Outcomes that matter to patients with cancer: living longer and living better.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 39309725.
- Also identified by DOI 10.1016/j.eclinm.2024.102833 and PMC identifier 11415949.
- Licence recorded as CC BY-NC-ND.
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Abstract
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 is not 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 advocates in meaningful ways in clinical trial design and reporting would be a step in this direction.