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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 SmallPopulations Research (InSPiRe) project. This paper summarizes the main resultsof the project, which was completed in 2017. The InSPiRe project has led todevelopment of novel statistical methodology for clinical trials in smallpopulations in four areas. We have explored new decision-making methods forsmall population clinical trials using a Bayesian decision-theoretic frameworkto compare costs with potential benefits, developed approaches for targetedtreatment trials, enabling simultaneous identification of subgroups andconfirmation of treatment effect for these patients, worked on early phaseclinical 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 oftrials to support the planning, analysis and interpretation of a trial as wellas enabling extrapolation between patient groups. In addition to scientificpublications, we have contributed to regulatory guidance and produced freesoftware in order to facilitate implementation of the novel methods.

Recent advances in methodology for clinical trials in small populations: the InSPiRe project