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The value of progression-free survival to patients with advanced-stage cancer

Nature Reviews Clinical Oncology volume 9, pages 41–47 (2012)Cite this article

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Abstract

Progression-free survival (PFS) is frequently used as a primary end point in oncology clinical trials. Employing PFS instead of overall survival as the primary outcome has the advantage that trial completion can be quicker with fewer patients required, and it is cheaper. PFS is sensitive to cytostatic as well as cytotoxic mechanisms of therapeutic intervention and directly measures the effect of the investigational treatment. Despite these practical advantages, it is unclear whether or not extending PFS provides discernable clinical benefit. New treatments that increase PFS may not be of sufficient value to patients with advanced-stage cancer unless accompanied by tangible quantity or quality of life advantages. Any symptom relief that patients gain from treatment resulting in tumor shrinkage or stabilization must be balanced against the toxic effects that drug therapy itself creates. Consequently, improved assessment of new treatments using patient-reported outcomes alongside PFS is crucial to enable communication between clinicians and patients and optimal decision-making about therapeutic options.

Key Points

  • Many clinical trials of novel cancer treatment report outcomes in terms of progression-free survival (PFS) rather than overall survival

  • Few trials have good quality-of-life (QoL) data collected via patient-reported outcome measures

  • There are insufficient studies directly measuring the value and putative benefits for patients of tumor stabilization or PFS

  • QoL data may be vital to assist with patient decision making as outcomes that clinicians consider important might not provide benefits to patients who may suffer the side effects of treatment

  • Discussing the harms and benefits of treatment with sick and anxious patients is challenging

  • More research on patients' experiences captured with standardized measures is needed and such data should be integrated and reported alongside more traditional trial outcomes to enable better decision making

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Authors and Affiliations

  1. Sussex Health Outcomes Research & Education in Cancer (SHORE-C), Brighton and Sussex Medical School, University of Sussex, Falmer, BN1 9RX, East Sussex, UK

    Lesley J. Fallowfield & Anne Fleissig

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  1. Lesley J. Fallowfield
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Both authors contributed equally to researching data for the article, writing, editing and reviewing the manuscript before and during the peer review and editing process.

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Correspondence to Lesley J. Fallowfield.

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The authors declare no competing financial interests.

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Fallowfield, L., Fleissig, A. The value of progression-free survival to patients with advanced-stage cancer. Nat Rev Clin Oncol 9, 41–47 (2012). https://doi.org/10.1038/nrclinonc.2011.156

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