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

Jan Bogaerts*, Matthew R. Sydes, Nicola Keat, Andrea McConnell, Al B Benson III, 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 PaulJean 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

*Corresponding author for this work

Research output: Contribution to journalReview article

59 Citations (Scopus)

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 randomised - 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.

Original languageEnglish (US)
Pages (from-to)271-281
Number of pages11
JournalEuropean Journal of Cancer
Volume51
Issue number3
DOIs
StatePublished - Jan 1 2015

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Rare Diseases
Clinical Trials
Neoplasms
Research
Randomized Controlled Trials
Research Personnel
Education

Keywords

  • Bayesian
  • Clinical trials
  • Frequentist
  • Methodology
  • Multi-arm
  • Randomised controlled trials
  • Rare cancers

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Bogaerts, Jan ; Sydes, Matthew R. ; Keat, Nicola ; McConnell, Andrea ; Benson III, Al B ; Ho, Alan ; Roth, Arnaud ; Fortpied, Catherine ; Eng, Cathy ; Peckitt, Clare ; Coens, Corneel ; Pettaway, Curtis ; Arnold, Dirk ; Hall, Emma ; Marshall, Ernie ; Sclafani, Francesco ; Hatcher, Helen ; Earl, Helena ; Ray-Coquard, Isabelle ; Paul, James ; Blay, Jean Yves ; Whelan, Jeremy ; Panageas, Kathy ; Wheatley, Keith ; Harrington, Kevin ; Licitra, Lisa ; Billingham, Lucinda ; Hensley, Martee ; McCabe, Martin ; Patel, Poulam M. ; Carvajal, Richard ; Wilson, Richard ; Glynne-Jones, Rob ; McWilliams, Rob ; Leyvraz, Serge ; Rao, Sheela ; Nicholson, Steve ; Filiaci, Virginia ; Negrouk, Anastassia ; Lacombe, Denis ; Dupont, Elisabeth ; Pauporté, Iris ; Welch, John J. ; Law, Kate ; Trimble, Ted ; Seymour, Matthew. / Clinical trial designs for rare diseases : Studies developed and discussed by the International Rare Cancers Initiative. In: European Journal of Cancer. 2015 ; Vol. 51, No. 3. pp. 271-281.
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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 randomised - 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.",
keywords = "Bayesian, Clinical trials, Frequentist, Methodology, Multi-arm, Randomised controlled trials, Rare cancers",
author = "Jan Bogaerts and Sydes, {Matthew R.} and Nicola Keat and Andrea McConnell and {Benson III}, {Al B} and Alan Ho and Arnaud Roth and Catherine Fortpied and Cathy Eng and Clare Peckitt and Corneel Coens and Curtis Pettaway and Dirk Arnold and Emma Hall and Ernie Marshall and Francesco Sclafani and Helen Hatcher and Helena Earl and Isabelle Ray-Coquard and James Paul and Blay, {Jean Yves} and Jeremy Whelan and Kathy Panageas and Keith Wheatley and Kevin Harrington and Lisa Licitra and Lucinda Billingham and Martee Hensley and Martin McCabe and Patel, {Poulam M.} and Richard Carvajal and Richard Wilson and Rob Glynne-Jones and Rob McWilliams and Serge Leyvraz and Sheela Rao and Steve Nicholson and Virginia Filiaci and Anastassia Negrouk and Denis Lacombe and Elisabeth Dupont and Iris Pauport{\'e} and Welch, {John J.} and Kate Law and Ted Trimble and Matthew Seymour",
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Bogaerts, J, Sydes, MR, Keat, N, McConnell, A, Benson III, AB, 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 2015, 'Clinical trial designs for rare diseases: Studies developed and discussed by the International Rare Cancers Initiative', European Journal of Cancer, vol. 51, no. 3, pp. 271-281. https://doi.org/10.1016/j.ejca.2014.10.027

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

In: European Journal of Cancer, Vol. 51, No. 3, 01.01.2015, p. 271-281.

Research output: Contribution to journalReview article

TY - JOUR

T1 - Clinical trial designs for rare diseases

T2 - Studies developed and discussed by the International Rare Cancers Initiative

AU - Bogaerts, Jan

AU - Sydes, Matthew R.

AU - Keat, Nicola

AU - McConnell, Andrea

AU - Benson III, Al B

AU - Ho, Alan

AU - Roth, Arnaud

AU - Fortpied, Catherine

AU - Eng, Cathy

AU - Peckitt, Clare

AU - Coens, Corneel

AU - Pettaway, Curtis

AU - Arnold, Dirk

AU - Hall, Emma

AU - Marshall, Ernie

AU - Sclafani, Francesco

AU - Hatcher, Helen

AU - Earl, Helena

AU - Ray-Coquard, Isabelle

AU - Paul, James

AU - Blay, Jean Yves

AU - Whelan, Jeremy

AU - Panageas, Kathy

AU - Wheatley, Keith

AU - Harrington, Kevin

AU - Licitra, Lisa

AU - Billingham, Lucinda

AU - Hensley, Martee

AU - McCabe, Martin

AU - Patel, Poulam M.

AU - Carvajal, Richard

AU - Wilson, Richard

AU - Glynne-Jones, Rob

AU - McWilliams, Rob

AU - Leyvraz, Serge

AU - Rao, Sheela

AU - Nicholson, Steve

AU - Filiaci, Virginia

AU - Negrouk, Anastassia

AU - Lacombe, Denis

AU - Dupont, Elisabeth

AU - Pauporté, Iris

AU - Welch, John J.

AU - Law, Kate

AU - Trimble, Ted

AU - Seymour, Matthew

PY - 2015/1/1

Y1 - 2015/1/1

N2 - 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 randomised - 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.

AB - 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 randomised - 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.

KW - Bayesian

KW - Clinical trials

KW - Frequentist

KW - Methodology

KW - Multi-arm

KW - Randomised controlled trials

KW - Rare cancers

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