Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations
Stefanie Schoenen,
Johan Verbeeck,
Lukas Koletzko
et al.
Abstract:Background
The 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.
Methods
In very small populations, methodological… Show more
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