for the EQ-5D-3L.

Conclusions

The Dutch EORTC QLU-C10D showed good comparative validity in patients undergoing treatment. Our results underline the benefit that can be achieved by using a cancer-specific PBM for generating health utilities for cancer patients from a measurement perspective.

中文翻译：

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EORTC QLU-C10D 是荷兰有效的基于癌症特定偏好的成本效益和卫生技术评估指标

背景

成本效用分析通常依赖于基于偏好的衡量标准 (PBM)。虽然通用 PBM 得到广泛使用，但针对特定疾病的 PBM 可以捕获与某些患者群体相关的方面。在此，EORTC QLU-C10D 是一种基于 QLQ-C30 的癌症特异性 PBM，使用荷兰试验数据和 EQ-5D-3L 作为通用比较测量进行了验证。

方法

我们回顾性分析了四项荷兰随机对照试验 (RCT) 的数据，包括 EORTC QLQ-C30 和 EQ-5D-3L。应用各自的荷兰值集。计算了工具之间的领域和指数得分的相关性。Bland-Altman 图和组内相关性 (ICC) 显示测量值之间的一致性。使用独立和配对t检验、效应大小和相对有效性指数来确定仪器在检测临床已知群体差异和健康随时间变化方面的性能。

结果

我们分析了来自四项不同试验的 602 名癌症患者的数据。总体而言，EORTC QLU-C10D 以 EQ-5D-3L 作为比较器显示出良好的相对有效性（指数得分相关性r  = 0.53-0.75，ICC 0.686-0.808，概念上相似的域比不相似的域显示出更高的相关性）。最重要的是，它检测到了接受治疗的患者中 63% 的预期临床组差异和 50% 的随时间变化。两种仪器在幸存者中的表现都很差。QLU-C10D 的检测率和测量效率明显高于 EQ-5D-3L。

结论

荷兰 EORTC QLU-C10D 在接受治疗的患者中表现出良好的比较有效性。我们的结果强调了从测量角度使用癌症特异性 PBM 为癌症患者生成健康实用程序可以实现的好处。