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Bogaerts J, Sydes MR, Keat N, et al. Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative. Eur J Cancer. 2014;51(3):271-81doi: 10.1016/j.ejca.2014.10.027.
Bogaerts, J., Sydes, M. R., Keat, N., McConnell, A., Benson, A., Ho, A., Roth, A., Fortpied, C., Eng, C., Peckitt, C., Coens, C., Pettaway, C., Arnold, D., Hall, E., Marshall, E., Sclafani, F., Hatcher, H., Earl, H., Ray-Coquard, I., Paul, J., Blay, J. Y., Whelan, J., Panageas, K., Wheatley, K., Harrington, K., Licitra, L., Billingham, L., Hensley, M., McCabe, M., Patel, P. M., Carvajal, R., Wilson, R., Glynne-Jones, R., McWilliams, R., Leyvraz, S., Rao, S., Nicholson, S., Filiaci, V., Negrouk, A., Lacombe, D., Dupont, E., Pauporté, I., Welch, J. J., Law, K., Trimble, T., & Seymour, M. (2015). Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative. European journal of cancer (Oxford, England : 1990), 51(3), 271-81. https://doi.org/10.1016/j.ejca.2014.10.027
Bogaerts, Jan, et al. "Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative." European journal of cancer (Oxford, England : 1990) vol. 51,3 (2015): 271-81. doi: https://doi.org/10.1016/j.ejca.2014.10.027
Bogaerts J, Sydes MR, Keat N, McConnell A, Benson A, Ho A, Roth A, Fortpied C, Eng C, Peckitt C, Coens C, Pettaway C, Arnold D, Hall E, Marshall E, Sclafani F, Hatcher H, Earl H, Ray-Coquard I, Paul J, Blay JY, Whelan J, Panageas K, Wheatley K, Harrington K, Licitra L, Billingham L, Hensley M, McCabe M, Patel PM, Carvajal R, Wilson R, Glynne-Jones R, McWilliams R, Leyvraz S, Rao S, Nicholson S, Filiaci V, Negrouk A, Lacombe D, Dupont E, Pauporté I, Welch JJ, Law K, Trimble T, Seymour M. Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative. Eur J Cancer. 2015 Feb;51(3):271-81. doi: 10.1016/j.ejca.2014.10.027. Epub 2014 Dec 24. PMID: 25542058; PMCID: PMC4639696.
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Eur J Cancer. 2015 Feb;51(3):271-81. doi: 10.1016/j.ejca.2014.10.027. Epub 2014 Dec 24.

Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative.

European journal of cancer (Oxford, England : 1990)

Jan Bogaerts, Matthew R Sydes, Nicola Keat, Andrea McConnell, Al Benson, Alan Ho, Arnaud Roth, Catherine Fortpied, Cathy Eng, Clare Peckitt, Corneel Coens, Curtis Pettaway, Dirk Arnold, Emma Hall, Ernie Marshall, Francesco Sclafani, Helen Hatcher, Helena Earl, Isabelle Ray-Coquard, James Paul, Jean-Yves Blay, Jeremy Whelan, Kathy Panageas, Keith Wheatley, Kevin Harrington, Lisa Licitra, Lucinda Billingham, Martee Hensley, Martin McCabe, Poulam M Patel, Richard Carvajal, Richard Wilson, Rob Glynne-Jones, Rob McWilliams, Serge Leyvraz, Sheela Rao, Steve Nicholson, Virginia Filiaci, Anastassia Negrouk, Denis Lacombe, Elisabeth Dupont, Iris Pauporté, John J Welch, Kate Law, Ted Trimble, Matthew Seymour

Affiliations

  1. European Organisation for Research and Treatment of Cancer, Belgium.
  2. Medical Research Council Clinical Trial Unit at University College London, United Kingdom.
  3. Cancer Research UK, United Kingdom.
  4. Robert H. Lurie Comprehensive Cancer Center of Northwestern University, United States.
  5. Memorial Sloan Kettering Cancer Center, United States.
  6. Geneva University Hospital, Medical Oncology, Switzerland.
  7. The University of Texas M.D. Anderson Cancer Center, United States.
  8. The Royal Marsden, United Kingdom.
  9. Hubertus Wald Tumorzentrum - University Cancer Centre Hamburg, Germany.
  10. The Institute of Cancer Research, London, United Kingdom.
  11. The University of Liverpool, United Kingdom.
  12. The University of Cambridge Department of Oncology and NIHR Cambridge Biomedical Research Centre, United Kingdom.
  13. Léon Bérard Cancer Center, France.
  14. Cancer Research UK Clinical Trials Unit, Institute of Cancer Sciences, University of Glasgow, United Kingdom.
  15. NIHR University College London Hospitals Biomedical Research Centre, United Kingdom.
  16. The University of Birmingham, United Kingdom.
  17. Istituto Nazionale dei Tumori Milan, Italy.
  18. Cancer Research UK Clinical Trials Unit and MRC Midland Hub for Trials Methodology Research, University of Birmingham, United Kingdom.
  19. The University of Manchester, United Kingdom.
  20. The University of Nottingham, United Kingdom.
  21. Queen's University Belfast, United Kingdom.
  22. Mount Vernon Cancer Centre, United Kingdom.
  23. Mayo Clinic, United States.
  24. Centre hospitalier universitaire vaudois, Switzerland.
  25. Locum Consultant in Medical Oncology, United Kingdom.
  26. Gynecologic Oncology Group/NRG Oncology, United States.
  27. Center for Global Health, US National Cancer Institute, United States.
  28. Institut National Du Cancer, France.
  29. National Institute for Health Research Clinical Research Network/Cancer, United Kingdom.

PMID: 25542058 PMCID: PMC4639696 DOI: 10.1016/j.ejca.2014.10.027

Abstract

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.

Copyright © 2014 The Authors. Published by Elsevier Ltd.. All rights reserved.

Keywords: Bayesian; Clinical trials; Frequentist; Methodology; Multi-arm; Randomised controlled trials; Rare cancers

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