Background/Objectives: In early-stage oncology clinical trials, the use of endpoints beyond overall survival (OS), including recurrence-free survival (RFS) or event-free survival (EFS), is becoming more common. To understand whether these outcomes are important to patients, this study explored the perceived value of non-OS endpoints among Canadians treated for early-stage cancer or with curative intent. Methods: Canadians treated for early-stage breast, lung, or gastrointestinal cancer participated in semi-structured interviews. Participants provided perspectives on OS, RFS, disease-free survival (DFS), EFS, and pathological complete response (pCR) endpoints. Reflexive thematic analysis was used to explore patterns in responses and alignment of trial endpoints with patient treatment goals, priorities and preferences. Results: The mean age of the 33 participants was 54.8 years, and 21 were female; 28 reported prior surgery, and 21 were also treated with chemotherapy (11 specified as neo-adjuvant; 9 specified adjuvant). All participants valued OS, and most viewed non-OS endpoints as reflective of their treatment priorities, including maintaining health-related quality of life and getting back to ‘normal’. They also valued timely and equitable treatment access and equated having access to new treatments with better options. While participants considered efficacy data from clinical trials provided by non-OS endpoints sufficient to want access to new treatments, the relative importance of being disease- or recurrence-free versus maximizing length of life differed according to recurrence status, prognosis, cancer type and life stage. Conclusions: These findings support the relevance and importance of non-OS endpoints to Canadians with early-stage cancer and highlight participants’ desire for rapid approval of treatments with demonstrated improvements in non-OS endpoints.
背景/目的：在早期肿瘤学临床试验中，使用总生存期（OS）以外的终点指标，包括无复发生存期（RFS）或无事件生存期（EFS），正变得越来越普遍。为了解这些结局对患者是否重要，本研究探讨了接受早期癌症治疗或以治愈为目的的加拿大人对非OS终点指标的认知价值。方法：接受早期乳腺癌、肺癌或胃肠道癌治疗的加拿大人参与了半结构化访谈。参与者就OS、RFS、无病生存期（DFS）、EFS和病理完全缓解（pCR）等终点指标提供了观点。采用反思性主题分析法，探讨了回答中的模式以及试验终点与患者治疗目标、优先事项和偏好的契合度。结果：33名参与者的平均年龄为54.8岁，其中21名为女性；28名参与者报告曾接受手术，21名还接受了化疗（11名明确为新辅助治疗；9名明确为辅助治疗）。所有参与者都重视OS，大多数人认为非OS终点反映了他们的治疗优先事项，包括维持与健康相关的生活质量以及回归“正常”生活。他们还重视及时和公平的治疗机会，并将获得新治疗等同于拥有更好的选择。尽管参与者认为非OS终点提供的临床试验疗效数据足以让他们希望获得新治疗，但无病或未复发与最大化生存期之间的相对重要性因复发状态、预后、癌症类型和生活阶段而异。结论：这些发现支持非OS终点对加拿大早期癌症患者的相关性和重要性，并突显了参与者对在非OS终点上显示出改善的治疗方法能够快速获批的渴望。
肿瘤(癌症)患者之家