1 Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Vienna, Austria.
2 Pediatric clinical Research Office, Academic Medical Center, University of Amsterdam, Netherlands.
Stat Methods Med Res. 2019 Jun;28(6):1893-1910. doi: 10.1177/0962280218777896. Epub 2018 Jun 19.
Goal Attainment Scaling is an assessment instrument to evaluate interventions on the basis of individual, patient-specific goals. The attainment of these goals is mapped in a pre-specified way to attainment levels on an ordinal scale, which is common to all goals. This approach is patient-centred and allows one to integrate the outcomes of patients with very heterogeneous symptoms. The latter is of particular importance in clinical trials in rare diseases because it enables larger sample sizes by including a broader patient population. In this paper, we focus on the statistical analysis of Goal Attainment Scaling outcomes for the comparison of two treatments in randomised clinical trials. Building on a general statistical model, we investigate the properties of different hypothesis testing approaches. Additionally, we propose a latent variable approach to generate Goal Attainment Scaling data in a simulation study, to assess the impact of model parameters such as the number of goals per patient and their correlation, the choice of discretisation thresholds and the type of design (parallel group or cross-over). Based on our findings, we give recommendations for the design of clinical trials with a Goal Attainment Scaling endpoint. Furthermore, we discuss an application of Goal Attainment Scaling in a clinical trial in mastocytosis.
目标达成度评分是一种评估干预措施的工具，它基于个体、患者特定的目标进行评估。这些目标的达成情况以预先指定的方式映射到一个有序尺度上的达成水平，这个尺度对所有目标都是通用的。这种方法以患者为中心，允许整合具有非常异质症状的患者的结果。在罕见病的临床试验中，这一点尤为重要，因为它通过纳入更广泛的患者群体来实现更大的样本量。在本文中，我们专注于目标达成度评分的统计分析，以比较随机临床试验中的两种治疗方法。在一般统计模型的基础上，我们研究了不同假设检验方法的性质。此外，我们提出了一种潜在变量方法来在模拟研究中生成目标达成度评分数据，以评估模型参数（如每个患者的目标数量及其相关性、离散化阈值的选择以及设计类型（平行组或交叉））的影响。基于我们的发现，我们为具有目标达成度评分终点的临床试验设计提供了建议。此外，我们还讨论了在肥大细胞增多症临床试验中应用目标达成度评分的情况。
