Harbeck Nadia, Schneeweiss Andreas, Thuss-Patience Peter, Miller Kurt, Garbe Claus, Griesinger Frank, Eberhardt Wilfried E E, Klussmann Jens P, Wollenberg Barbara, Grimm Marc-Oliver, Zander Thomas, Lüftner Diana
Breast Center, Department of Obstetrics and Gynecology and Comprehensive Cancer Center, University of Munich (LMU), 81377 Munich, Germany.
National Center for Tumor Diseases, Division of Gynecologic Oncology, University Hospital and German Cancer Research Center, 69120 Heidelberg, Germany.
Eur J Cancer. 2021 Apr;147:40-50. doi: 10.1016/j.ejca.2021.01.006. Epub 2021 Feb 19.
Health technology assessment (HTA) of clinical and economic value of a new intervention is an integral step in providing the access of patients to innovative cancer care and treatment. Overall survival (OS) is the preferred criterion for demonstrating the therapeutic efficacy in HTA given its direct clinical and patient relevance. However, with often long life expectancy of patients with early cancer, analysis of OS becomes less practical. Partially due to this reason, pathological complete response (pCR) and time-to-event end-points like disease-free survival are frequently incorporated into the pivotal clinical trials in the neoadjuvant and adjuvant settings. However, there exists a discrepancy between different national HTA bodies regarding the acknowledgement of patient relevance of these end-points. In this article, we analysed the perspectives of patients on different aspects of end-points used in clinical trials in early cancer. Gathered evidence strongly suggests that complete tumour eradication and reduced risk of recurrence provide important psychological benefits thus signifying that pCR and time-to-event end-points are directly relevant to patients. Additionally, we reviewed opinions on patient relevance of neoadjuvant and adjuvant therapy end-points adopted by HTA bodies during the recent evaluations. We found that improvements in end-points used in the adjuvant setting were commonly considered as valuable to patients. In contrast, opinions on patient relevance of neoadjuvant therapy end-points varied between the national HTA bodies. Universal acknowledgement of patient relevance of therapeutic end-points for early cancer by HTA bodies is necessary to balance the inequality in uptake of innovative therapies into national healthcare systems.
对新干预措施的临床和经济价值进行卫生技术评估(HTA)是使患者获得创新癌症护理和治疗的一个不可或缺的步骤。总生存期(OS)因其直接的临床意义和与患者的相关性,是HTA中证明治疗效果的首选标准。然而,早期癌症患者的预期寿命往往较长,对总生存期的分析变得不太实用。部分由于这个原因,病理完全缓解(pCR)和无病生存期等事件发生时间终点经常被纳入新辅助和辅助治疗环境中的关键临床试验。然而,不同国家的卫生技术评估机构在承认这些终点与患者的相关性方面存在差异。在本文中,我们分析了患者对早期癌症临床试验中使用的终点不同方面的看法。收集到的证据有力地表明,肿瘤完全根除和复发风险降低能带来重要的心理益处,因此表明病理完全缓解和事件发生时间终点与患者直接相关。此外,我们回顾了卫生技术评估机构在近期评估中对新辅助和辅助治疗终点与患者相关性的意见。我们发现,辅助治疗环境中使用的终点改善通常被认为对患者有价值。相比之下,各国卫生技术评估机构对新辅助治疗终点与患者相关性的意见各不相同。卫生技术评估机构普遍承认早期癌症治疗终点与患者的相关性,对于平衡创新疗法在国家医疗保健系统中的采用不平等至关重要。