Latimer Nicholas R, Rutherford Mark J
University of Sheffield, Sheffield, UK.
Delta Hat, Nottingham, UK.
Pharmacoeconomics. 2024 Oct;42(10):1073-1090. doi: 10.1007/s40273-024-01406-7. Epub 2024 Jul 5.
There is increasing interest in the use of cure modelling to inform health technology assessment (HTA) due to the development of new treatments that appear to offer the potential for cure in some patients. However, cure models are often not included in evidence dossiers submitted to HTA agencies, and they are relatively rarely relied upon to inform decision-making. This is likely due to a lack of understanding of how cure models work, what they assume, and how reliable they are. In this tutorial we explain why and when cure models may be useful for HTA, describe the key characteristics of mixture and non-mixture cure models, and demonstrate their use in a range of scenarios, providing Stata code. We highlight key issues that must be taken into account by analysts when fitting these models and by reviewers and decision-makers when interpreting their predictions. In particular, we note that flexible parametric non-mixture cure models have not been used in HTA, but they offer advantages that make them well suited to an HTA context when a cure assumption is valid but follow-up is limited.
由于新治疗方法的发展,这些新疗法似乎为某些患者提供了治愈的潜力,因此使用治愈模型为卫生技术评估(HTA)提供信息的兴趣与日俱增。然而,治愈模型通常不包含在提交给HTA机构的证据档案中,并且相对很少被用于为决策提供信息。这可能是由于对治愈模型如何工作、它们的假设以及它们的可靠性缺乏了解。在本教程中,我们解释了治愈模型为何以及何时可能对HTA有用,描述了混合和非混合治愈模型的关键特征,并展示了它们在一系列场景中的使用,同时提供了Stata代码。我们强调了分析师在拟合这些模型时以及评审人员和决策者在解释其预测时必须考虑的关键问题。特别是,我们注意到灵活的参数化非混合治愈模型尚未在HTA中使用,但当治愈假设有效但随访有限时,它们具有一些优势,使其非常适合HTA背景。