Outcomes that matter to patients with cancer - living longer and living better

Sep 21, 2026

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Advanced cancer trials increasingly rely on surrogate endpoints such as progression-free survival (PFS), even though this metric does not directly measure how patients feel, function, or ultimately survive. Evidence indicates that when patients understand what PFS means, many would decline additional treatment unless overall survival improves. The article argues for designing trials around clinically meaningful questions and reporting patient-centered outcomes, including meaningful engagement of educated patient advocates in trial design and dissemination.

LanguageEnglish

CategoryHealthcare

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Pages4

Reading Time10 min

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Summary

Introduction

Defining progression-free survival, its advantages, and limitations

Viewpoint Outcomes that matter to patients with cancer: living longer and living better Michelle Tregear∗ and Fran Visco National Breast Cancer Coalition, Washington, DC, USA Summary Oncologists and cancer patients generally agree that the primary goals of advanced cancer treatment are to lengthen and/or improve patient survival. Yet over the last two decades, clinical trials of new cancer treatments have moved away from measuring outcomes that matter to patients. Increasingly, new drugs for advanced cancer treatment reach the market by demonstrating improvements in surrogate endpoints such as progression-free survival (PFS), which isnot a measure of how a patient feels, functions, or survives. Research has shown that when patients are fully informed about the meaning of PFS, about half would not choose additional treatment for any magnitude of gain in PFS in the absence of an overall survival improvement. It’s time to get back to designing trials that answer clinically meaningful questions and measure the outcomes that truly matter to patients. Engaging educated patient advocatesin meaningful ways in clinical trial design and reporting would be a step in this direction. Copyright © 2024 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY-NC-ND license ([http://creativecommons.org/licenses/by-nc-nd/4.0/](http://creativecommons.org/licenses/by-nc-nd/4.0/)). Keywords: Oncology clinical trial endpoints; Patient centered outcomes research; Patient preferences; Progressionfree survival eClinicalMedicine 2024;76: 102833 Published Online xxx [https://doi.org/10](https://doi.org/10) . 1016/j.eclinm.2024 . 102833 Introduction Oncologists and cancer patients generally agree that the primary goals of cancer treatment are to lengthen survival and, especially in the advanced disease setting, to improve quality of life.1,2 Yet over the last two decades, clinical trials of new cancer treatments have moved away from measuring outcomes that matter to patients. Until the early 2000s, overall survival was the predominant endpoint in clinical trials of new cancer drugs. Today, however, most cancer drugs and biologics come to market based on clinical trials that use surrogate endpoints such as progression-free survival (PFS) to demonstrate beneﬁt.3,4 Patients and the public must understand what actual beneﬁt if any these surrogate endpoints mean for their lives. To help ensure this, we need public education on this issue, and educated patient advocates who represent a constituency need to play meaningful roles in the design, conduct, and reporting of clinical trials. Deﬁning progression-free survival, its advantages, and limitations Despite what the term implies, PFS is not a measure of clinical beneﬁt: how a patient feels or functions, or how long they survive.5 PFS is one of a number of tumor response metrics adopted to help standardize measures of anticancer activity in clinical research among varying *Corresponding author. 2001 L Street NW Suite 500 PMB\#50111, Washington, DC, 20036 . E-mail [address:](address: mtregear@stopbreastcancer.org)[ mtregear@stopbreastcancer.org](address: mtregear@stopbreastcancer.org) (M. Tregear). investigators and institutions.6,7 Moertel and Hanley were among the ﬁrst to investigate oncologists’ ability to detect tumor progression and shrinkage using simulated tumors (marbles of varying size) placed under simulated skin (mattress covered in rubber foam).8 They reported that erroneous measures of tumor progression and regression were minimized when simulated tumors increased in diameter by at least 25 percent or shrunk by at least 75 percent. Today, PFS is deﬁned according to the Response Evaluation Criteria in Solid Tumors ﬁrst published in 2000 and updated in 2009 . It represents the time from randomization or the initiation of treatment to measurable disease progression (typically by radiographic assessment in solid tumors) or death, with p

FAQs

Why do advanced cancer trials use progression-free survival (PFS) as an endpoint?

Because PFS can be measured more quickly and with smaller sample sizes than overall survival, potentially speeding access to new therapies and reducing development costs. It is also less affected by subsequent treatments after progression.

What is the main limitation of PFS for patients?

PFS is not a direct measure of clinical benefit; it does not reflect how a patient feels or functions, nor how long they survive. Patients may not perceive health deterioration even when tumors progress by the definition used.

How does understanding PFS affect patient treatment decisions?

When patients are fully informed about the meaning of PFS, about half would not choose additional treatment for any magnitude of PFS gain unless there is an overall survival improvement.

Sep 21, 2026

Upload by Riley