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Basic & Clinical Pharmacology & Toxicology
Article . 2018 . Peer-reviewed
License: Wiley Online Library User Agreement
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Progression‐free survival in oncology: Caveat emptor!

caveat emptor!
Authors: Per Damkier; Per Damkier; Maija Bruun Haastrup; Maija Bruun Haastrup; Troels K Bergmann; Troels K Bergmann; Mette Marie Hougaard Christensen; +3 Authors

Progression‐free survival in oncology: Caveat emptor!

Abstract

AbstractOverall survival (OS) is the undisputed gold standard efficacy end‐point in cancer drug trials. It is with growing concern that we observe how progression‐free survival (PFS) gains ground as surrogate end‐point in its place. PFS has appeal because it is resource‐efficient, but it has severe shortcomings. Our concern is that uncritical use of PFS will harm the evidence‐based evaluation of cancer drugs when considering them for standard use in publicly financed health care systems. PFS is only valid as a surrogate end‐point for OS if it correlates strongly with OS and if the cancer drug being investigated has the same effect on PFS and OS such that effects on one predict effects on the other. The latter might be less obvious than the former but is no less critical. Research indicates that in a majority of cases, correlation between surrogate end‐points and OS is of medium strength or lower. PFS is therefore unreliable as a surrogate for OS. We do not find it justified to use PFS as surrogate for OS without first having assessed its validity. Stakeholders who take part in evaluating cancer drugs considered for standard use in a health care system must be particularly vigilant about this issue to minimize the risk of introducing cancer drugs that have an unacceptable cost‐risk‐benefit profile.

Country
Denmark
Keywords

Clinical Trials as Topic, overall survival, Cost-Benefit Analysis, Antineoplastic Agents, Survival Analysis, Progression-Free Survival, quality of life, Neoplasms, oncology, Quality of Life, Humans, surrogate end-point, cancer drugs, progression-free survival, Biomarkers

  • BIP!
    Impact byBIP!
    citations
    This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
    6
    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Top 10%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
    Top 10%
    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
    Average
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citations
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
6
Top 10%
Top 10%
Average
Green
bronze