Lessons learned from IDeAl — 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials

Ralf-Dieter HilgersEmail author View ORCID ID profile,
Malgorzata Bogdan,
Carl-Fredrik Burman,
Holger Dette,
Mats Karlsson,
Franz König,
Christoph Male,
France Mentré,
Geert Molenberghs and
Stephen Senn

Orphanet Journal of Rare Diseases201813:77

https://doi.org/10.1186/s13023-018-0820-8

Received: 2 November 2017

Accepted: 1 May 2018

Published: 11 May 2018

Abstract

Background

IDeAl (Integrated designs and analysis of small population clinical trials) is an EU funded project developing new statistical design and analysis methodologies for clinical trials in small population groups. Here we provide an overview of IDeAl findings and give recommendations to applied researchers.

Method

The description of the findings is broken down by the nine scientific IDeAl work packages and summarizes results from the project’s more than 60 publications to date in peer reviewed journals. In addition, we applied text mining to evaluate the publications and the IDeAl work packages’ output in relation to the design and analysis terms derived from in the IRDiRC task force report on small population clinical trials.

Results

The results are summarized, describing the developments from an applied viewpoint. The main result presented here are 33 practical recommendations drawn from the work, giving researchers a comprehensive guidance to the improved methodology. In particular, the findings will help design and analyse efficient clinical trials in rare diseases with limited number of patients available. We developed a network representation relating the hot topics developed by the IRDiRC task force on small population clinical trials to IDeAl’s work as well as relating important methodologies by IDeAl’s definition necessary to consider in design and analysis of small-population clinical trials. These network representation establish a new perspective on design and analysis of small-population clinical trials.

Conclusion

IDeAl has provided a huge number of options to refine the statistical methodology for small-population clinical trials from various perspectives. A total of 33 recommendations developed and related to the work packages help the researcher to design small population clinical trial. The route to improvements is displayed in IDeAl-network representing important statistical methodological skills necessary to design and analysis of small-population clinical trials. The methods are ready for use.