Pignatti Francesco, Wilking Ulla, Postmus Douwe, Wilking Nils, Delgado Julio, Bergh Jonas
Oncology & Haematology Office, European Medicines Agency, Amsterdam, Netherlands.
Department of Oncology-Pathology, Karolinska Institutet, Stockholm, Sweden.
Nat Rev Clin Oncol. 2022 Mar;19(3):207-215. doi: 10.1038/s41571-021-00584-z. Epub 2021 Dec 6.
The high prices of new anticancer drugs and the marginal added benefit perceived by some stakeholders have fuelled a debate on the value of anticancer drugs in the European Union, even though an agreed definition of what constitutes a drug's value does not exist. In this Perspective, we discuss the value of drugs from different viewpoints and objectives of decision makers: for regulators, assessment of the benefit-risk balance of a drug is a cornerstone for approval; payers rely on cost-effectiveness analyses carried out by health technology assessment agencies for reimbursement decisions; for patients, treatment choices are based on personal preferences and attitudes to risk; and clinicians can use several scales (such as the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS)) that have been developed as an attempt to measure value objectively. Although a unique definition that fully captures the concept of value is unlikely to emerge, herein we discuss the importance of understanding different perspectives, and how regulators can help to inform different decision makers.
新抗癌药物的高昂价格以及一些利益相关者所认为的边际额外获益，引发了欧盟关于抗癌药物价值的争论，尽管对于什么构成药物价值尚无商定的定义。在这篇观点文章中，我们从决策者的不同观点和目标来讨论药物的价值：对于监管机构而言，评估药物的获益-风险平衡是批准的基石；支付方依靠卫生技术评估机构开展的成本效益分析来做出报销决定；对于患者来说，治疗选择基于个人偏好和对风险的态度；而临床医生可以使用已开发出的几种量表（如欧洲肿瘤内科学会临床获益程度量表（ESMO-MCBS））来尝试客观地衡量价值。尽管不太可能出现一个能完全涵盖价值概念的独特定义，但在此我们讨论理解不同观点的重要性，以及监管机构如何能够帮助为不同的决策者提供信息。
