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Journal of Cancer Research and Clinical Oncology

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01-11-2016 | Original Article – Clinical Oncology

Trends in endpoint selection in clinical trials of advanced breast cancer

Authors: Seung Yeon Song, Heenam Seo, Gyungjin Kim, Ah Rong Kim, Eun Young Kim

Published in: Journal of Cancer Research and Clinical Oncology | Issue 11/2016

Abstract

Purpose

The selection of appropriate endpoints is crucial for the evaluation of clinical benefits and approval of novel anticancer agents. To our knowledge, this is the first study to evaluate endpoint selection and the shift in trends in phase II and phase III trials of advanced breast cancer treatment.

Methods

All phase II and phase III trials of advanced breast cancer registered in the ClinicalTrials.gov registry between October 2000 and September 2012 were included in our study. Two study periods were considered for comparison: October 2000 to September 2007 (cohort A) and October 2007 to September 2012 (cohort B). Information on primary and secondary outcome measures, as well as trial characteristics, was extracted by two independent reviewers.

Results

Of the 398 phase II and 120 phase III trials, the most frequently intended primary endpoint was progression-free survival (phase II: 28.1 %; phase III: 50.0 %). For phase II trials, a shifting trend in primary outcome was observed from cohort A to cohort B: the use of objective response rate, the most frequently intended primary outcome, significantly declined (cohort A: 60.6 %; cohort B: 39.0 %; P < 0.001), while the use of progression-free survival significantly increased (cohort A: 35.9 %; cohort B: 66.1 %; P < 0.001).

Conclusions

Progression-free survival is the most frequently intended primary outcome measure in phase II and phase III trials of advanced breast cancer treatment, with a shifting trend observed from objective response rate to progression-free survival in phase II trials.

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Title: Trends in endpoint selection in clinical trials of advanced breast cancer
Authors: Seung Yeon Song
Heenam Seo
Gyungjin Kim
Ah Rong Kim
Eun Young Kim
Publication date: 01-11-2016
Publisher: Springer Berlin Heidelberg
Published in: Journal of Cancer Research and Clinical Oncology / Issue 11/2016
Print ISSN: 0171-5216
Electronic ISSN: 1432-1335
DOI: https://doi.org/10.1007/s00432-016-2221-5

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