Journal article
Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative.
- Bogaerts J European Organisation for Research and Treatment of Cancer, Belgium.
- Sydes MR Medical Research Council Clinical Trial Unit at University College London, United Kingdom.
- Keat N Cancer Research UK, United Kingdom.
- McConnell A Cancer Research UK, United Kingdom.
- Benson A Robert H. Lurie Comprehensive Cancer Center of Northwestern University, United States.
- Ho A Memorial Sloan Kettering Cancer Center, United States.
- Roth A Geneva University Hospital, Medical Oncology, Switzerland.
- Fortpied C European Organisation for Research and Treatment of Cancer, Belgium.
- Eng C The University of Texas M.D. Anderson Cancer Center, United States.
- Peckitt C The Royal Marsden, United Kingdom.
- Coens C European Organisation for Research and Treatment of Cancer, Belgium.
- Pettaway C The University of Texas M.D. Anderson Cancer Center, United States.
- Arnold D Hubertus Wald Tumorzentrum - University Cancer Centre Hamburg, Germany.
- Hall E The Institute of Cancer Research, London, United Kingdom.
- Marshall E The University of Liverpool, United Kingdom.
- Sclafani F The Royal Marsden, United Kingdom.
- Hatcher H The University of Cambridge Department of Oncology and NIHR Cambridge Biomedical Research Centre, United Kingdom.
- Earl H The University of Cambridge Department of Oncology and NIHR Cambridge Biomedical Research Centre, United Kingdom.
- Ray-Coquard I Léon Bérard Cancer Center, France.
- Paul J Cancer Research UK Clinical Trials Unit, Institute of Cancer Sciences, University of Glasgow, United Kingdom.
- Blay JY Léon Bérard Cancer Center, France.
- Whelan J NIHR University College London Hospitals Biomedical Research Centre, United Kingdom.
- Panageas K Memorial Sloan Kettering Cancer Center, United States.
- Wheatley K The University of Birmingham, United Kingdom.
- Harrington K The Institute of Cancer Research, London, United Kingdom.
- Licitra L Istituto Nazionale dei Tumori Milan, Italy.
- Billingham L Cancer Research UK Clinical Trials Unit and MRC Midland Hub for Trials Methodology Research, University of Birmingham, United Kingdom.
- Hensley M Memorial Sloan Kettering Cancer Center, United States.
- McCabe M The University of Manchester, United Kingdom.
- Patel PM The University of Nottingham, United Kingdom.
- Carvajal R Memorial Sloan Kettering Cancer Center, United States.
- Wilson R Queen's University Belfast, United Kingdom.
- Glynne-Jones R Mount Vernon Cancer Centre, United Kingdom.
- McWilliams R Mayo Clinic, United States.
- Leyvraz S Centre hospitalier universitaire vaudois, Switzerland.
- Rao S The Royal Marsden, United Kingdom.
- Nicholson S Locum Consultant in Medical Oncology, United Kingdom.
- Filiaci V Gynecologic Oncology Group/NRG Oncology, United States.
- Negrouk A European Organisation for Research and Treatment of Cancer, Belgium.
- Lacombe D European Organisation for Research and Treatment of Cancer, Belgium.
- Dupont E Center for Global Health, US National Cancer Institute, United States.
- Pauporté I Institut National Du Cancer, France.
- Welch JJ Center for Global Health, US National Cancer Institute, United States.
- Law K Cancer Research UK, United Kingdom.
- Trimble T Center for Global Health, US National Cancer Institute, United States.
- Seymour M National Institute for Health Research Clinical Research Network/Cancer, United Kingdom.
- 2014-12-28
Published in:
- European journal of cancer (Oxford, England : 1990). - 2015
Bayesian
Clinical trials
Frequentist
Methodology
Multi-arm
Randomised controlled trials
Rare cancers
Humans
International Cooperation
Neoplasms
Public-Private Sector Partnerships
Randomized Controlled Trials as Topic
Rare Diseases
Research Design
English
BACKGROUND
The past three decades have seen rapid improvements in the diagnosis and treatment of most cancers and the most important contributor has been research. Progress in rare cancers has been slower, not least because of the challenges of undertaking research.
SETTINGS
The International Rare Cancers Initiative (IRCI) is a partnership which aims to stimulate and facilitate the development of international clinical trials for patients with rare cancers. It is focused on interventional--usually randomized--clinical trials with the clear goal of improving outcomes for patients. The key challenges are organisational and methodological. A multi-disciplinary workshop to review the methods used in ICRI portfolio trials was held in Amsterdam in September 2013. Other as-yet unrealised methods were also discussed.
RESULTS
The IRCI trials are each presented to exemplify possible approaches to designing credible trials in rare cancers. Researchers may consider these for use in future trials and understand the choices made for each design.
INTERPRETATION
Trials can be designed using a wide array of possibilities. There is no 'one size fits all' solution. In order to make progress in the rare diseases, decisions to change practice will have to be based on less direct evidence from clinical trials than in more common diseases.
The past three decades have seen rapid improvements in the diagnosis and treatment of most cancers and the most important contributor has been research. Progress in rare cancers has been slower, not least because of the challenges of undertaking research.
SETTINGS
The International Rare Cancers Initiative (IRCI) is a partnership which aims to stimulate and facilitate the development of international clinical trials for patients with rare cancers. It is focused on interventional--usually randomized--clinical trials with the clear goal of improving outcomes for patients. The key challenges are organisational and methodological. A multi-disciplinary workshop to review the methods used in ICRI portfolio trials was held in Amsterdam in September 2013. Other as-yet unrealised methods were also discussed.
RESULTS
The IRCI trials are each presented to exemplify possible approaches to designing credible trials in rare cancers. Researchers may consider these for use in future trials and understand the choices made for each design.
INTERPRETATION
Trials can be designed using a wide array of possibilities. There is no 'one size fits all' solution. In order to make progress in the rare diseases, decisions to change practice will have to be based on less direct evidence from clinical trials than in more common diseases.
- Language
-
- English
- Open access status
- hybrid
- Identifiers
-
- DOI 10.1016/j.ejca.2014.10.027
- PMID 25542058
- Persistent URL
- https://folia.unifr.ch/global/documents/76571
Statistics
Document views: 23
File downloads: