ArticleOrphanet journal of rare diseases2024
Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.
Article in Orphanet journal of rare diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- Areas of research priorities in epilepsy: A position paper of the European Reference Network for Rare and Complex Epilepsies, EpiCARE.Epilepsia open · 2026Article
- Optimising clinical trial methods for complex regional pain syndrome: a methodological framework (OptiMeth-CRPS).Pain reports · 2025Article
- Ketogenic Metabolism in Neurodegenerative Diseases: Mechanisms of Action and Therapeutic Potential.Metabolites · 2025Review
- Multi-stakeholder sessions on major innovation topics in rare disease clinical trials.Orphanet journal of rare diseases · 2024Article
- Innovative methodologies for rare diseases clinical trials.Orphanet journal of rare diseases · 2024Article
Corrections and comments
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Authors and funding
11 authors.
Funding
Abstract
backgroundThe conduct of rare disease clinical trials is still hampered by methodological problems. The number of patients suffering from a rare condition is variable, but may be very small and unfortunately statistical problems for small and finite populations have received less consideration. This paper describes the outline of the iSTORE project, its ambitions, and its methodological approaches.
methodsIn very small populations, methodological challenges exacerbate. iSTORE's ambition is to develop a comprehensive perspective on natural history course modelling through multiple endpoint methodologies, subgroup similarity identification, and improving level of evidence.
resultsThe methodological approaches cover methods for sound scientific modeling of natural history course data, showing similarity between subgroups, defining, and analyzing multiple endpoints and quantifying the level of evidence in multiple endpoint trials that are often hampered by bias.
conclusionThrough its expected results, iSTORE will contribute to the rare diseases research field by providing an approach to better inform about and thus being able to plan a clinical trial. The methodological derivations can be synchronized and transferability will be outlined.
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