Biomedical Engineering Reference
In-Depth Information
10.3.4
Summary of Simulation Results ::::::::::::::::::::::::: 295
10.4
Discussions and Recommendations :::::::::::::::::::::::::::::: 298
Acknowledgments :::::::::::::::::::::::::::::::::::::::::::::::: 302
Appendix: SAS Programs :::::::::::::::::::::::::::::::::::::::: 303
Bibliography :::::::::::::::::::::::::::::::::::::::::::::::::::::: 307
10.1
Introduction
10.1.1
Overview of Progression-Free Survival
In oncology clinical trials, the primary goal of anti-cancer drugs is to prolong
the survival of patients. Hence, overall survival (OS: time from randomiza-
tion to death due to any cause) thus becomes the most desirable endpoint for
evaluating treatment effect because it is the most objective, least biased, and
precise to measure endpoint. In addition, OS is also the best measure of clinical
benefit in any disease indication. However, in some solid tumor settings, OS
may not always be the most appropriate endpoint as it takes a longer time
to follow up patients, requires a larger sample size, and, in addition, treat-
ment effect can be confounded due to post-treatment antineoplastic therapies
received by the patients.
Progression-free survival (PFS), which is defined as the time from ran-
domization to the date of disease progression or death, is used widely as a
primary or secondary endpoint in oncology clinical trials. It is an imaging-
based endpoint and derived based on lesion assessments done by the investi-
gator. Given the diculties of evaluating OS in the presence of cross-over and
second-line treatments, PFS is often seen as a desirable endpoint because it is
not confounded by follow-up treatments. In addition, PFS will require shorter
follow-up of patients and, hence, it is available earlier than OS, potentially
leading to an earlier regulatory approval and making the treatment available
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