title: Relevance of randomised controlled trials in oncology.
abstract: Well-designed randomised controlled trials (RCTs) can prevent bias in the comparison of treatments and provide a sound basis for changes in clinical practice. However, the design and reporting of many RCTs can render their results of little relevance to clinical practice. In this Personal View, we discuss the limitations of RCT data and suggest some ways to improve the clinical relevance of RCTs in the everyday management of patients with cancer. RCTs should ask questions of clinical rather than commercial interest, avoid non-validated surrogate endpoints in registration trials, and have entry criteria that allow inclusion of all patients who are fit to receive treatment. Furthermore, RCTs should be reported with complete accounting of frequency and management of toxicities, and with strict guidelines to ensure freedom from bias. Premature reporting of results should be avoided. The bar for clinical benefit should be raised for drug registration, which should require publication and review of mature data from RCTs, post-marketing health outcome studies, and value-based pricing.
authors.affiliation: Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre and University of Toronto, Toronto, ON, Canada. Electronic address: ian.tannock@uhn.ca.
authors.forename: Ian F
authors.initials: IF
authors.lastname: Tannock
authors.affiliation: Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre and University of Toronto, Toronto, ON, Canada.
authors.forename: Eitan
authors.initials: E
authors.lastname: Amir
authors.affiliation: Departments of Oncology and Public Health Sciences, Queen's University, Kingston, ON, Canada.
authors.forename: Christopher M
authors.initials: CM
authors.lastname: Booth
authors.affiliation: Departments of Medical Oncology and Haematology, University of Manitoba and CancerCare Manitoba, Winnipeg, MB, Canada.
authors.forename: Saroj
authors.initials: S
authors.lastname: Niraula
authors.affiliation: Translational Research Unit, Albacete University Hospital, Albacete, Spain.
authors.forename: Alberto
authors.initials: A
authors.lastname: Ocana
authors.affiliation: Department of Medical Oncology, Institute of Oncology Ljubljana and University of Ljubljana, Slovenia.
authors.forename: Bostjan
authors.initials: B
authors.lastname: Seruga
authors.affiliation: Department of Medical Oncology, St Claraspital and Faculty of Medicine, University of Basel, Basel, Switzerland.
authors.forename: Arnoud J
authors.initials: AJ
authors.lastname: Templeton
authors.affiliation: Canadian Cancer Trials Group and Department of Oncology, Queen's University, Kingston, ON, Canada; Tecnológico de Monterrey School of Medicine, Monterrey, Nuevo León, Mexico.
authors.forename: Francisco
authors.initials: F
authors.lastname: Vera-Badillo
mesh_terms: D015982:Bias; D006801:Humans; D009369:Neoplasms / Q000523:psychology / Q000628:therapy*; D018579:Patient Selection; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic; D012107:Research Design