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

Orphanet Journal of Rare Diseases
Ralf-Dieter HilgersStephen Senn

Abstract

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. 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. 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 ID...Continue Reading

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Citations

Jan 22, 2019·Stroke; a Journal of Cerebral Circulation·Lori C Jordan
Nov 8, 2018·Orphanet Journal of Rare Diseases·Simon DayNigel Stallard
Feb 9, 2019·Orphanet Journal of Rare Diseases·C M W GaasterlandJ H van der Lee
Oct 28, 2019·The Medical Journal of Australia·Katherine J LeeJohn B Carlin
Jul 13, 2021·Frontiers in Pediatrics·Venkatesh Sampath, Ramani Ramchandran
Jul 29, 2021·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·Peter OttMichael L Schilsky

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Methods Mentioned

BETA
Structure
Transcription Assay
PCA

Software Mentioned

EffectTreat
R package “ EffectTreat
R Package
R package “ geneSLOPE
- net 2
IRDiRC
Mod
IDeAl
R package “ TestingSimilarity
R MIXFIM

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