Francois Clement, Zhou Junwen, Pochopien Michał, Achour Leila, Toumi Mondher
Public Health Department, Research Unit EA 3279, Aix-Marseille University, Marseille, France.
University Paris Dauphine, Paris, France.
Recent Results Cancer Res. 2019;213:25-38. doi: 10.1007/978-3-030-01207-6_3.
In this chapter, we will present and discuss the challenges of assessing oncology products from a health economic perspective. We will provide a brief introduction on the need for economic evaluation in health care and focus on cost-effectiveness and comparative aspects of the evaluation of oncology products, which are of paramount interest to HTA decision-making bodies using economic evaluation in their decision-making framework. As the burden of oncology is well-documented, we do not discuss it in detail here. Before we address the specific issue of oncology, we will briefly define the critical aspects of HTA assessment and also define what a cost-effectiveness analysis is and why economic modelling is the most appropriate tool to assess the cost-effectiveness of oncology products. We will touch upon the prices of oncology drugs and the questions that high prices raise regarding funding and availability. We then present an overview of the general structure of an oncology cost-effectiveness model. Usually, this is quite simple, representing response, progression, advanced-stage disease and death. Despite the relative simplicity of these models, some issues may render the evaluation more complex; we will touch upon these in this chapter: Issue with clinical inputs due to the design of randomised clinical trials (e.g. cross-over designs involving a treatment switch) Need for survival extrapolation and limitations of current parametric models Rare conditions with limited economic and comparative evidence available High pace of clinical development Finally, we will conclude with a discussion of the uncertainty around the evaluation of oncology products and the major evolution expected in health economics in oncology.
在本章中，我们将阐述并讨论从卫生经济学角度评估肿瘤学产品所面临的挑战。我们将简要介绍卫生保健中进行经济评估的必要性，并着重探讨肿瘤学产品评估中的成本效益及比较方面，这对于在决策框架中运用经济评估的卫生技术评估（HTA）决策机构而言至关重要。鉴于肿瘤负担已有充分记录，我们在此不再详述。在探讨肿瘤学的具体问题之前，我们将简要界定HTA评估的关键方面，同时定义成本效益分析是什么，以及为何经济建模是评估肿瘤学产品成本效益的最合适工具。我们将提及肿瘤药物的价格以及高价所引发的关于资金和可及性的问题。接着，我们将概述肿瘤学成本效益模型的总体结构。通常，该结构相当简单，涵盖反应、进展、晚期疾病和死亡情况。尽管这些模型相对简单，但某些问题可能会使评估更为复杂；我们将在本章中探讨这些问题：由于随机临床试验的设计导致的临床输入问题（例如涉及治疗转换的交叉设计）生存外推的必要性以及当前参数模型的局限性经济和比较证据有限的罕见病症临床开发的快速步伐最后，我们将以对肿瘤学产品评估的不确定性以及肿瘤学卫生经济学预期的主要发展变化的讨论作为总结。
