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abstract
breast, colorectal, lung, ovarian, and pancreatic cancer72,600 SEER-Medicare patients similar to RCT participants
Objective It is unclear how well different
outcome measures in randomized controlled trials (RCTs) perform in
predicting real-world cancer survival. We assess the ability of RCT
overall survival (OS) and surrogate endpoints – progression-free
survival (PFS) and time to progression (TTP) – to predict real-world OS
across five cancers.
Conclusions
Among the five tumor types investigated, trial OS and surrogates were
each independently valuable in predicting real-world OS outcomes for
patients similar to trial participants. In broader real-world
populations, however, trial OS added little incremental value over
surrogates alone.
Methods We
identified 20 treatments and 31 indications for breast, colorectal,
lung, ovarian, and pancreatic cancer that had a phase III RCT reporting
median OS and median PFS or TTP. Median real-world OS was determined
using a Kaplan–Meier estimator applied to patients in the Surveillance
and Epidemiology End Results (SEER)-Medicare database (1991–2010).
Performance of RCT OS and PFS/TTP in predicting real-world OS was
measured using t-tests, median absolute prediction error, and R2 from linear regressions.
Results
Among 72,600 SEER-Medicare patients similar to RCT participants, median
survival was 5.9 months for trial surrogates, 14.1 months for trial OS,
and 13.4 months for real-world OS. For this sample, regression models
using clinical trial OS and trial surrogates as independent variables
predicted real-world OS significantly better than models using
surrogates alone (P = 0.026). Among all real-world patients using sample treatments (N = 309,182), however, adding trial OS did not improve predictive power over predictions based on surrogates alone (P = 0.194). Results were qualitatively similar using median absolute prediction error and R2 metrics.
Conclusions
Among the five tumor types investigated, trial OS and surrogates were
each independently valuable in predicting real-world OS outcomes for
patients similar to trial participants. In broader real-world
populations, however, trial OS added little incremental value over
surrogates alone.
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