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Recent advances in methodology for clinical trials in small populations : the InSPiRe project
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(2018) Recent advances in methodology for clinical trials in small populations : the InSPiRe project. Orphanet Journal of Rare Diseases, 13 (1). 186. doi:10.1186/s13023-018-0919-y ISSN 1750-1172.
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WRAP-recent-advances-methodology-clinical-trials-small-populations-Stallard-2018.pdf - Published Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (1329Kb) | Preview |
Official URL: http://dx.doi.org/10.1186/s13023-018-0919-y
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.
Item Type: | Journal Article | ||||||
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Subjects: | R Medicine > R Medicine (General) | ||||||
Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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Library of Congress Subject Headings (LCSH): | Clinical trials -- Methodology, Clinical trials -- Statistics, Bayesian statistical decision theory | ||||||
Journal or Publication Title: | Orphanet Journal of Rare Diseases | ||||||
Publisher: | BioMed Central Ltd. | ||||||
ISSN: | 1750-1172 | ||||||
Official Date: | 25 October 2018 | ||||||
Dates: |
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Volume: | 13 | ||||||
Number: | 1 | ||||||
Article Number: | 186 | ||||||
DOI: | 10.1186/s13023-018-0919-y | ||||||
Status: | Peer Reviewed | ||||||
Publication Status: | Published | ||||||
Access rights to Published version: | Open Access (Creative Commons) | ||||||
Date of first compliant deposit: | 30 October 2018 | ||||||
Date of first compliant Open Access: | 30 October 2018 | ||||||
RIOXX Funder/Project Grant: |
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