# “Are You Just Looking to ‘Survive’?”: A Qualitative Study of Importance of Oncology Endpoints Beyond Overall Survival in Early-Stage Cancer

## TL;DR

This study shows that cancer patients value outcomes like being free of cancer recurrence and returning to normal life, not just living longer.

## Contribution

It demonstrates patient perspectives on non-survival endpoints in oncology trials and supports their use for faster treatment access.

## Key findings

- Patients value non-survival endpoints like recurrence-free survival as meaningful outcomes.
- Participants emphasized timely and equitable access to new treatments based on non-survival endpoints.
- Importance of endpoints varied by cancer type, prognosis, and life stage.

## Abstract

This study explored how people treated for early-stage or curable cancer in Canada view outcomes measured in clinical trials. Traditionally, trials focus on overall survival (how long people live), but newer studies often use other outcomes, such as being free of cancer returning (recurrence-free survival), staying free from signs that the cancer is worsening (event-free survival), or showing no cancer after treatment (pathological complete response). Thirty-three people treated for breast, lung, or gastrointestinal cancers were interviewed. Participants said living longer was important, but they also valued outcomes that reflect living without cancer and being able to return to normal life. Many emphasized the importance of timely and fair access to new treatments, even when results are based on these non-survival outcomes. Overall, the study shows that patients see these measures as meaningful and support using them to speed up access to promising treatments.

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.