Abstract
Progression-free survival (PFS) is an increasingly important end-point in cancer drug
development. However, several concerns exist regarding the use of PFS as a basis to
compare treatments. Unlike survival, the exact time of progression is unknown, so
progression times might be over-estimated (or under-estimated) and, consequently,
bias may be introduced when comparing treatments. In addition, the assessment of progression
is subject to measurement variability which may introduce error or bias. Ideally trials
with PFS as the primary end-point should be randomised and, when feasible, double-blinded.
All patients eligible for study should be evaluable for the primary end-point and
thus, in general, have measurable disease at baseline. Appropriate definitions should
be provided in the protocol and data collected on the case-report forms, if patients
with only non-measurable disease are eligible and/or clinical, or symptomatic progression
are to be considered progression events for analysis. Protocol defined assessments
of disease burden should be obtained at intervals that are symmetrical between arms.
Independent review of imaging may be of value in randomised phase II trials and phase
III trials as an auditing tool to detect possible bias.
Keywords
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Article info
Publication history
Published online: December 22, 2008
Accepted:
October 29,
2008
Received:
October 17,
2008
Identification
Copyright
© 2008 Elsevier Ltd. Published by Elsevier Inc. All rights reserved.
