Hunger Matthias, Eriksson Jennifer, Regnier Stephane A, Mori Katsuya, Spertus John A, Cristino Joaquim
Real-World Evidence Strategy & Analytics, ICON plc, Munich, Germany.
Real-World Evidence Strategy & Analytics, ICON plc, Stockholm, Sweden.
MDM Policy Pract. 2020 Dec 7;5(2):2381468320971606. doi: 10.1177/2381468320971606. eCollection 2020 Jul-Dec.
Health technology assessment bodies in several countries, including Japan and the United Kingdom, recommend mapping techniques to obtain utility scores in clinical trials that do not have a preference-based measure of health. This study sought to develop mapping algorithms to predict EQ-5D-3L scores from the Kansas City Cardiomyopathy Questionnaire (KCCQ) in patients with heart failure (HF). Data from the randomized, double-blind PARADIGM-HF trial were analyzed, and EQ-5D-3L scores were calculated using the Japanese and UK value sets. Several different model specifications were explored to best fit EQ-5D data collected at baseline with KCCQ scores, including ordinary least square regression, two-part, Tobit, and three-part models. Generalized estimating equations models were also fitted to analyze longitudinal EQ-5D data. To validate model predictions, the data set was split into a derivation ( = 4,465) from which the models were developed and a separate sample ( = 1,892) for validation. There were only small differences between the different model classes tested. Model performance and predictive power was better for the item-level models than for the models including KCCQ domain scores. statistics for the item-level models ranged from 0.45 to 0.52. Mean absolute error in the validation sample was 0.10 for the models using the Japanese value set and 0.114 for the UK models. All models showed some underprediction of utility above 0.75 and overprediction of utility below 0.5, but performed well for population-level estimates. Using data from a large clinical trial in HF, we found that EQ-5D-3L scores can be estimated from responses to the KCCQ and can facilitate cost-utility analysis from existing HF trials where only the KCCQ was administered. Future validation in other HF populations is warranted.
包括日本和英国在内的几个国家的卫生技术评估机构建议采用映射技术,以便在那些没有基于偏好的健康测量方法的临床试验中获得效用评分。本研究旨在开发映射算法,以根据堪萨斯城心肌病问卷(KCCQ)预测心力衰竭(HF)患者的EQ-5D-3L评分。对随机、双盲的PARADIGM-HF试验数据进行了分析,并使用日本和英国的值集计算了EQ-5D-3L评分。探索了几种不同的模型规格,以使其与基线时收集的KCCQ评分的EQ-5D数据最佳拟合,包括普通最小二乘回归、两部分模型、托比特模型和三部分模型。还拟合了广义估计方程模型,以分析纵向EQ-5D数据。为了验证模型预测,将数据集分为用于开发模型的推导数据集(n = 4465)和用于验证的单独样本(n = 1892)。所测试的不同模型类别之间只有微小差异。项目级模型的模型性能和预测能力优于包含KCCQ领域评分的模型。项目级模型的R统计量范围为0.45至0.52。使用日本值集的模型在验证样本中的平均绝对误差为0.10,英国模型为0.114。所有模型在效用高于0.75时均表现出一定程度的低估,在效用低于0.5时则表现出高估,但在人群水平估计方面表现良好。利用HF大型临床试验的数据,我们发现可以根据对KCCQ的回答来估计EQ-5D-3L评分,并且可以促进仅实施了KCCQ的现有HF试验的成本效用分析。未来有必要在其他HF人群中进行验证。