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Abstract:
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In some rare disease areas and in pediatric population, enrollment challenges are unavoidable due to low prevalence. Sometimes, it is not ethical also to administer placebo in the clinical trial. Nowadays even in regulatory requirement trials and pivotal clinical trials, borrowing control information from historical trials and real-world data are being taken into considerations. Extrapolation from adult data to pediatric trials utilizing earlier treatment information are also in practice limiting exposure of children to unnecessary studies. Borrowing available control data or treatment data from outside utilize efficiently the limited resources to obtain trial results more quickly increasing the access to efficacious medications while maintaining the integrity of trial results. This roundtable will focus on the discussion of the design elements (as appropriate), commonly used statistical methodologies, practical challenges, and any concern to borrow or utilize data from outside clinical trial. The participants will get the opportunity to discuss their thoughts, experiences, and research works.
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