Overcoming the Challenges of Using Overall Survival as an Endpoint in Real-World Research

A July supplement article in Value in Health suggest that alternate endpoints should be considered in oncology clinical trials to better inform pricing and reimbursement decisions because of changes in the outcomes and approaches to treatment associated with many cancer diagnoses over recent years (Fameli A., et al., July 2023, Looking beyond survival data in oncology reimbursement decisions, Vol 9, No. 5, S1). I am sure many would agree that Dr. Fameli and colleagues' argument also extends to conducting real-world studies to inform product effectiveness.

As is true in clinical trials, evaluating the performance of oncology products in a real-world setting solely based on overall survival (OS) may not always be the most suitable approach. Several issues arise with OS as a measure of effectiveness. First, it often requires a lengthy follow-up period, particularly as survival rates have improved in many cancer types, unless the study includes patients with advanced disease, making retrospective analyses challenging. Second, establishing cause-and-effect relationships between a single product and OS is problematic since most patients receive subsequent therapies after initial treatments fail. Consequently, attributing survival outcomes solely to a specific product becomes complicated.

It is important to consider alternative endpoints when addressing these challenges to gain a comprehensive understanding of the effectiveness of treatments in real-world populations. By incorporating other clinical measures, such as progression-free survival (PFS) and objective response rate (ORR), real-world data can offer valuable and timely insights into treatment efficacy. PFS provides information on the duration of disease control, while ORR offers an immediate assessment of treatment activity based on tumor shrinkage or disappearance.

Additionally, to gain a more holistic understanding of the impact of treatments, real-world studies should include assessments of patients' quality of life (QOL). Evaluating QOL enables researchers to consider survival outcomes and patients' physical, emotional, and social well-being. By combining clinical endpoints and QOL assessments, real-world research can offer meaningful insights that benefit all stakeholders involved in oncology product evaluation.

Our team welcomes the challenge of identifying solutions to your real-world evidence-generation challenges. Contact [email protected] if you would like to arrange a time to talk.

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