ReviewOrphanet journal of rare diseases2018
Lessons learned from IDeAl - 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials.
Review in Orphanet journal of rare diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Systematic review of statistical methods for safety data in malaria chemoprevention in pregnancy trials.Malaria journal · 2020Pooled it
- Trial
- Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases.Annals of translational medicine · 2022Trial
- Overview of Regulatory Guidelines Related to Methods and Models for Small Populations.Clinical pharmacology and therapeutics · 2026Review
- Does randomization matter in rare disease clinical trials.PloS one · 2026Article
- Optimising clinical trial methods for complex regional pain syndrome: a methodological framework (OptiMeth-CRPS).Pain reports · 2025Article
- A Pharmacometrics-Informed Trial Simulation Framework for Optimizing Study Designs for Disease-Modifying Treatments in Rare Neurological Disorders.CPT: pharmacometrics & systems pharmacology · 2025Article
- Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges.Orphanet journal of rare diseases · 2024Review
- From Klinefelter Syndrome to High Grade Aneuploidies: Expanding the Gene-dosage Effect of Supernumerary X Chromosomes.The Journal of clinical endocrinology and metabolism · 2024Article
- Innovative methodologies for rare diseases clinical trials.Orphanet journal of rare diseases · 2024Article
- Statistical recommendations for count, binary, and ordinal data in rare disease cross-over trials.Orphanet journal of rare diseases · 2023Article
- Article
- Dwarfs on the Shoulders of Giants: Bayesian Analysis With Informative Priors in Elite Sports Research and Decision Making.Frontiers in sports and active living · 2022Article
- Designing Clinical Trials in Wilson's Disease.Hepatology (Baltimore, Md.) · 2021Article
- Randomized and non-randomized designs for causal inference with longitudinal data in rare disorders.Orphanet journal of rare diseases · 2021Review
- Design and analysis considerations for utilizing a mapping function in a small sample, sequential, multiple assignment, randomized trials with continuous outcomes.Statistics in medicine · 2021Article
- The Yin and the Yang of Transformative Research During the COVID-19 Pandemic-A Perspective.Frontiers in pediatrics · 2021Article
- Small molecule drug development for rare genodermatoses - evaluation of the current status in epidermolysis bullosa.Orphanet journal of rare diseases · 2020Review
- Biostatistics: a fundamental discipline at the core of modern health data science.The Medical journal of Australia · 2019Article
- The patient's view on rare disease trial design - a qualitative study.Orphanet journal of rare diseases · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
Abstract
backgroundIDeAl (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.
methodThe 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.
resultsThe 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.
conclusionIDeAl 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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.