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Activity Number: 304 - Extrapolation as a Default Strategy in Pediatric Drug Development: Implications to Clinical Trial Designs and Statistical Analysis
Type: Topic Contributed
Date/Time: Wednesday, August 5, 2020 : 10:00 AM to 11:50 AM
Sponsor: Biopharmaceutical Section
Abstract #312632
Title: Hierarchical Models for Clinical Trials Involving Multiple Pediatric Cohorts
Author(s): Margaret Gamalo-Siebers*
Companies: Eli Lilly & Co
Keywords: extrapolation; bayesian methods; hierarchical models; exchangeability

Depending on the degree of extrapolation, streamlining the pediatric drug development program is done with the awareness of the indication being pursued, the mechanism of action of the drug, and the strategic goal being pursued within a landscape to ensure timely access to drugs. With these considerations, there are a common development archetypes, e.g., whether to combine certain cohorts in single trial mindful of how it can inform the next cohort or analysis of the overall cohorts. This presentation will focus on two-level hierarchical models and how it fits into the overall pediatric clinical development. For this model, cohort treatment effects or means are generally assumed exchangeable, and patients are exchangeable within studies. We will compare this model with straightforward borrowing, e.g., power priors, and other data-driven dynamic priors. Analysis strategies for open label trials in younger cohorts within the hierarchical model will also be discussed as well as the assessment of relevant operating characteristics mindful the extrapolation strategy. The whole theoretical framework is balanced within the context of providing safe and effective medicines for children.

Authors who are presenting talks have a * after their name.

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