title: Accepting risk in the acceleration of drug development for rare cancers.
abstract: Rare cancers collectively contribute a disproportionate fraction of the total burden of cancer. The oncology community is increasingly facing small numbers of patients with each cancer subtype, requiring cooperation and collaboration to complete multicentre trials that advance knowledge and patient care. At the same time, new insights into the biology of rare cancers have led to an explosion in knowledge and development of targeted agents. These insights and techniques are set to revolutionise the care of patients with cancer. However, drug development strategies and the availability of new agents for rare cancers are at risk of stalling owing to the ever-increasing complexity and costs of clinical trials. Finding solutions to these problems is imperative to the future of cancer care. We propose that a greater degree of risk sharing is needed than is currently accepted to enable the use of new methods with confidence, and to keep pace with scientific advancement.
authors.affiliation: School of Medicine, Deakin University, Geelong, VIC, Australia; Andrew Love Cancer Centre, Barwon Health, Geelong, VIC, Australia. Electronic address: dashle@barwonhealth.org.au.
authors.forename: David
authors.initials: D
authors.lastname: Ashley
authors.affiliation: Kinghorn Cancer Centre, Darlinghurst, NSW, Australia; Cancer Division, Garvan Institute of Medical Research, Darlinghurst, NSW, Australia.
authors.forename: David
authors.initials: D
authors.lastname: Thomas
authors.affiliation: Children's Hospital Colorado, Aurora, CO, USA; Center for Cancer and Blood Disorders, Aurora, CO, USA.
authors.forename: Lia
authors.initials: L
authors.lastname: Gore
authors.affiliation: Deakin Health Economics, Deakin University, Burwood, VIC, Australia.
authors.forename: Rob
authors.initials: R
authors.lastname: Carter
authors.affiliation: Cancer Research Program, School of Public Health and Preventive Medicine, Faculty of Medicine, Nursing and Health Sciences, Monash University, Melbourne, VIC, Australia.
authors.forename: John R
authors.initials: JR
authors.lastname: Zalcberg
authors.affiliation: School of Medicine, Deakin University, Geelong, VIC, Australia.
authors.forename: Renée
authors.initials: R
authors.lastname: Otmar
authors.affiliation: Oxford Uehiro Centre for Practical Ethics, University of Oxford, Oxford, UK.
authors.forename: Julian
authors.initials: J
authors.lastname: Savulescu
mesh_terms: D055808:Drug Discovery; D006801:Humans; D058990:Molecular Targeted Therapy; D009369:Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D035583:Rare Diseases / Q000188:drug therapy* / Q000473:pathology; D012106:Research