surrogate endpoint
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listen (SER-uh-gut END-poynt)
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In clinical trials, an indicator or sign used in
place of another to tell if a treatment works. Surrogate endpoints
include a shrinking tumor or lower biomarker levels. They may be used
instead of stronger indicators, such as longer survival or improved
quality of life, because the results of the trial can be measured
sooner. The use of surrogate endpoints in clinical trials may allow
earlier approval of new drugs to treat serious or life-threatening
diseases, such as cancer. Surrogate endpoints are not always true
indicators or signs of how well a treatment works.
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abstract - Mayo Clinic Proceedings
Objective
To determine the strength of the surrogate-survival correlation for cancer drug approvals based on a surrogate.
Participants and Methods
We
performed a retrospective study of the US Food and Drug Administration
(FDA) database, with focused searches of MEDLINE and Google Scholar.
Among cancer drugs approved based on a surrogate end point, we examined
previous publications assessing the strength of the surrogate-survival
correlation. Specifically, we identified the percentage of surrogate
approvals lacking any formal analysis of the strength of the
surrogate-survival correlation, and when conducted, the strength of such
correlations.
Results
Between
January 1, 2009, and December 31, 2014, the FDA approved marketing
applications for 55 indications based on a surrogate, of which 25 were
accelerated approvals and 30 were traditional approvals.
We could not
find any formal analyses of the strength of the surrogate-survival
correlation in 14 out of 25 accelerated approvals (56%) and
11 out of 30
traditional approvals (37%). For accelerated approvals, just 4
approvals (16%) were made where a level 1 analysis (the most robust way
to validate a surrogate) had been performed, with all 4 studies
reporting low correlation (r≤0.7). For traditional approvals, a level 1
analysis had been performed for 15 approvals (50%): 8 (53%) reported low
correlation (r≤0.7), 4 (27%) medium correlation (r>0.7 to
r<0.85), and 3 (20%) high correlation (r≥0.85) with survival.
Conclusions
The
use of surrogate end points for drug approval often lacks formal
empirical verification of the strength of the surrogate-survival
association.
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