title: Outcomes and endpoints in cancer trials: bridging the divide.
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.
authors: University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada.
authors: Michelle K
authors: MK
authors: Wilson
authors: Patient Advocates In Research, Danville, CA, USA.
authors: Deborah
authors: D
authors: Collyar
authors: The Noreen Fraser Foundation, Los Angeles, CA, USA.
authors: Diana T
authors: DT
authors: Chingos
authors: Prince of Wales Clinical School, University of New South Wales, Sydney, Australia.
authors: Michael
authors: M
authors: Friedlander
authors: AstraZeneca, Wilmington DE 19850-5437, USA.
authors: Tony W
authors: TW
authors: Ho
authors: University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada.
authors: Katherine
authors: K
authors: Karakasis
authors: Drug Development Unit and Gynaecology Unit, Royal Marsden Hospital and Institute of Cancer Research, London, UK.
authors: Stan
authors: S
authors: Kaye
authors: MRC Clinical Trials Unit, University College London, London, UK.
authors: Mahesh K B
authors: MK
authors: Parmar
authors: MRC Clinical Trials Unit, University College London, London, UK.
authors: Matthew R
authors: MR
authors: Sydes
authors: University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada.
authors: Ian F
authors: IF
authors: Tannock
authors: University of Toronto Princess Margaret Cancer Centre, Toronto, ON, Canada. Electronic address: amit.oza@uhn.ca.
authors: Amit M
authors: AM
authors: Oza
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D018450:Disease Progression; D018572:Disease-Free Survival; D023381:Endpoint Determination; D006801:Humans; D009369:Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011788:Quality of Life; D012107:Research Design; D013997:Time Factors; D016896:Treatment Outcome