10.1186/s13023-018-0820-8
https://zenodo.org/records/1246330
oai:zenodo.org:1246330
Hilgers, Ralf-Dieter
Ralf-Dieter
Hilgers
0000-0002-5945-1119
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Bogdan, Malgorzata
Malgorzata
Bogdan
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Burman, Carl-Fredrik
Carl-Fredrik
Burman
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Dette, Holger
Holger
Dette
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Karlsson, Mats
Mats
Karlsson
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
König, Franz
Franz
König
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Male, Christoph
Christoph
Male
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Mentré, France
France
Mentré
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Molenberghs, Geert
Geert
Molenberghs
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Senn, Stephen
Stephen
Senn
Department of Medical Statistics, RWTH Aachen University, Pauwelsstr. 19, D-52074, Aachen, Germany
Lessons learned from IDeAl — 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials
Zenodo
2018
Statistical methodology
Statistical design
Statistical analysis
Small population clinical trials
Rare disease
2018-05-11
https://zenodo.org/communities/fp7-bmc
https://zenodo.org/communities/eu
Creative Commons Attribution 4.0 International
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.
European Commission
10.13039/501100000780
602552
Integrated DEsign and AnaLysis of small population group trials