European Institute of Oncology, IRCCS, 20141 Milan, Italy.
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Curr Oncol. 2022 Aug 16;29(8):5774-5791. doi: 10.3390/curroncol29080455.
Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data.
Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review.
A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks.
A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation.
癌症药物的进步带来了切实的健康影响，但已批准的癌症药物的获益程度可能存在很大差异。卫生技术评估（HTA）是一个多学科的过程，通过对卫生技术进行系统的价值评估来为资源配置提供信息。本文综述了肿瘤试验设计和安全性-疗效数据不确定性给癌症药物 HTA 带来的挑战。
通过对多个数据库（PubMed、Scopus 和 Google Scholar）和灰色文献（公共卫生机构和政府报告）的检索，为这篇政策叙述性综述提供了信息。
临床试验和其他相关证据来源缺乏稳健的疗效-安全性数据，使得癌症药物的 HTA 具有挑战性。近年来，通过加速途径批准癌症药物的情况有所增加，其中患者选择的替代终点或生物标志物已被广泛使用。使用这些替代终点会在将替代指标转化为以患者为中心的临床（生存和生活质量）和经济（成本效益和预算影响）有意义的结果方面产生不确定性，可能会对将稀缺的卫生资源转移到低价值或有害的干预措施产生影响。潜在的解决方案包括监管和 HTA 当局之间的政策协调、承诺生成稳健的上市后疗效-安全性数据、通过风险分担协议管理不确定性，以及使用价值框架。
缺乏稳健的疗效-安全性数据是进行癌症药物 HTA 的核心问题，可能导致资源配置信息不足。
