The execution of clinical trials for rare diseases continues to face methodological challenges. The variable and often very small number of patients afflicted by these conditions exacerbates statistical difficulties, which unfortunately have received inadequate attention. This paper introduces the iSTORE project, shedding light on its objectives and methodological strategies.
In the realm of very limited populations, iSTORE acknowledges and seeks to address heightened methodological challenges. The overarching goal is to cultivate a comprehensive understanding of natural history course modeling by employing multiple endpoint methodologies, identifying subgroup similarities, and elevating the level of evidence.
The methodological approaches employed by iSTORE encompass the development of robust scientific models for natural history course data. This involves showcasing similarities among subgroups, defining and analyzing multiple endpoints, and quantifying the level of evidence in trials that are often hindered by bias.
iSTORE aims to make significant contributions to rare disease research by offering an informed perspective, enabling better planning of clinical trials. The anticipated outcomes of the project will not only enhance understanding but also facilitate the synchronization of methodological derivations and outline the transferability of these approaches. Read the full article here.