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Lancet Oncol. 2015 Jan;16(1):e43-52. doi: 10.1016/S1470-2045(14)70380-8. Epub 2014 Dec 29.

Outcomes and endpoints in cancer trials: bridging the divide.

Author information

1
University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada.
2
Patient Advocates In Research, Danville, CA, USA.
3
The Noreen Fraser Foundation, Los Angeles, CA, USA.
4
Prince of Wales Clinical School, University of New South Wales, Sydney, Australia.
5
AstraZeneca, Wilmington DE 19850-5437, USA.
6
Drug Development Unit and Gynaecology Unit, Royal Marsden Hospital and Institute of Cancer Research, London, UK.
7
MRC Clinical Trials Unit, University College London, London, UK.
8
University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada. Electronic address: amit.oza@uhn.ca.

Abstract

Cancer is not one disease. Outcomes and endpoints in trials should incorporate the therapeutic modality and cancer type because these factors affect clinician and patient expectations. In this Review, we discuss how to: define the importance of endpoints; make endpoints understandable to patients; improve the use of patient-reported outcomes; advance endpoints to parallel changes in trial design and therapeutic interventions; and integrate these improvements into trials and practice. Endpoints need to reflect benefit to patients, and show that changes in tumour size either in absolute terms (response and progression) or relative to control (progression) are clinically relevant. Improvements in trial design should be accompanied by improvements in available endpoints. Stakeholders need to come together to determine the best approach for research that ensures accountability and optimises the use of available resources.

PMID:
25638556
DOI:
10.1016/S1470-2045(14)70380-8
[Indexed for MEDLINE]

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