Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations.

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 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.

Results

The methodological approaches cover methods for sound scientific modeling of natural his

SUBMITTER: Schoenen S 

PROVIDER: S-EPMC10909280 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature