Department of Economics, Stockholm School of Economics, Stockholm, Sweden.
Clin Cancer Res. 2013 Jan 1;19(1):6-11. doi: 10.1158/1078-0432.CCR-12-1819.
Health technology assessment (HTA) has become the key health policy instrument for managing the introduction and use of new oncology drugs in Europe. While the methodology of technology assessment, including calculations of cost-effectiveness, is applicable in principle also to oncology, the implementation in practice has its specific problems and consequences. Most of them are linked to the specific need to do the assessment early in the development, with limited data on outcome in clinical practice. Technology assessments ask for estimates of gains in mean survival, whereas trials are powered to study differences in progression-free or overall median survival. The development of targeted therapies and personalized cancer medicine offers opportunities but also increases the complexity of the assessment. Joint assessment of a diagnostic and a new treatment increases the number intervention strategies that must be considered, and thus the need for data. The translation from efficacy in trials to relative effectiveness in clinical practice must also be considered. The close link between pricing of new oncology drugs and their cost-effectiveness makes the use of technology assessment for policy decisions complicated for all stakeholders involved. But without an obvious alternative that is better, the likely future is that HTA will play an increasing role in informing policy decisions aimed at evidence-based cancer care.
健康技术评估（HTA）已成为管理欧洲新肿瘤药物引入和使用的关键卫生政策工具。虽然技术评估方法，包括成本效益计算，原则上也适用于肿瘤学，但在实践中的实施存在其特定的问题和后果。其中大多数与在开发早期进行评估的特殊需求有关，因为在临床实践中关于结果的数据有限。技术评估需要估计平均生存时间的增加，而试验则有能力研究无进展或总体中位生存时间的差异。靶向治疗和个体化癌症治疗的发展提供了机会，但也增加了评估的复杂性。诊断和新治疗方法的联合评估增加了必须考虑的干预策略数量，因此需要更多的数据。还必须考虑从临床试验中的疗效到临床实践中的相对疗效的转化。新肿瘤药物定价与其成本效益之间的紧密联系使得所有相关利益相关者在做出政策决策时都难以使用技术评估。但是，由于没有更好的明显替代方案，未来 HTA 很可能会在旨在基于证据的癌症护理的政策决策中发挥越来越重要的作用。
