Recent advances in methodology for clinical trials in small populations: the InSPiRe project
Abstract Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This led to an EU FP7 call for proposals in 2013. One of the three projects funded was the Innovative Methodology for...
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doaj-9b388cef7daa4326aa630be227e4381e2020-11-25T01:49:56ZengBMCOrphanet Journal of Rare Diseases1750-11722018-10-011311910.1186/s13023-018-0919-yRecent advances in methodology for clinical trials in small populations: the InSPiRe projectTim Friede0Martin Posch1Sarah Zohar2Corinne Alberti3Norbert Benda4Emmanuelle Comets5Simon Day6Alex Dmitrienko7Alexandra Graf8Burak Kürsad Günhan9Siew Wan Hee10Frederike Lentz11Jason Madan12Frank Miller13Thomas Ondra14Michael Pearce15Christian Röver16Artemis Toumazi17Steffen Unkel18Moreno Ursino19Gernot Wassmer20Nigel Stallard21UMGSection of Medical Statistics, CeMSIIS, Medical University of ViennaINSERM, U1138, team 22, Centre de Recherche des Cordeliers, Université Paris 5, Université Paris 6INSERM, Hôpital Robert-Debré, APHP, University Paris 7BfArMINSERM, IAME, UMR 1137, Univ Paris Diderot, Sorbonne Paris CitéClinical Trials Consulting and Training LimitedMediana Inc.Section of Medical Statistics, CeMSIIS, Medical University of ViennaUMGWarwick Medical School, University of WarwickBfArMWarwick Medical School, University of WarwickDepartment of Statistics, Stockholm UniversitySection of Medical Statistics, CeMSIIS, Medical University of ViennaComplexity Science, University of WarwickUMGINSERM, Hôpital Robert-Debré, APHP, University Paris 7UMGINSERM, U1138, team 22, Centre de Recherche des Cordeliers, Université Paris 5, Université Paris 6Section of Medical Statistics, CeMSIIS, Medical University of ViennaWarwick Medical School, University of WarwickAbstract Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This led to an EU FP7 call for proposals in 2013. One of the three projects funded was the Innovative Methodology for Small Populations Research (InSPiRe) project. This paper summarizes the main results of the project, which was completed in 2017. The InSPiRe project has led to development of novel statistical methodology for clinical trials in small populations in four areas. We have explored new decision-making methods for small population clinical trials using a Bayesian decision-theoretic framework to compare costs with potential benefits, developed approaches for targeted treatment trials, enabling simultaneous identification of subgroups and confirmation of treatment effect for these patients, worked on early phase clinical trial design and on extrapolation from adult to pediatric studies, developing methods to enable use of pharmacokinetics and pharmacodynamics data, and also developed improved robust meta-analysis methods for a small number of trials to support the planning, analysis and interpretation of a trial as well as enabling extrapolation between patient groups. In addition to scientific publications, we have contributed to regulatory guidance and produced free software in order to facilitate implementation of the novel methods.http://link.springer.com/article/10.1186/s13023-018-0919-yFP7 small populations methodology projectsStatistical methodsRare disease clinical trial |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Tim Friede Martin Posch Sarah Zohar Corinne Alberti Norbert Benda Emmanuelle Comets Simon Day Alex Dmitrienko Alexandra Graf Burak Kürsad Günhan Siew Wan Hee Frederike Lentz Jason Madan Frank Miller Thomas Ondra Michael Pearce Christian Röver Artemis Toumazi Steffen Unkel Moreno Ursino Gernot Wassmer Nigel Stallard |
spellingShingle |
Tim Friede Martin Posch Sarah Zohar Corinne Alberti Norbert Benda Emmanuelle Comets Simon Day Alex Dmitrienko Alexandra Graf Burak Kürsad Günhan Siew Wan Hee Frederike Lentz Jason Madan Frank Miller Thomas Ondra Michael Pearce Christian Röver Artemis Toumazi Steffen Unkel Moreno Ursino Gernot Wassmer Nigel Stallard Recent advances in methodology for clinical trials in small populations: the InSPiRe project Orphanet Journal of Rare Diseases FP7 small populations methodology projects Statistical methods Rare disease clinical trial |
author_facet |
Tim Friede Martin Posch Sarah Zohar Corinne Alberti Norbert Benda Emmanuelle Comets Simon Day Alex Dmitrienko Alexandra Graf Burak Kürsad Günhan Siew Wan Hee Frederike Lentz Jason Madan Frank Miller Thomas Ondra Michael Pearce Christian Röver Artemis Toumazi Steffen Unkel Moreno Ursino Gernot Wassmer Nigel Stallard |
author_sort |
Tim Friede |
title |
Recent advances in methodology for clinical trials in small populations: the InSPiRe project |
title_short |
Recent advances in methodology for clinical trials in small populations: the InSPiRe project |
title_full |
Recent advances in methodology for clinical trials in small populations: the InSPiRe project |
title_fullStr |
Recent advances in methodology for clinical trials in small populations: the InSPiRe project |
title_full_unstemmed |
Recent advances in methodology for clinical trials in small populations: the InSPiRe project |
title_sort |
recent advances in methodology for clinical trials in small populations: the inspire project |
publisher |
BMC |
series |
Orphanet Journal of Rare Diseases |
issn |
1750-1172 |
publishDate |
2018-10-01 |
description |
Abstract Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This led to an EU FP7 call for proposals in 2013. One of the three projects funded was the Innovative Methodology for Small Populations Research (InSPiRe) project. This paper summarizes the main results of the project, which was completed in 2017. The InSPiRe project has led to development of novel statistical methodology for clinical trials in small populations in four areas. We have explored new decision-making methods for small population clinical trials using a Bayesian decision-theoretic framework to compare costs with potential benefits, developed approaches for targeted treatment trials, enabling simultaneous identification of subgroups and confirmation of treatment effect for these patients, worked on early phase clinical trial design and on extrapolation from adult to pediatric studies, developing methods to enable use of pharmacokinetics and pharmacodynamics data, and also developed improved robust meta-analysis methods for a small number of trials to support the planning, analysis and interpretation of a trial as well as enabling extrapolation between patient groups. In addition to scientific publications, we have contributed to regulatory guidance and produced free software in order to facilitate implementation of the novel methods. |
topic |
FP7 small populations methodology projects Statistical methods Rare disease clinical trial |
url |
http://link.springer.com/article/10.1186/s13023-018-0919-y |
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